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

Evaluating DNL952 for safety in adults with late-onset Pompe disease.

Recruiting
18-75 years
All
Phase 1

This study is for adults with late-onset Pompe disease, a rare genetic disorder that affects muscles. Researchers want to learn about the safety and effects of a new drug called DNL952. Pharmacokinetics (PK) is how the body processes a drug, and pharmacodynamics (PD) is how a drug affects the body. Participants will try different doses of DNL952 to see which is safest and most tolerable.

Key points to consider:

  • The study involves multiple visits to the research center over a period of time.
  • Participants must be able to walk at least 40 meters. Using walking aids is allowed.
  • There may be risks involved, like side effects, but these will be monitored closely.

Participants must weigh at least 40 kg and have a confirmed diagnosis of late-onset Pompe disease. They should not have other major health issues unless well-controlled. Some may need to have been on specific enzyme-replacement therapies for the past year. If you rely heavily on machines to help you breathe while awake or have taken an investigational drug recently, you may not be eligible.

Study details
    Late-onset Pompe Disease

NCT07354724

Denali Therapeutics Inc.

8 July 2026

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