Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Spark Therapeutics, Inc.
- Enrollment
- 4
- Locations
- 29
- Primary Endpoint
- Occurrence of immune response against GAA transgene
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- 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.
Arms & Interventions
SPK-3006
All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
Intervention: SPK-3006 (Genetic)
Outcomes
Primary Outcomes
Occurrence of immune response against GAA transgene
Time Frame: Up to 5 years
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
Time Frame: Up to 5 years
Adverse events.
Occurrence of immune response against AAV capsid
Time Frame: Up to 5 years
Secondary Outcomes
No secondary outcomes reported