Health Trials

Late-onset Pompe Disease

1

Review clinical trials related to Late-onset Pompe Disease. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult participants with late-onset Pompe disease. The principal aim of this study is to obtain safety and tolerability data across varous dose levels of DNL952 in participants with late-onset Pompe disease (LOPD).

Participants needed: 32
Trial details
Phase: Phase 1Age: 18-75Biological sex: AllType: InterventionalSponsor: Denali Therapeutics Inc.Updated: Jun 29, 2026Locations: 3
Eligibility criteria

Body weight ≥40 kg [+5]

Any ongoing, clinically significant, unstable, or poorly controlled neurological... [+3]

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