Condition: Late-onset Pompe Disease · Sponsor: Denali Therapeutics Inc.
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).
This description comes directly from the study's public registry record.
Contact details are listed per location below or on the registry record.
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| University of California-Irvine | Irvine, California, 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 |
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Source record: clinicaltrials.gov/study/NCT07354724