Ignite Creation Date:
2025-12-25 @ 12:34 AM
Ignite Modification Date:
2025-12-25 @ 10:43 PM
Study NCT ID:
NCT01222767
Status:
COMPLETED
Last Update Posted:
2021-10-29
First Post:
2010-10-08
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Study of Zalypsis® (PM00104) in Patients With Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D012512', 'term': 'Sarcoma, Ewing'}, {'id': 'D018242', 'term': 'Neuroectodermal Tumors, Primitive'}], 'ancestors': [{'id': 'D012516', 'term': 'Osteosarcoma'}, {'id': 'D018213', 'term': 'Neoplasms, Bone Tissue'}, {'id': 'D009372', 'term': 'Neoplasms, Connective Tissue'}, {'id': 'D018204', 'term': 'Neoplasms, Connective and Soft Tissue'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D012509', 'term': 'Sarcoma'}, {'id': 'D018302', 'term': 'Neoplasms, Neuroepithelial'}, {'id': 'D017599', 'term': 'Neuroectodermal Tumors'}, {'id': 'D009373', 'term': 'Neoplasms, Germ Cell and Embryonal'}, {'id': 'D009375', 'term': 'Neoplasms, Glandular and Epithelial'}, {'id': 'D009380', 'term': 'Neoplasms, Nerve Tissue'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C500383', 'term': 'PM 00104'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@pharmamar.com', 'phone': '0034 91846 60 00', 'title': 'Clinical Developtment, Department of PharmaMar´s Oncology., Business Unit.', 'organization': 'Pharma Mar S.A.'}, 'certainAgreement': {'otherDetails': 'The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From first infusion to study termination, up to 2 years', 'description': 'Sixteen of 17 patients included in this study were treated with PM00104 and were therefore evaluable for safety', 'eventGroups': [{'id': 'EG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks", 'otherNumAtRisk': 16, 'deathsNumAtRisk': 16, 'otherNumAffected': 16, 'seriousNumAtRisk': 16, 'deathsNumAffected': 7, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 11, 'numAffected': 7}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pericardial effusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Ear congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Eye swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Myopia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Vision blurred', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 4, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Stomach discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Stomatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Chills', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 10, 'numAffected': 7}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Non-cardiac chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Breath sounds abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Anorexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Hyperglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Hypocalcaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Hypokalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Bone pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Tumour pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Depressed level of consciousness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dysgeusia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Peripheral sensory neuropathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Urinary retention', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 10, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dry throat', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dyspnoea exertional', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Hypoxia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Oropharyngeal blistering', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pleuritic pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pneumothorax', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Throat irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Alopecia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Hyperhidrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Night sweats', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pallor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Superior vena caval occlusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Venous thrombosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}], 'seriousEvents': [{'term': 'Acute sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Overall Response Rate (ORR)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'At baseline and every other cycle (± 1 week) until evidence of PD, up to 2 years', 'description': 'Overall response rate (ORR), defined as the percentage of patients with confirmed objective response (OR), either CR or PR according to the RECIST v.1.1.\n\nCR, complete response: disappearance of all lesions; PD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density; SD, stable disease: none of the CR, PR, or PD criteria met; RECIST, Response Evaluation Criteria in Solid Tumors', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'One patient never treated'}, {'type': 'SECONDARY', 'title': 'Best Tumor Response', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'title': 'SD', 'measurements': [{'value': '4', 'groupId': 'OG000'}]}, {'title': 'PD', 'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'At baseline and every other cycle (± 1 week) until evidence of PD, up to 2 years', 'description': 'Best tumor response was defined as the best response achieved during the study according to RECIST v1.1 CR, complete response: disappearance of all lesions; PD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density; SD, stable disease: none of the CR, PR, or PD criteria met; RECIST, Response Evaluation Criteria in Solid Tumors', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'One patient never treated Two patients were non-evaluable for efficacy because they were withdrawn due to toxicity without any tumor assessment after the start of study treatment and were considered as "treatment failures"'}, {'type': 'SECONDARY', 'title': 'Progression-free Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': '1.8', 'groupId': 'OG000', 'lowerLimit': '0.9', 'upperLimit': '3.5'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'From the first day of study treatment to the day of negative assessment (progression or death), start of subsequent antitumor therapy, or last tumor evaluation, up to 2 years', 'description': 'Progression-free survival (PFS), defined as the time from the first day of study treatment to the day of negative assessment (progression or death), start of subsequent antitumor therapy, or last tumor evaluation.\n\nPD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density', 'unitOfMeasure': 'months', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'One patient never treated'}, {'type': 'SECONDARY', 'title': 'Progression-free Survival at 3 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': '28.6', 'groupId': 'OG000', 'lowerLimit': '4.9', 'upperLimit': '52.2'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At 3 months', 'description': 'Progression-free survival (PFS), defined as the time from the first day of study treatment to the day of negative assessment (progression or death), start of subsequent antitumor therapy, or last tumor evaluation.\n\nPD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'One patient never treated'}, {'type': 'SECONDARY', 'title': 'Overall Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': 'NA', 'comment': 'Median survival could not be calculated because more than half of patients were still living', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'from the first day of treatment to the date of death, up to 2 years', 'description': 'Overall survival (OS), defined as the time from the first day of treatment to the date of death (or the last day when the patient was known to be alive). Survival was to be followed for up to six months after the last treatment visit of the last patient, or 12 months after the last patient was included, whichever occurred first.', 'unitOfMeasure': 'months', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'One patient never treated'}, {'type': 'SECONDARY', 'title': 'Overall Survival Rate at 6 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': '62.5', 'groupId': 'OG000', 'lowerLimit': '38.8', 'upperLimit': '86.2'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At 6 months', 'description': 'Overall survival (OS), defined as the time from the first day of treatment to the date of death (or the last day when the patient was known to be alive). Survival was to be followed for up to six months after the last treatment visit of the last patient, or 12 months after the last patient was included, whichever occurred first.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'One patient never treated'}, {'type': 'SECONDARY', 'title': 'Overall Survival Rate at 12 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': '54.7', 'groupId': 'OG000', 'lowerLimit': '29.5', 'upperLimit': '79.9'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At 12 months', 'description': 'Overall survival (OS), defined as the time from the first day of treatment to the date of death (or the last day when the patient was known to be alive). Survival was to be followed for up to six months after the last treatment visit of the last patient, or 12 months after the last patient was included, whichever occurred first.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'One patient never treated'}, {'type': 'SECONDARY', 'title': 'PM00104 Plasma PK Parameters (Cmax) at First Infusion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': '21.23', 'spread': '8.35', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (Pre-infusion) and 5 min, 30 min, 2 hours, 6 hours, 24 hours, 48 hours and 168 hours after the end of first infusion (Day 1)', 'description': 'Cmax Maximum plasma concentration, directly determined from the experimental data', 'unitOfMeasure': 'μg/l', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Fourteen of the 16 patients treated in this study had plasma profiles'}, {'type': 'SECONDARY', 'title': 'PM00104 Plasma PK Parameters (AUC) at First Infusion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': '87.06', 'spread': '75.49', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (Pre-infusion) and 5 min, 30 min, 2 hours, 6 hours, 24 hours, 48 hours and 168 hours after the end of first infusion (Day 1)', 'description': 'AUC Area under the plasma concentration-time curve from time zero to infinity', 'unitOfMeasure': 'h*μg/l', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Fourteen of the 16 patients treated in this study had plasma profiles'}, {'type': 'SECONDARY', 'title': 'PM00104 Plasma PK Parameters (Cmax) at Second Infusion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': '22.37', 'spread': '8.77', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (Pre-infusion) and 5 min, 30 min, 2 hours, 6 hours, 24 hours, 48 hours and 168 hours after the end of second infusion (Day 8)', 'description': 'Cmax Maximum plasma concentration, directly determined from the experimental data', 'unitOfMeasure': 'μg/l', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Eleven of the 16 patients treated in this study had plasma profiles at second infusion'}, {'type': 'SECONDARY', 'title': 'PM00104 Plasma PK Parameters (AUC) at Second Infusion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'classes': [{'categories': [{'measurements': [{'value': '69.76', 'spread': '17.69', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (Pre-infusion) and 5 min, 30 min, 2 hours, 6 hours, 24 hours, 48 hours and 168 hours after the end of second infusion (Day 8)', 'description': 'AUC Area under the plasma concentration-time curve from time zero to infinity', 'unitOfMeasure': 'h*μg/l', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Eleven of the 16 patients treated in this study had plasma profiles at second infusion'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '17'}]}, {'type': 'Received Treatment', 'achievements': [{'groupId': 'FG000', 'numSubjects': '16'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '17'}]}], 'dropWithdraws': [{'type': 'Progressive disease', 'reasons': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Surgical resection', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Never treated', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'A total of 17 patients were included and 16 of them were treated with PM00104 at five investigational sites from the USA (n=3), France and Italy. The patients participated in this study between 22 December 2010 (first consent) and 21 May 2012 (last follow-up). First and last doses were administered on 4 January 2011 and 24 January 2012, respectively'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'PM00104', 'description': "PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks"}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'title': '<=18 years', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '23', 'groupId': 'BG000', 'lowerLimit': '15', 'upperLimit': '53'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race and Ethnicity Not Collected', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Race and Ethnicity were not collected from any participant.'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}]}]}, {'title': 'Italy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}, {'title': 'France', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'ECOG PS', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'title': '0', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': '1', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': '2', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Eastern Cooperative Oncology Group Performance Status, ECOG PS 0 Fully active, able to carry on all pre-disease performance without restriction\n\n1. Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature\n2. Ambulatory and capable of all selfcare but unable to carry out any work activities. Up and about more than 50% of waking hours\n3. Capable of only limited selfcare, confined to bed or chair more than 50% of waking hours\n4. Completely disabled. Cannot carry on any selfcare. Totally confined to bed or chair\n5. Dead', 'unitOfMeasure': 'Participants'}, {'title': 'Tumor diagnosis', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'title': "Ewing's bone sarcoma", 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'Extraosseous sarcoma', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Primary location at diagnosis', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Lower extremity', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'Trunk/abdominal wall', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'Upper extremity', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Unknown', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Metastatic disease', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '17', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Time from diagnosis to first infusion', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '43.3', 'groupId': 'BG000', 'lowerLimit': '13.8', 'upperLimit': '81.3'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'months', 'dispersionType': 'FULL_RANGE'}, {'title': 'Time from last disease progression to first infusion', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'groupId': 'BG000', 'lowerLimit': '0.1', 'upperLimit': '1.8'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'months', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sites of disease at diagnosis', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'title': '1', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': '2', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': '3', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': '>3', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Prior radiotherapy', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Prior Antitumor surgery (palliative or curative)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Prior Systemic anticancer therapy', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '17', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2010-07-30', 'size': 869340, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2021-06-10T08:55', 'hasProtocol': True}, {'date': '2013-02-07', 'size': 1195380, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2021-06-10T08:55', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 17}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-07', 'completionDateStruct': {'date': '2012-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-09-30', 'studyFirstSubmitDate': '2010-10-08', 'resultsFirstSubmitDate': '2021-07-13', 'studyFirstSubmitQcDate': '2010-10-15', 'lastUpdatePostDateStruct': {'date': '2021-10-29', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-09-30', 'studyFirstPostDateStruct': {'date': '2010-10-18', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-10-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2012-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Overall Response Rate (ORR)', 'timeFrame': 'At baseline and every other cycle (± 1 week) until evidence of PD, up to 2 years', 'description': 'Overall response rate (ORR), defined as the percentage of patients with confirmed objective response (OR), either CR or PR according to the RECIST v.1.1.\n\nCR, complete response: disappearance of all lesions; PD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density; SD, stable disease: none of the CR, PR, or PD criteria met; RECIST, Response Evaluation Criteria in Solid Tumors'}], 'secondaryOutcomes': [{'measure': 'Best Tumor Response', 'timeFrame': 'At baseline and every other cycle (± 1 week) until evidence of PD, up to 2 years', 'description': 'Best tumor response was defined as the best response achieved during the study according to RECIST v1.1 CR, complete response: disappearance of all lesions; PD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density; SD, stable disease: none of the CR, PR, or PD criteria met; RECIST, Response Evaluation Criteria in Solid Tumors'}, {'measure': 'Progression-free Survival', 'timeFrame': 'From the first day of study treatment to the day of negative assessment (progression or death), start of subsequent antitumor therapy, or last tumor evaluation, up to 2 years', 'description': 'Progression-free survival (PFS), defined as the time from the first day of study treatment to the day of negative assessment (progression or death), start of subsequent antitumor therapy, or last tumor evaluation.\n\nPD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density'}, {'measure': 'Progression-free Survival at 3 Months', 'timeFrame': 'At 3 months', 'description': 'Progression-free survival (PFS), defined as the time from the first day of study treatment to the day of negative assessment (progression or death), start of subsequent antitumor therapy, or last tumor evaluation.\n\nPD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density'}, {'measure': 'Overall Survival', 'timeFrame': 'from the first day of treatment to the date of death, up to 2 years', 'description': 'Overall survival (OS), defined as the time from the first day of treatment to the date of death (or the last day when the patient was known to be alive). Survival was to be followed for up to six months after the last treatment visit of the last patient, or 12 months after the last patient was included, whichever occurred first.'}, {'measure': 'Overall Survival Rate at 6 Months', 'timeFrame': 'At 6 months', 'description': 'Overall survival (OS), defined as the time from the first day of treatment to the date of death (or the last day when the patient was known to be alive). Survival was to be followed for up to six months after the last treatment visit of the last patient, or 12 months after the last patient was included, whichever occurred first.'}, {'measure': 'Overall Survival Rate at 12 Months', 'timeFrame': 'At 12 months', 'description': 'Overall survival (OS), defined as the time from the first day of treatment to the date of death (or the last day when the patient was known to be alive). Survival was to be followed for up to six months after the last treatment visit of the last patient, or 12 months after the last patient was included, whichever occurred first.'}, {'measure': 'PM00104 Plasma PK Parameters (Cmax) at First Infusion', 'timeFrame': '0 (Pre-infusion) and 5 min, 30 min, 2 hours, 6 hours, 24 hours, 48 hours and 168 hours after the end of first infusion (Day 1)', 'description': 'Cmax Maximum plasma concentration, directly determined from the experimental data'}, {'measure': 'PM00104 Plasma PK Parameters (AUC) at First Infusion', 'timeFrame': '0 (Pre-infusion) and 5 min, 30 min, 2 hours, 6 hours, 24 hours, 48 hours and 168 hours after the end of first infusion (Day 1)', 'description': 'AUC Area under the plasma concentration-time curve from time zero to infinity'}, {'measure': 'PM00104 Plasma PK Parameters (Cmax) at Second Infusion', 'timeFrame': '0 (Pre-infusion) and 5 min, 30 min, 2 hours, 6 hours, 24 hours, 48 hours and 168 hours after the end of second infusion (Day 8)', 'description': 'Cmax Maximum plasma concentration, directly determined from the experimental data'}, {'measure': 'PM00104 Plasma PK Parameters (AUC) at Second Infusion', 'timeFrame': '0 (Pre-infusion) and 5 min, 30 min, 2 hours, 6 hours, 24 hours, 48 hours and 168 hours after the end of second infusion (Day 8)', 'description': 'AUC Area under the plasma concentration-time curve from time zero to infinity'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['EFT', 'PNET', 'EOE'], 'conditions': ["Ewing's Sarcoma", 'Primitive Neuroectodermal Tumor (PNET)', "Askin's Tumor of the Chest Wall", "Extraosseous Ewing's Sarcoma (EOE)"]}, 'descriptionModule': {'briefSummary': 'This is a phase II Multicenter, Open-label, Clinical and Pharmacokinetic Study of Zalypsis® (PM00104) in Patients with Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy to determine the antitumor activity of Zalypsis.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Voluntary written informed consent, obtained from the patient or his/her representative before the beginning of any specific study procedures.\n2. Age ≥ 16 years.\n3. Histologically or cytologically confirmed EFT (Ewing Family of Tumors), with recurrent disease.\n4. Documented failure to at least one prior chemotherapy regimen for their disease.\n5. Radiographic documentation of disease progression at study entry.\n6. Eastern Cooperative Oncology Group (ECOG) performance status (PS) score ≤ 2.\n7. Life expectancy ≥ 3 months.\n8. Complete recovery from the effects of drug-related adverse events (AEs) derived from previous treatments, excluding alopecia and grade 1 peripheral neuropathy, according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) v. 4.0.\n9. At least one measurable lesion ("target lesion" according to the RECIST v.1.1), located in a non-irradiated area and adequately measured less than four weeks before study entry. Tumors within a previously irradiated field will be designated as "non-target" lesions unless progression is clearly documented or biopsy proven.\n10. Absolute neutrophil count (ANC) ≥ 1.5 x 109/l; platelet count ≥ 100 x 109/l, and hemoglobin ≥ 9 g/dl.\n11. Adequate renal function: calculated creatinine clearance (using Cockcroft and Gault\'s formula) ≥ 30 ml/min.\n12. Adequate hepatic function:\n\n * Total bilirubin ≤ 1.5 x upper limit or normality (ULN), unless due to Gilbert\'s syndrome.\n * Alanine aminotransferase (ALT), aspartate aminotransferase (AST) ≤ 3 x ULN (≤ 5 x ULN in case of hepatic metastases), and alkaline phosphatase (AP) ≤ 2.5 x ULN (≤ 5 x ULN in case of extensive bone involvement).\n * Albumin ≥ 25 g/l.\n13. Left ventricular ejection fraction (LVEF) within normal limits (LVEF of at least 50%).\n14. Women of childbearing potential must have a negative serum pregnancy test before study entry. Both women and men must agree to use a medically acceptable method of contraception throughout the treatment period and for three months after discontinuation of treatment. Acceptable methods of contraception include complete abstinence, intrauterine device (IUD), oral contraceptive, subdermal implant and double barrier (condom with a contraceptive sponge or contraceptive suppository).\n\nExclusion Criteria:\n\n1. Prior therapy with Zalypsis®.\n2. Pregnant or lactating women or women of childbearing potential not using an appropriate contraceptive method.\n3. Less than three weeks from prior radiation therapy, biological therapy or chemotherapy.\n4. Less than six weeks from prior nitrosourea, mitomycin C, high-dose chemotherapy or radiotherapy involving the whole pelvis or over 50% of the spine, provided that acute effects of radiation treatment have resolved. Hormonal therapy and palliative radiation therapy (i.e., for control of pain from bone metastases) must be discontinued before study entry.\n5. Patients with a prior invasive malignancy (except non-melanoma skin cancer and in situ cervix carcinoma) who have had any evidence of disease within the last five years or whose prior malignancy treatment contraindicates the current protocol therapy.\n6. Evidence of progressive or symptomatic central nervous system (CNS) metastases or leptomeningeal metastases.\n7. Other diseases or serious conditions:\n\n * Increased cardiac risk, as defined by:\n\n * Unstable angina or myocardial infarction within 12 months before inclusion in the study.\n * New York Heart Association (NYHA) grade II or greater congestive heart failure.\n * Symptomatic arrhythmia or any arrhythmia requiring ongoing treatment.\n * Abnormal electrocardiogram (ECG), i.e., patients with the following are excluded: QT prolongation - QTc \\> 480 msec; signs of cardiac enlargement or hypertrophy; bundle branch block; partial blocks; signs of ischemia or necrosis, and Wolff Parkinson White patterns.\n * History or presence of valvular heart disease.\n * Uncontrolled arterial hypertension despite optimal medical therapy.\n * Previous mediastinal radiotherapy.\n * Previous treatment with doxorubicin at cumulative doses exceeding 400 mg/m2.\n * History of significant neurological or psychiatric disorders.\n * Active infection requiring systemic treatment.\n * Significant non-neoplastic liver disease (e.g., cirrhosis).\n * Known hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.\n * Immunocompromised patients, including those known to be infected with the human immunodeficiency virus (HIV).\n * Uncontrolled (i.e., requiring relevant changes in medication within the last month or hospital admission within the last three months) endocrine diseases (e.g., diabetes mellitus, hypo- or hyperthyroidism, adrenal disorder).\n8. Any other major illness that, in the Investigator\'s judgment, will substantially increase the risk associated with the patient\'s participation in the study. The Investigator should feel free to consult the Study Coordinator or the Sponsor(s) in case of uncertainty in this regard.\n9. Limitation of the patient\'s ability to comply with the treatment or to follow-up at a participating center. Patients enrolled into this trial must be treated and followed at a participating center.\n10. Treatment with any investigational product within 30 days prior to inclusion in the study.\n11. Known hypersensitivity to any component of Zalypsis®.'}, 'identificationModule': {'nctId': 'NCT01222767', 'briefTitle': 'Study of Zalypsis® (PM00104) in Patients With Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy', 'organization': {'class': 'INDUSTRY', 'fullName': 'PharmaMar'}, 'officialTitle': 'Phase II Multicenter, Open-label, Clinical and Pharmacokinetic Study of Zalypsis® (PM00104) in Patients With Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy', 'orgStudyIdInfo': {'id': 'PM104-B-003-10'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Arm 1', 'interventionNames': ['Drug: Zalypsis']}], 'interventions': [{'name': 'Zalypsis', 'type': 'DRUG', 'otherNames': ['PM00104'], 'description': 'Zalypsis is provided as a lyophilized powder for concentrate for solution for infusion in a strength of 2.5 mg/vial.', 'armGroupLabels': ['Arm 1']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90403', 'city': 'Santa Monica', 'state': 'California', 'country': 'United States', 'facility': 'Sarcoma Oncology Center', 'geoPoint': {'lat': 34.01949, 'lon': -118.49138}}, {'zip': '38105A', 'city': 'Memphis', 'state': 'Tennessee', 'country': 'United States', 'facility': "St. Jude Children 's Research Hospital", 'geoPoint': {'lat': 35.14953, 'lon': -90.04898}}, {'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': 'Seattle Cancer Care Alliance', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '69373', 'city': 'Lyon', 'country': 'France', 'facility': 'Centre Léon Bérard', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'zip': '40136', 'city': 'Bologna', 'country': 'Italy', 'facility': 'Istituto Ortopedici Rizzoli', 'geoPoint': {'lat': 44.49381, 'lon': 11.33875}}, {'zip': '20133', 'city': 'Milan', 'country': 'Italy', 'facility': 'Istituto Nazionale dei Tumori', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}, {'zip': '20089', 'city': 'Rozzano', 'country': 'Italy', 'facility': 'Istituto Clinico Humanitas', 'geoPoint': {'lat': 45.38193, 'lon': 9.1559}}], 'overallOfficials': [{'name': 'Fariba Navid, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "St. Jude Children 's Research Hospital"}, {'name': 'Sant P Chawla, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Sarcoma Oncology Center'}, {'name': 'Jean Yves Blay, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Centre Leon Berard'}, {'name': 'Stefano Ferrari, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Istituto Ortopedici Rizzoli'}, {'name': 'Armando Santoro, Prof.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Istituto Clinico Humanitas'}, {'name': 'Paolo Casali, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Fondazione IRCCS Istituto Nazionale dei Tumori, Milano'}, {'name': 'Robin L. Jones, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Seattle Cancer Care Alliance'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'PharmaMar', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}