Actively Recruiting
A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, 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
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying DNL952 in adults with late-onset Pompe disease (LOPD) to understand its safety, how the body processes it, and its effects. This Phase 1, open-label, multicenter trial focuses on evaluating safety and tolerability across different dose levels of DNL952 in participants with LOPD. The study is sponsored by Denali Therapeutics Inc. and aims to gather important information about treatment impact on this condition. The study involves giving DNL952 through repeated intravenous doses to adult participants diagnosed with LOPD. Participants are grouped into several cohorts, some of which require prior enzyme-replacement therapy with specific drugs, while others have not received such therapy recently. The study is non-randomized and open-label, meaning all participants receive the investigational drug and know the treatment they receive. Participants will be monitored for 48 weeks to assess treatment-emergent adverse events, infusion-related reactions, and pharmacokinetic parameters such as drug concentration over time. Safety and tolerability data will be collected alongside laboratory and clinical assessments. This involves tracking how the drug behaves in the body and its potential side effects. The study lasts about a year, including screening and treatment periods, with careful follow-up to understand the drug's effects in people with LOPD.
CONDITIONS
Brief 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 or more
- Diagnosis of late-onset Pompe disease (LOPD)
- Upright forced vital capacity (FVC) 30% or more of predicted normal value
- Able to walk 40 meters or more (assistive devices allowed)
- For Cohorts A1 to A4: Received avalglucosidase alfa or cipaglucosidase alfa at 20 mg/kg every 2 weeks for at least 12 months before screening
- For Cohorts B1 and B2: No enzyme-replacement therapy for Pompe disease in the 12 months before screening
You will not qualify if you...
- Any ongoing, unstable, or poorly controlled neurological, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematological, immunological, allergic, or eye disease not related to Pompe disease, unless well-controlled and agreed by investigator and sponsor
- Wheelchair-dependent
- Require noninvasive ventilation more than 6 hours per day while awake or any invasive ventilation (noninvasive ventilation during sleep is allowed)
- Received experimental gene therapy at any time
- Participation in other investigational drug trials or use of investigational drugs within 60 days or 5 half-lives before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 48 weeks
Participants receive intravenous repeating doses of DNL952 to evaluate safety and pharmacokinetics.
Regular visits for dosing and assessments during treatment
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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