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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT00430768 |
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Date of registration:
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31/01/2007 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency
AAT |
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Scientific title:
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Preclinical & Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV1-CB-hAAT) Gene Vector to AAT-Deficient Adults |
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Date of first enrolment:
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February 2006 |
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Target sample size:
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9 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00430768 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Terence R. Flotte, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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UMass Medical School |
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Name:
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Mark L Brantly, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Florida |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Diagnosed with AAT deficiency
- Forced expiratory volume in one second (FEV1) greater than 24% of predicted value
(post bronchodilator)
- Willing to discontinue AAT protein replacement 4 weeks (Group 1) and 8 weeks (Groups 2
and 3) prior to study entry, and to resume 11 weeks after rAAV1-CB-hAAT has been
administered
- Willing to discontinue aspirin, aspirin-containing products, and other drugs that may
alter platelet function 7 days prior to study entry, and to resume 24 hours after
rAAV1-CB-hAAT has been administered
- Willing to use contraception throughout the study
Exclusion Criteria:
- Required antibiotic therapy for a respiratory infection in the 28 days prior to
rAAV1-CB-hAAT administration
- Required oral or systemic corticosteroids in the 28 days prior to rAAV1-CB-hAAT
administration
- Liver disease
- Currently receiving or has received an investigational study agent in the 30 days
prior to study entry
- Received gene transfer agents in the 6 months prior to study entry
- Currently smokes cigarettes or uses illegal drugs
- History of immune response to human AAT replacement
- History of platelet dysfunction
- Abnormal ECG, heart disease, pulmonary edema, or embolism in the 6 months prior to
study entry
- Current or recent facial or chest trauma that makes it medically impossible to perform
pulmonary function tests (PFTs)
- Any other medical condition that the investigator deems unsuitable for study
participation
- Pregnant or breastfeeding
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Alpha 1-Antitrypsin Deficiency
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Intervention(s)
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Biological: rAAV1-CB-hAAT
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Primary Outcome(s)
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Adverse Events Possibly, Probably or Definitely Related to Study Drug
[Time Frame: During 1 year after study agent administration]
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Secondary Outcome(s)
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hAAT Expression in Blood Measured Using M-specific Allele ELISA
[Time Frame: Baseline, Days 14, 30, 45, 60, 90, (180, 270, and 365 if not on protein replacement therapy)]
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Secondary ID(s)
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AGTC-AAV1-001
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438
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5R01HL069877
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UF IBC RD 2630
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NCRR-supported GCRC# 611
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RR00032, RR00082
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WIRB # 20052374
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BB-IND 12728
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NIH-OBA 0404-638
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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