An Open-label Phase 1b/2 Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Wave Life Sciences Ltd.
Summary
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.
Description
Following completion of Part A, eligible patients rolled over into Part B to continue to receive treatment. In addition, new patients were enrolled up to a total of 11 patients in Part B. All patients received WVE-N531 at 10 mg/kg every other week (Q2W) until competent authority approval of a protocol update, when all patients were switched to Q4W dosing. Muscle biopsies were performed following 24 weeks and for new Part B patients (those that did not take part in Part A) following 48 weeks of treatment. Following completion of Part B, all patients elected to continue to receive study drug at…
Eligibility
- Age range
- 4–18 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: Part A and Part B: 1. Part A patients may be screened for Part B upon completion of a washout period of ≥18 weeks from last dose in Part A. New patients may also be screened for Part B 2. Diagnosis of DMD based on clinical phenotype. 3. Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention 4. Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) (Part B ). 5. Ambulatory or non-ambulatory male 6. Stable pulmonary and cardiac function, as measured by the following: (Part B): 1\. Reproducib…
Interventions
- DrugWVE-N531
WVE-N531 is an antisense oligonucleotide (ASO)
Locations (5)
- Arkansas Children's HospitalLittle Rock, Arkansas
- Rare Disease Research LLCAtlanta, Georgia
- Istiklal Hospital/ Clinical Research UnitAmman
- The Specialty Hospital (TSH)/ Advanced Clinical CenterAmman
- Oxford Children's Hospital, Oxford University Hospitals NHS Foundation TrustHeadington, Oxford