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
Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs) — 48 weeks
Incidence and severity of infusion-related reacations (IRRs) — 48 weeks
Trial sites (4)
Facility
City
Region
Status
University of California-Irvine
Irvine
California
Recruiting
Washington University School of Medicine
St Louis
Missouri
Recruiting
Duke University School of Medicine - Early Phase Research Unit
Durham
North Carolina
Recruiting
The Lysosomal & Rare Disorders Research & Treatment Center
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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