NCT07354724 A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
| NCT ID | NCT07354724 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Denali Therapeutics Inc. |
| Condition | Late-onset Pompe Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 32 participants |
| Start Date | 2026-05-12 |
| Primary Completion | 2028-08 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 32 participants in total. It began in 2026-05-12 with a primary completion date of 2028-08.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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).
Eligibility 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
Contact & Investigator
Medical Monitor
STUDY DIRECTOR
Denali Therapeutics Inc.
Frequently Asked Questions
Who can join the NCT07354724 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 75 Years, studying Late-onset Pompe Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07354724 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07354724 currently recruiting?
Yes, NCT07354724 is actively recruiting participants. Visit ClinicalTrials.gov or contact Denali Therapeutics Inc. to inquire about joining.
Where is the NCT07354724 trial being conducted?
This trial is being conducted at Irvine, United States, Durham, United States, Fairfax, United States.
Who is sponsoring the NCT07354724 clinical trial?
NCT07354724 is sponsored by Denali Therapeutics Inc.. The principal investigator is Medical Monitor at Denali Therapeutics Inc.. The trial plans to enroll 32 participants.