Ignite Creation Date:
2025-12-24 @ 4:05 PM
Ignite Modification Date:
2025-12-25 @ 6:27 PM
Study NCT ID:
NCT00301366
Status:
COMPLETED
Last Update Posted:
2014-08-19
First Post:
2006-03-08
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency
Sponsor:
Organization:
Raw JSON
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This will be reviewed promptly and approval will not be withheld unreasonably. In case of a difference of opinion between the sponsor and the investigator(s), the contents of the publication will be discussed in order to find a solution that satisfies both parties.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Alpha-1MP Treatment Group', 'description': 'All subjects received open-label weekly IV infusions of 60 mg/kg body weight of functional Alpha-1 MP for 20 weeks', 'otherNumAtRisk': 38, 'otherNumAffected': 26, 'seriousNumAtRisk': 38, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'General disorders'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders'}, {'term': 'Chills', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Infections and infestations'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Nervous system disorders'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders'}, {'term': 'Pharyngolaryngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders'}, {'term': 'Hot flush', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Vascular disorders'}], 'seriousEvents': [{'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 38, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Alpha-1 MP Treatment Group'}], 'classes': [{'title': 'Number of Participants Analyzed', 'categories': [{'measurements': [{'value': '38', 'groupId': 'OG000'}]}]}, {'title': 'Subjects experiencing TEAEs', 'categories': [{'measurements': [{'value': '26', 'groupId': 'OG000'}]}]}, {'title': 'Subjects with TEAEs <24 hrs. of A-1 MP infusion', 'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000'}]}]}, {'title': 'Alpha-1 MP subjects with ≥1 drug-related TEAE', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '24 weeks', 'description': 'An adverse event is any untoward medical occurrence in a subject or clinical investigation subject administered with a pharmaceutical product. The adverse event does not necessarily have to have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the medicinal product.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': '38 subjects received study medication and one subject discontinued from the study due to an AE. Therefore, 37 subjects completed the study. 18 subjects were naive ((i.e., never having received previous Alpha-1 protease inhibitor augmentation therapy) and 19 subjects were non-naive.', 'anticipatedPostingDate': '2009-09'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Alpha-1MP Treatment Group', 'description': 'All subjects received open-label weekly IV infusions of 60 mg/kg body weight of functional Alpha-1 MP for 20 weeks'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '38'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '37'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'First-subject-first-dose was 12 June 2006, last-subject-last-visit was 20 March 2007. The study was performed in 10 clinical sites, 8 sites in the United States and 2 sites in the United Kingdom.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Entered Study'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '32', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '53.4', 'spread': '8.63', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '15', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '23', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '35', 'groupId': 'BG000'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 38}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2006-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-07', 'completionDateStruct': {'date': '2007-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-07-30', 'studyFirstSubmitDate': '2006-03-08', 'resultsFirstSubmitDate': '2009-08-28', 'studyFirstSubmitQcDate': '2006-03-08', 'lastUpdatePostDateStruct': {'date': '2014-08-19', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2014-07-30', 'studyFirstPostDateStruct': {'date': '2006-03-10', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-08-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2007-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.', 'timeFrame': '24 weeks', 'description': 'An adverse event is any untoward medical occurrence in a subject or clinical investigation subject administered with a pharmaceutical product. The adverse event does not necessarily have to have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the medicinal product.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['alpha 1-Antitrypsin Deficiency', 'alpha 1-Antitrypsin', 'pulmonary emphysema'], 'conditions': ['Alpha 1-Antitrypsin Deficiency']}, 'referencesModule': {'references': [{'pmid': '3494198', 'type': 'BACKGROUND', 'citation': 'Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, Crystal RG. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med. 1987 Apr 23;316(17):1055-62. doi: 10.1056/NEJM198704233161704.'}, {'pmid': '7028785', 'type': 'BACKGROUND', 'citation': 'Gadek JE, Klein HG, Holland PV, Crystal RG. Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects. J Clin Invest. 1981 Nov;68(5):1158-65. doi: 10.1172/jci110360.'}, {'pmid': '6169740', 'type': 'BACKGROUND', 'citation': 'Gadek JE, Fells GA, Zimmerman RL, Rennard SI, Crystal RG. Antielastases of the human alveolar structures. Implications for the protease-antiprotease theory of emphysema. J Clin Invest. 1981 Oct;68(4):889-98. doi: 10.1172/jci110344.'}], 'seeAlsoLinks': [{'url': 'http://www.alphaone.org', 'label': 'The "Alpha-1 Foundation", dedicated to providing the leadership and resources that will result in increased research, improved health, worldwide detection and a cure for Alpha-1 Antitrypsin Deficiency'}, {'url': 'http://www.alphanet.org', 'label': 'AlphaNet, Inc devoted to improving the lives of individuals with Alpha-1 antitrypsin deficiency through comprehensive disease management services, clinical research administration, and consultative services'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this clinical study is to assess the safety and tolerability of Alpha-1 MP in adult Alpha1-antitrypsin deficient patients.', 'detailedDescription': 'The objective of this clinical trial (STAMP: Safety and Tolerability of Alpha-1 Modified Process) is to study the safety and tolerability of Alpha-1 MP in adult Alpha 1-antitrypsin deficient subjects as reported over 20 weeks of therapy. The primary objective is to describe the nature and frequency of treatment-emergent adverse events with "treatment-emergent" defined as any adverse event occurring after the start of the first study drug infusion.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Documented diagnosis of congenital Alpha1-antitrypsin deficiency\n* Documented forced expiratory volume in 1 second (FEV1 ) between 20% - 80% of predicted value within last 6 months.\n* Signed written informed consent prior to initiation of any study related procedures.\n\nExclusion Criteria:\n\n* Females who are pregnant, breast feeding, or if of child-bearing potential, unwilling to practice adequate contraception throughout the study\n* Use of systemic steroids within the 2 weeks prior to receiving study treatment (this does not include the use of inhaled steroids used on a routine or as needed basis).\n* Subjects who have had exacerbations of their disease within one month of trial entry'}, 'identificationModule': {'nctId': 'NCT00301366', 'acronym': 'STAMP', 'briefTitle': 'The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency', 'organization': {'class': 'INDUSTRY', 'fullName': 'Grifols Therapeutics LLC'}, 'officialTitle': 'Multi-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) Deficiency', 'orgStudyIdInfo': {'id': '11815'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Alpha-1 Proteinase Inhibitor (Human), modified process', 'description': 'Study the safety and tolerability of weekly infusions of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP, 60 mg/kg) over 20 weeks of therapy in adult Alpha-1 antitrypsin deficient subjects.', 'interventionNames': ['Drug: alpha-1 proteinase inhibitor (human)']}], 'interventions': [{'name': 'alpha-1 proteinase inhibitor (human)', 'type': 'DRUG', 'otherNames': ['Prolastin', 'Alpha-1 antitrypsin (AAT)', 'BAY x 5747', 'BAY 10-5233', 'TAL-05-00007'], 'description': '60 mg/kg weekly for 20 weeks', 'armGroupLabels': ['Alpha-1 Proteinase Inhibitor (Human), modified process']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80206', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'National Jewish Medical and Research Center', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '32610-0225', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida College of Medicine', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '33101', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami School of Medicine', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '10019', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'St Lukes-Roosevelt Hospital Center, New York', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '44122', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cleveland Clinic Foundation', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '19140', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Temple University Hospital', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '75708-3154', 'city': 'Tyler', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas Health Center at Tyler', 'geoPoint': {'lat': 32.35126, 'lon': -95.30106}}, {'zip': 'CB2 2XY', 'city': 'Cambridge', 'state': 'England', 'country': 'United Kingdom', 'facility': 'University of Cambridge - Cambridge Institute for Medical Research', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}, {'zip': 'EH8 9AG', 'city': 'Edinburgh', 'state': 'Scotland', 'country': 'United Kingdom', 'facility': 'University Teaching Hospital of Edinburgh', 'geoPoint': {'lat': 55.95206, 'lon': -3.19648}}], 'overallOfficials': [{'name': 'Kim Hanna, MSc', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Grifols Therapeutics LLC'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Grifols Therapeutics LLC', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}