A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
Recruiting
Phase 1
Phase 2
Interventional Study
Pompe Disease (Late-onset)
Pompe Disease Late-Onset
LOPD
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 – 65
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 12 (estimated)
- Sponsor
- AskBio Inc · Industry
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About This Trial
This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. Participant must be ≥18 to ≤65 years of age at the time of signing the informed consent form.
2. Confirmed GAA enzyme deficiency from any tissue source and/or confirmed biallelic GAA gene mutations.
3. Undergone enzyme replacement treatment (ERT) (either alglucosidase alfa (L…
Contacts