DNL952 treatment for adults with late-onset Pompe disease
Official title: 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
Run by Denali Therapeutics Inc. · NCT07354724 · Data updated 2026-11-12
This Phase 1 trial will test DNL952 in adults with late-onset Pompe disease to see if it is safe and how the drug is handled by the body.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Enrollment | 32 (estimated) |
| Sponsor | Denali Therapeutics Inc. Industry-sponsored |
| Locations | 4 sites (Irvine, California and 3 other locations) |
| Status | Recruiting |
| Started | May 12, 2026 |
| Main results expected | August 2028 |
| Study ends | August 2028 |
| Registry record updated | August 17, 2026 |
| Trial ID | NCT07354724 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults with confirmed late-onset Pompe disease who weigh ≥40 kg, can walk at least 40 meters, have upright FVC ≥30% predicted, and meet the cohort-specific enzyme-replacement therapy history are the intended participants.
✕Probably not a fit
Patients who are wheelchair-dependent, require noninvasive ventilation for more than 6 hours per day while awake, have unstable major medical conditions unrelated to Pompe disease, or do not meet cohort-specific prior ERT criteria are unlikely to benefit from participation in this early-phase safety study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is an open-label, multicenter Phase 1 dose-ranging study enrolling adults with late-onset Pompe disease to collect safety, tolerability, pharmacokinetic, and pharmacodynamic data for DNL952. Participants are grouped into cohorts with differing prior enzyme-replacement therapy histories and will receive DNL952 at escalating dose levels. Key entry requirements include a confirmed LOPD diagnosis, minimum body weight, upright FVC ≥30% predicted, and the ability to ambulate at least 40 meters. Major exclusions include wheelchair dependence, need for noninvasive ventilation more than 6 hours per day while awake, or other unstable major medical conditions not related to Pompe disease.
Why it matters
Potential benefit: If DNL952 is safe and shows favorable PK/PD, it could advance into later testing and potentially become a new option to help preserve breathing and muscle function in adults with late-onset Pompe disease.
How similar studies have gone: Approved enzyme-replacement therapies such as avalglucosidase alfa and cipaglucosidase alfa have helped many patients with Pompe disease, but DNL952 is an investigational agent in early human testing with limited prior clinical data.
Who can join: the full rules
Show full inclusion / exclusion criteria
Key Inclusion Criteria: * Body weight ≥40 kg * Diagnosis of LOPD * Upright FVC ≥ 30% of predicted normal value * Able to ambulate ≥ 40 meters (use of assistive devices is acceptable) * \[Cohorts A1-A4 only\] Have received avalglucosidase alfa or cipaglucosidase alfa at a dose of 20 mg/kg every 2 weeks for at least 12 months prior to screening * \[Cohorts B1-B2 only\] Must not have received any enzyme-replacement therapy for Pompe disease in the 12 months prior to screening Key Exclusion Criteria: * Any ongoing, clinically significant, unstable, or poorly controlled neurological, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematological, immunological, allergic, or ophthalmic disease not related to Pompe disease, or other major disorders. Well-controlled conditions are permitted if investigator and Sponsor agree. * Wheelchair-dependent * Require noninvasive ventilation for an average of more than 6 hours per day while awake or any invasive ventilation. Use of noninvasive ventilation during sleep is acceptable. * Received an experimental gene therapy at any time or participation in any other investigational drug trial or use of investigational drug within 60 days or 5 half-lives, whichever is longer, before screening
Where it's running
-
University of California-Irvine
Irvine, California, United States Recruiting -
Washington University School of Medicine
St Louis, Missouri, United States Recruiting -
Duke University School of Medicine - Early Phase Research Unit
Durham, North Carolina, United States Recruiting -
The Lysosomal & Rare Disorders Research & Treatment Center
Fairfax, Virginia, United States Recruiting
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
AB-1009 gene therapy for adults with late-onset Pompe disease
This study tests a one-time IV gene therapy (AB-1009) to try to restore GAA enzyme activity and improve muscle and breathing function in adults with late-onset Pompe disease who are already on enzyme replacement therapy.
-
Gene therapy for late-onset Pompe disease
This study is testing a new gene therapy called GC301 to see if it can improve breathing and overall health in kids and adults with late-onset Pompe disease.
-
Tracking pregnancy outcomes in women with Pompe disease
This study looks at the pregnancy experiences and outcomes for women with Pompe disease to see how it affects both them and their babies.
-
Using RNA analysis to improve diagnosis in patients with rare muscle diseases
This study is testing if analyzing RNA from muscle samples can help doctors better diagnose patients with rare muscle diseases who have previously received negative DNA test results.
-
Registry for patients with lysosomal storage diseases
This study is collecting information from patients with lysosomal storage diseases to see how these conditions progress and how treatments can improve care for them and their families.
-
In utero enzyme replacement therapy for lysosomal storage disorders
This study is testing whether giving enzyme replacement therapy to unborn babies with lysosomal storage disorders can improve their health and development before they are born.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.