A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Recruiting now · Phase 1
Conditions studied: Late-onset Pompe Disease
In brief
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).
Key facts
- Study ID
- NCT07354724
- Run by
- Denali Therapeutics Inc.
- People needed
- 32
- Starts
- 2026-05-12
- Expected to finish
- 2028-08-01
- Last updated by the study team
- 2026-06-29
Who can join
Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California-Irvine — Irvine, California, United States (enrolling)
- Duke University School of Medicine - Early Phase Research Unit — Durham, North Carolina, United States (enrolling)
- The Lysosomal & Rare Disorders Research & Treatment Center — Fairfax, Virginia, United States (enrolling)
Full record on ClinicalTrials.gov
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