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 - rAAV1-CB-hAAT in Patients with Alpha 1 Antitrypsin Deficiency

Phase 1

• Conditions: Alpha 1 antitrypsin deficiency.This is an inherited, genetic condition characterized by reduced serum levels of alpha-1 antitrypsin (AAT) which increases the risk of developing emphysema and liver disease; MedDRA version: 12.1Level: PTClassification code 10001806Term: Alpha-1 anti-trypsin deficiency

• Registration Number: EUCTR2009-014286-20-IE
• Lead Sponsor: Applied Genetic Technologies Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-05-31
• Last Update Posted: 31 October 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

Have a diagnosis of AAT-deficiency, as defined by a serum AAT level of less than 11 µM and a phenotype or genotype either homozygous for PI*Z or compound heterozygous consisting of PI*Z and another allele known to be associated with disease,
Be at least 18 and not more than 75 years of age,
Have a FEV1 >25% of predicted value (post bronchodilator),
Weigh = 90 kg,
Not receiving AAT augmentation therapy currently or with the past 3 months, and not planning to begin such therapy for at least 12 months after administration of rAAV1 CB hAAT,
Be willing to discontinue aspirin, aspirin-containing products, and other drugs that may alter platelet function, 7 days prior to dosing, resuming no earlier than 24 hours after the dose has been administered,
Have acceptable laboratory parameters as defined in the protocol
For females of childbearing potential:
A negative pregnancy test (urine or serum) at screening and at baseline (within 2 days before administration of study agent),
Agreement to consistently use barrier contraception (condoms, diaphragm or cervical cap with spermicide) or another form of contraception (e.g. intrauterine device or hormonal contraception) from the screening visit until 12 months after administration of rAAV1 CB hAAT, for sexual activity that could lead to pregnancy.
For males of reproductive potential, agreement to consistently use barrier contraception (condoms with spermicide) for 12 months after administration of rAAV1 CB hAAT, for sexual activity that could lead to pregnancy,
Provide signed informed consent before screening.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Prior receipt of any AAV gene therapy product,
Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration,
History of immune response to human AAT augmentation therapy as indicated by clinical history of an adverse immune response to infusion and/or decreased therapeutic effect in combination with documentation of serum anti-AAT antibodies,
Use of acute oral or intravenous antibiotic therapy for a respiratory infection within 28 days prior to study agent administration (long-term maintenance or chronic suppressive oral antibiotics, and antibiotics for a non-respiratory indication, are allowed),
Use of oral or systemic corticosteroids within 28 days prior to study agent administration,
Use of any investigational agent, or any immunosuppressive drug(s), within 3 months prior to enrollment,
For females of childbearing potential, a positive pregnancy test at screening or baseline (within 2 days before rAAV1 CB hAAT administration),
Females who are breast feeding,
Have a significant abnormal EKG finding at screening and/or cardiac disease (e.g. recent myocardial infarction or CHF) within past 6 months,
Have had pulmonary edema or a pulmonary embolism within the past 6 months,
Have a history of immunodeficiency or other medical condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified