Phase 1 Pompe Disease Clinical Trials

4 recruitingPhase 1

What is a Phase 1 trial?

Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.

Showing 1–4 of 4 trials

Recruiting

Phase 1Phase 2

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)

Pompe Disease (Late-onset)LOPD

AskBio Inc12 enrolled9 locationsNCT07282847

Recruiting

Phase 1

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Late-onset Pompe Disease

Denali Therapeutics Inc.32 enrolled1 locationNCT07354724

Recruiting

Phase 1

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Pompe Disease (Infantile-Onset)MPS VIIMPS I+6 more

University of California, San Francisco10 enrolled1 locationNCT04532047

Recruiting

Phase 1Phase 2

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

Pompe Disease (Late-onset)

GeneCradle Inc33 enrolled1 locationNCT06391736