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
Denali Therapeutics Inc.
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
- Age range
- 18–75 years
- Sex
- All
- 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…
Interventions
- DrugDNL952
Intravenous repeating dose
Location
- The Lysosomal & Rare Disorders Research & Treatment CenterFairfax, Virginia