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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
NCTID
NCT01054339
(View at clinicaltrials.gov)
Description
Assessment of the safety and efficacy of intramuscular (IM) administration of a recombinant adenoassociated virus (rAAV) alpha-1 antitrypsin (AAT) vector (rAAV1-CB-hAAT) in AAT-deficient adults at three dosage levels \[6.0 × 10e11, 1.9 × 10e12 and 6.0 × 10e12 vector genome particles (vg) per kg body weight\]. Funding Sources - The FDA Office of Orphan Products Development and NIH National Heart, Lung, and Blood Institute
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Development Status
Inactive
Indication
Alpha-1 Antitrypsin Deficiency (AATD)
Disease Ontology Term
DOID:13372
Compound Name
AGTC-0106
Compound Description
rAAV1-CB-hAAT
Sponsor
Beacon Therapeutics
Funder Type
Industry
Recruitment Status
Completed
Enrollment Count
9
Results Posted
View Results
Therapy Information
Target Gene/Variant
SERPINA1
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intramuscular
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV1
Editor Type
none
Dose 1
6E11 vg/kg
Dose 2
1.9E12 vg/kg
Dose 3
6E12 vg/kg
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase2
Submit Date
2010-01-21
Completion Date
2015-10
Last Update
2019-03-28
Participation Criteria
Eligible Age
18 Years - 75 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
4
Locations
United States,Ireland
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
IM route resulted in poor efficacy, product development abandoned
Resources/Links
Clinical Publications
Gene therapy for alpha-1 antitrypsin deficiency: an update
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression
News and Press Releases
SEC Form 10-Q: Q1 2015
Preclinical Publications
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study
Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy
Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans
(Abstract #101) Toxicology and Biodistribution Studies of a Recombinant Adeno-Associated Virus 2 (rAAV2) Alpha-1 Antitrypsin (AAT) Vector - ASGCT 2004
Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors
Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector
Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites
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