Phase 1 N=9 Treatment

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency

Alpha 1-Antitrypsin Deficiency

Bottom Line

View on ClinicalTrials.gov: NCT00430768 ↗

Enrolled (actual)

Serious AEs

11.1%

Results posted

Dec 2016

Primary outcome: Primary: Adverse Events Possibly, Probably or Definitely Related to Study Drug — 2; 2; 3; 2 participants

Study Design & Population

Study type: Interventional
Phase: Phase 1
Interventions: rAAV1-CB-hAAT (Biological)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: University of Massachusetts, Worcester
Primary completion: Jan 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Adverse Events Possibly, Probably or Definitely Related to Study Drug	2; 2; 3; 2; 0; 0	—
SECONDARY hAAT Expression in Blood Measured Using M-specific Allele ELISA	33; 10.8; 11.2; 9.6; 7.1; 18.3	—

Summary

Individuals with a deficiency of the alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells with the expectation that the AAT protein may be produced at normal levels. This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency. The study will also determine what dose may be required to achieve normal levels of AAT.

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00430768). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.