Phase 2
N=6
Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency
Alpha 1-Antitrypsin Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT02168686 ↗Enrolled (actual)
6
Serious AEs
50.0%
Results posted
Aug 2022
Primary outcome: Primary: Treatment-emergent Adverse Events Related to ADVM-043 — 0; 1; 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- ADVM-043 (Genetic)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Adverum Biotechnologies, Inc.
- Primary completion
- Aug 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Treatment-emergent Adverse Events Related to ADVM-043 |
0; 1; 2 | — |
| PRIMARY Abnormal Changes in Clinical Laboratory Parameters |
2; 1; 2; 5; 0; 0 | — |
| SECONDARY Change in Plasma Concentrations of M-specific A1AT up to 52 Weeks |
88.050; 88.050 | — |
| SECONDARY Changes in Total Plasma Concentrations of A1AT up to 52 Weeks |
1.230; 1.870; 1.145; 1.415; 1.220; 0.640 | — |
Summary
The ADVANCE study is being conducted by Adverum Biotechnologies, Inc. as an open-label, multicenter, dose-escalation study in order to assess the safety and protein expression of ADVM-043 following a single intravenous or intrapleural administration.
Eligibility Criteria
Key Inclusion Criteria
- Capable of providing informed consent
- Alpha1AT genotype of ZZ or Z Null
- Males and females 18 years and older
- Ongoing treatment with A1AT augmentation is not required, however any subject receiving A1AT augmentation therapy must be willing to washout. Washout is defined as at least 8 weeks between last augmentation therapy and pre-treatment plasma A1AT level
- Willing to remain off PAT for at least 3 months following treatment
- Body mass index 18 to 35 kg/m2
- Fertile men and women of childbearing potential must agree to use barrier contraception for 3 months after treatment
Key Exclusion Criteria
- FEV1 <35 percent of predicted value at the Screening visit
- Receiving systemic corticosteroids or other immunosuppressive medications
- Immunodeficiency disease or evidence of active infection of any type, including human immunodeficiency virus
- Abnormal liver function tests
- Organ transplant recipient or awaiting transplantation
- Participation in another current or previous gene transfer study
- AAVrh.10 neutralizing antibody titer ≥ 1:5
- Female who is pregnant or lactating
- History of alcohol or drug abuse within the past 5 years
- Any history of allergies that may prohibit study-specific investigations
- Receiving an investigational medicinal product or participating in another investigational study within 3 months prior to consent
- Cigarette smoking, or any other tobacco use, e-cigarettes or other recreational inhalant within 1 year of the Screening Visit
Data sourced from ClinicalTrials.gov (NCT02168686). 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.