Condition: Duchenne Muscular Dystrophy · Sponsor: Wave Life Sciences USA, Inc.
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 3 parts, Part A, Part B, including Part B Extension Arm, and Part C. Part A is completed. Part B is completed. Following completion of Part B, all patients elected to continue to receive study drug in the optional Part B open-label Extension Arm. Part C has been added to the study and will enroll new patients.
This description comes directly from the study's public registry record.
Clinical Operations · 855-215-4687 · clinicaltrials@wavelifesci.com
Always discuss trial participation with your own doctor first.
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| The Specialty Hospital (TSH)/ Advanced Clinical Center | Amman, Jordan | Recruiting |
| Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust | Headington, Oxford, United Kingdom | Recruiting |
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Source record: clinicaltrials.gov/study/NCT04906460