A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Purpose
The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.
Conditions
- Pompe Disease
- Pompe Disease (Late-onset)
- Glycogen Storage Disease Type 2
- Glycogen Storage Disease Type II
- LOPD
- Lysosomal Storage Diseases
- Acid Maltase Deficiency
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Provide written informed consent; - Males and Females ≥18 years of age with late-onset Pompe disease; - Received ERT for at least the previous 24 months - Have clinically moderate, late-onset Pompe disease characteristics; - Agree to use reliable contraception.
Exclusion Criteria
- Active hepatitis B and/or C; - Significant underlying liver disease; - Human immunodeficiency virus (HIV) infection; - Prior hypersensitivity to rhGAA; - Pre-existing anti-AAV neutralizing antibody titers; - High titer antibody responses to rhGAA; - Requires any invasive ventilation or requires noninvasive ventilation while awake and upright; - Received any prior vector or gene transfer agent; - Active malignancy (except non-melanoma skin cancer); - History of liver cancer; - Pregnant or nursing women; - Any evidence of active infection at the time of SPK-3006 infusion.
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental SPK-3006 |
All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006. |
|
More Details
- Status
- Active, not recruiting
- Sponsor
- Spark Therapeutics, Inc.