Ignite Creation Date:
2025-12-24 @ 6:37 PM
Ignite Modification Date:
2026-01-02 @ 8:36 AM
Study NCT ID:
NCT04678557
Status:
TERMINATED
Last Update Posted:
2023-10-18
First Post:
2020-11-30
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Evaluate Safety, Engraftment, and Efficacy of VC-01 in Subjects With T1 Diabetes Mellitus
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-05-03', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D003922', 'term': 'Diabetes Mellitus, Type 1'}], 'ancestors': [{'id': 'D003920', 'term': 'Diabetes Mellitus'}, {'id': 'D044882', 'term': 'Glucose Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medicalinfo@vrtx.com', 'phone': '617-341-6777', 'title': 'Medical Information', 'organization': 'Vertex'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Cohort 2 (Limitation): Stimulated C-peptide data from the MMTT at Baseline and Week 26 showed undetectable C-peptide (\\<0.1 ng/mL) for all but one subject. For those subjects, AUC were not generated and assumed "zero" as serial undetectable C-peptide results cannot create a curve.'}}, 'adverseEventsModule': {'timeFrame': 'Cohort 1 subjects reported adverse events over approximately 7 months (e.g., subjects implanted/treated for 6 months). Cohort 2 subjects reported adverse events over approximately 13 months (e.g., subjects implanted/treated for 12 months).', 'description': 'AE/SAE Definitions used are the same as the ClinicalTrials.gov definitions.\n\nSystematic Assessment of AE Collection was used (e.g., regular investigator assessment was required at each study visit per protocol). In addition, routine monitoring visits to the site provided 100% source document verification of the AE Collection.', 'eventGroups': [{'id': 'EG000', 'title': 'Sentinel Units (Aka Cohort 1)', 'description': 'VC-01 Combination Product; Up to ten (10) VC-01 sentinels\n\nVC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System', 'otherNumAtRisk': 17, 'deathsNumAtRisk': 17, 'otherNumAffected': 17, 'seriousNumAtRisk': 17, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Dose-finding Units (Aka Cohort 2)', 'description': 'VC-01 Combination Product; Up to twelve units implanted of which up to nine (9) are VC-01-DF (dose-finding) implants and the rest are VC-01 sentinels\n\nVC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System', 'otherNumAtRisk': 14, 'deathsNumAtRisk': 14, 'otherNumAffected': 14, 'seriousNumAtRisk': 14, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Abdominal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 6, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Complication of Device Removal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Implant Site Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 5, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Implant Site Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Infusion Site Haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Postoperative wound infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Incision site complication', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 6, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Incision site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Incision site haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Incision site haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Incision site hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Incision site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 30, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 60, 'numAffected': 12}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Incision site pruritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 6, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Incision site rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Incision site swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Muscle strain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Post procedural complication', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Post procedural contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 12, 'numAffected': 7}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Post procedural discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Post procedural haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Post procedural oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Post procedural swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 8, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Procedural nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Procedural pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 6, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 31, 'numAffected': 7}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Seroma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 10, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Migraine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Tension headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}], 'seriousEvents': [{'term': 'Vanishing Twin Syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Small Intestinal Obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Hypoglycemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Subarachnoid hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Cohort 1: The Percentage of Graft Cells Present at Post-implant Time Points Relative to Pre-clinical Models', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}, {'units': 'VC01 sentinels (contains PEC-01 cells)', 'counts': [{'value': '81', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Sentinel Units (Aka Cohort 1)', 'description': 'VC-01 Combination Product; Up to ten (10) VC-01 sentinels\n\nVC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System'}], 'classes': [{'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}, {'units': 'VC01 sentinels (contains PEC-01 cells)', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18.27', 'spread': '13.94', 'groupId': 'OG000'}]}]}, {'title': 'Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}, {'units': 'VC01 sentinels (contains PEC-01 cells)', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.23', 'spread': '4.63', 'groupId': 'OG000'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}, {'units': 'VC01 sentinels (contains PEC-01 cells)', 'counts': [{'value': '23', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.58', 'spread': '7.27', 'groupId': 'OG000'}]}]}, {'title': 'Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}, {'units': 'VC01 sentinels (contains PEC-01 cells)', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.02', 'spread': '8.09', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Weeks 4, 8, 12 and 26', 'description': "Through histology, the potential for functional engraftment of VC-01 combination product could be assessed in Cohort 1 subjects. Explanted sentinel units from subjects were processed and stained for markers identifying the number of graft cell nuclei (i.e., signifying the cells were viable). The percentage of viable graft cells in these explanted units were then compared to the percentage of viable graft cells from the pre-clinical (i.e., animal) models. These pre-clinical models are based on animal studies performed at ViaCyte (ref: internal study report). The data in Outcome Measure Data Table represent the % of viable graft cells present in the explanted sentinels compared to what was expected based on the animal model at Weeks 4, 8, 12, and 26 (e.g., At Week 4, there was an average of 18.27% viable graft cells in the participant's explants compared to what was expected based on the animal models).", 'unitOfMeasure': '%cells in sentinels rel to animal models', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'VC01 sentinels (contains PEC-01 cells)', 'denomUnitsSelected': 'VC01 sentinels (contains PEC-01 cells)', 'populationDescription': 'For each timepoint, explanted sentinel units containing PEC-01 cells from the 17 subjects were stained and evaluated for graft cell survival (i.e., viability). Not all subjects had units explanted at every timepoint, and units explanted that had "de minimus" graft cell survival (i.e., minimal, \\< 500 cells, \\< 4% of pre-clinical controls) could not be evaluated and were not included in the summary table.'}, {'type': 'PRIMARY', 'title': 'Cohort 2: The Change in AUC (Area Under Curve) From Baseline to Week 26 in C-peptide During 4-hour MMTT', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Dose-finding Units (Aka Cohort 2)', 'description': 'VC-01 Combination Product; Up to twelve units implanted of which up to nine (9) are VC-01-DF (dose-finding) implants and the rest are VC-01 sentinels\n\nVC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System'}], 'classes': [{'categories': [{'measurements': [{'value': '0.325', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'To Week 26', 'description': 'Evaluation of clinical efficacy of VC-01 combination product in Cohort 2 subjects was intended by measuring C-peptide levels during a 4-hour Mixed Meal Tolerance Test (MMTT). Blood glucose and C-peptide data were collected from subjects at timepoints 0, 30, 60, 90, 120, 180, 240 minutes after ingestion of a "meal" (i.e., BOOST drink). These C-peptide data points could be used to create the AUC calculation. If the implanted units contained mature, insulin-producing cells, stimulated C-peptide levels would be expected to increase over the time course in reaction to the meal.', 'unitOfMeasure': 'ng*h/mL', 'reportingStatus': 'POSTED', 'populationDescription': 'Stimulated C-peptide data from the MMTT showed every value from baseline and Week 26 samples at all timepoints to be "undetectable" (\\<0.1 ng/mL) except for one subject for whom the AUC could be calculated. Analyzing the data was deemed futile because the number of surviving graft cells was noted as insufficient for establishing the biological critical mass. The change from baseline to Week 26 in AUC for this one subject is provided below.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': '8 or 10 Sentinel Units Implanted (Cohort 1)', 'description': 'VC-01 combination product is comprised of PEC-01 cells loaded into an Encaptra Drug Delivery System. In Cohort 1 of this study, 8 or 10 VC-01 sentinel units were surgically implanted in each subject during a single procedure. Sentinel units are smaller versions of the product not intended to provide efficacy but are instead explanted at various timepoints over the course of the study and examined histologically.'}, {'id': 'FG001', 'title': '9 Dose Finding Units Implanted (Cohort 2)', 'description': 'A total of 12 VC-01 units were implanted in each of the Cohort 2 subjects in a single surgical procedure. Of those units, 9 were VC-01 Dose Finding (DF) units. DF units are larger than sentinels and intended to remain implanted during the duration of participation in the trial to assess efficacy.\n\nThe remaining 3 units implanted were the smaller VC-01 sentinel units intended for explant at various time points for analysis histologically.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '17'}, {'groupId': 'FG001', 'numSubjects': '14'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '16'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '14'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Lack of Efficacy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '13'}]}, {'type': 'Safety Evaluation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '31', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Sentinel Units (Aka Cohort 1)', 'description': 'VC-01 Combination Product; Up to ten (10) VC-01 sentinels\n\nVC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System'}, {'id': 'BG001', 'title': 'Dose-finding Units (Aka Cohort 2)', 'description': 'VC-01 Combination Product; Up to twelve units implanted of which up to nine (9) are VC-01-DF (dose-finding) implants and the rest are VC-01 sentinels\n\nVC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '37.0', 'spread': '13.11', 'groupId': 'BG000'}, {'value': '44.1', 'spread': '8.53', 'groupId': 'BG001'}, {'value': '40.2', 'spread': '11.7', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '28', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '17', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '31', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Body Mass Index', 'classes': [{'categories': [{'measurements': [{'value': '24.98', 'spread': '2.992', 'groupId': 'BG000'}, {'value': '27.28', 'spread': '2.807', 'groupId': 'BG001'}, {'value': '26.00', 'spread': '3.09', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kg/m^2', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Duration of Type 1 Diabetes', 'classes': [{'categories': [{'measurements': [{'value': '19.4', 'spread': '12.08', 'groupId': 'BG000'}, {'value': '26.4', 'spread': '11.24', 'groupId': 'BG001'}, {'value': '22.5', 'spread': '12.0', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-09-29', 'size': 1107123, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-03-28T16:08', 'hasProtocol': True}, {'date': '2021-03-15', 'size': 628085, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-03-28T16:31', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'OTHER', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'There are two Cohorts in this study design. Cohort 1 (Phase 1) enrolls up to 30 subjects total. After Cohort 1 enrollment is completed, Cohort 2 enrollment then occurs with up to an additional 40 subjects. A total of up to 70 subjects will be enrolled under this Phase 1/2 protocol.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 31}}, 'statusModule': {'whyStopped': 'Insufficient functional product engraftment', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2019-06-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-09', 'completionDateStruct': {'date': '2021-11-19', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-09-28', 'studyFirstSubmitDate': '2020-11-30', 'resultsFirstSubmitDate': '2023-04-03', 'studyFirstSubmitQcDate': '2020-12-16', 'lastUpdatePostDateStruct': {'date': '2023-10-18', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-09-28', 'studyFirstPostDateStruct': {'date': '2020-12-22', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-10-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-11-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Cohort 1: The Percentage of Graft Cells Present at Post-implant Time Points Relative to Pre-clinical Models', 'timeFrame': 'Weeks 4, 8, 12 and 26', 'description': "Through histology, the potential for functional engraftment of VC-01 combination product could be assessed in Cohort 1 subjects. Explanted sentinel units from subjects were processed and stained for markers identifying the number of graft cell nuclei (i.e., signifying the cells were viable). The percentage of viable graft cells in these explanted units were then compared to the percentage of viable graft cells from the pre-clinical (i.e., animal) models. These pre-clinical models are based on animal studies performed at ViaCyte (ref: internal study report). The data in Outcome Measure Data Table represent the % of viable graft cells present in the explanted sentinels compared to what was expected based on the animal model at Weeks 4, 8, 12, and 26 (e.g., At Week 4, there was an average of 18.27% viable graft cells in the participant's explants compared to what was expected based on the animal models)."}, {'measure': 'Cohort 2: The Change in AUC (Area Under Curve) From Baseline to Week 26 in C-peptide During 4-hour MMTT', 'timeFrame': 'To Week 26', 'description': 'Evaluation of clinical efficacy of VC-01 combination product in Cohort 2 subjects was intended by measuring C-peptide levels during a 4-hour Mixed Meal Tolerance Test (MMTT). Blood glucose and C-peptide data were collected from subjects at timepoints 0, 30, 60, 90, 120, 180, 240 minutes after ingestion of a "meal" (i.e., BOOST drink). These C-peptide data points could be used to create the AUC calculation. If the implanted units contained mature, insulin-producing cells, stimulated C-peptide levels would be expected to increase over the time course in reaction to the meal.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Type 1 Diabetes']}, 'descriptionModule': {'briefSummary': 'VC01-103 will evaluate an experimental combination product, cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes.', 'detailedDescription': 'This trial will test if VC-01 combination product can be implanted and maintained with safety, tolerability, and efficacy for up to Month 12/Week 52.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Men and non-pregnant women\n* Diagnosis of T1DM for a minimum of 3 years.\n* Stable, optimized diabetic regimen\n* Acceptable candidate for implant and explant procedures.\n* Willing and able to comply with protocol requirements.\n* Meet insulin dosing requirements per protocol\n\nExclusion Criteria:\n\n• Advanced complications associated with diabetes'}, 'identificationModule': {'nctId': 'NCT04678557', 'acronym': 'VC01-103', 'briefTitle': 'A Study to Evaluate Safety, Engraftment, and Efficacy of VC-01 in Subjects With T1 Diabetes Mellitus', 'organization': {'class': 'INDUSTRY', 'fullName': 'ViaCyte'}, 'officialTitle': 'An Open-Label Phase 1/2 Study to Evaluate the Safety, Engraftment, and Efficacy of VC-01™Combination Product in Subjects With Type 1 Diabetes Mellitus [T1DM]', 'orgStudyIdInfo': {'id': 'VC01-103'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Sentinel units (aka Cohort 1)', 'description': 'VC-01 Combination Product; Up to ten (10) VC-01 sentinels', 'interventionNames': ['Combination Product: VC-01 Combination Product']}, {'type': 'EXPERIMENTAL', 'label': 'Dose-finding units (aka Cohort 2)', 'description': 'VC-01 Combination Product; Up to twelve units implanted of which up to nine (9) are VC-01-DF (dose-finding) implants and the rest are VC-01 sentinels', 'interventionNames': ['Combination Product: VC-01 Combination Product']}], 'interventions': [{'name': 'VC-01 Combination Product', 'type': 'COMBINATION_PRODUCT', 'otherNames': ['PEC-Encap'], 'description': 'PEC-01 cells loaded into an Encaptra Drug Delivery System', 'armGroupLabels': ['Dose-finding units (aka Cohort 2)', 'Sentinel units (aka Cohort 1)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92025', 'city': 'Escondido', 'state': 'California', 'country': 'United States', 'facility': 'AMCR Institute', 'geoPoint': {'lat': 33.11921, 'lon': -117.08642}}, {'zip': '94598', 'city': 'Walnut Creek', 'state': 'California', 'country': 'United States', 'facility': 'Diablo Clinical Research', 'geoPoint': {'lat': 37.90631, 'lon': -122.06496}}, {'zip': '30318', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Atlanta Diabetes Associates', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '78731', 'city': 'Austin', 'state': 'Texas', 'country': 'United States', 'facility': 'Texas Diabetes & Endocrinology', 'geoPoint': {'lat': 30.26715, 'lon': -97.74306}}], 'overallOfficials': [{'name': 'Manasi Jaiman, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'ViaCyte, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'ViaCyte', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}