Phase 1 Study of DNL952 Safety and Effects in Adults With Late-Onset Pompe Disease

What this Trial Is About

Researchers are evaluating DNL952 in adult participants with late-onset Pompe disease LOPD through a Phase 1, open-label, multicenter study. The main goal is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of DNL952 across different dose levels in participants with LOPD. This study focuses on gathering data to understand the treatments effects in this population. Participants will receive DNL952 as an intravenous repeating dose. The study includes multiple cohorts, some of which have prior enzyme replacement therapy and others who have not received such treatment recently. The trial is non-randomized and open-label, allowing observation of safety and drug behavior over time. During the study, participants will be monitored for up to 48 weeks to assess adverse events, infusion-related reactions, and the drugs concentration and elimination in the blood. Researchers will conduct various evaluations including safety assessments and pharmacokinetic measurements. The total participation duration aligns with the 48-week primary outcome measurement period.

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease