CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 9 enrolled
Drug / intervention
rAAV1-CB-hAATdrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01054339
NCT01054339Phase 2Completed
CompareCT.gov

A Multiple-Site, Phase 2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Alpha-1 Antitrypsin (rAAV1-CB-hAAT) in Patients With Alpha-1 Antitrypsin Deficiency

Beacon Therapeutics·interventional·Posted Jan 22, 2010·Updated Mar 28, 2019

In Brief

A Phase 2 clinical trial evaluating rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency. Completed, enrolled 9 participants across 4 sites in 2 countries.

Detailed Summary

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

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsAlpha-1 Antitrypsin Deficiency
CountriesIreland, United States
CollaboratorsNational Heart, Lung, and Blood Institute (NHLBI)

Timeline

Phase 2CompletedFinished
20102011201220132014201520162017201820192020202120222023202420252026
First PostedJan 22, 2010
Enrollment StartJun 1, 2010
Primary CompletionOct 1, 2011
Study CompletionOct 1, 2015
TodayJul 2, 2026
Enrollment to primary: 1.3 yearsPosted 16.4 years ago

Interventions

rAAV1-CB-hAATdrug

Recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin