Actively Recruiting
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Led by Denali Therapeutics Inc. · Updated on 2026-05-12
32
Participants Needed
1
Research Sites
117 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Body weight ≥40 kg
- Diagnosis of late-onset Pompe disease
- Upright forced vital capacity (FVC) ≥30% of predicted normal value
- Able to walk at least 40 meters (assistive devices allowed)
- For Cohorts A1-A4: Received avalglucosidase alfa or cipaglucosidase alfa 20 mg/kg every 2 weeks for at least 12 months before screening
- For Cohorts B1-B2: No enzyme-replacement therapy for Pompe disease in the 12 months before screening
You will not qualify if you...
- Any ongoing, clinically significant, unstable, or poorly controlled neurological, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematological, immunological, allergic, or ophthalmic disease unrelated to Pompe disease (well-controlled conditions allowed with agreement)
- Wheelchair-dependent
- Require noninvasive ventilation more than 6 hours per day while awake or any invasive ventilation (noninvasive ventilation during sleep allowed)
- Received experimental gene therapy at any time
- Participation in another investigational drug trial or use of investigational drug within 60 days or 5 half-lives before screening
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
The Lysosomal & Rare Disorders Research & Treatment Center
Fairfax, Virginia, United States, 22030
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
6
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