DMD Duchenne Muscular Dystrophy
Conditions
Interventions
WVE-210201 will be provided as a solution for dilution for infusion. In this
OLE study, all patients will receive
weekly IV doses of WVE-210201 for 13 weeks at one of the following four dose
levels:
Sponsors
Wave Life Sciences Ltd.
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Patient and/or parent or legal guardian must have the ability and be willing to provide written informed consent/minor assent prior to any study-related procedures. 2. Patient successfully completed the Phase 1 study with WVE-210201, WVE-DMDX51-001. 3. Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, study restrictions, and all study procedures. 4. Stable pulmonary and cardiac function, as measured by: a. Reproducible percent predicted forced vital capacity (FVC) >=50% b. Left ventricular ejection fraction (LVEF) >55% in patients 45% in patients >=10 years of age, as measured (and documented) by echocardiogram. 5. Sexually mature males must be willing to use contraception for the duration of the study, if the patient is sexually active. 6. Patient and caregivers must agree not to post any study-related information on social media
Exclusion criteria
Exclusion criteria: 1. Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator will make the patient unsuitable for participation in, and/or unable to complete the study procedures. 2. Other prior or ongoing medical conditions including: a. Acute illness within Screening period; b. Abnormal physical findings, other than those associated with musculoskeletal findings attributable to DMD. 3. Laboratory abnormality, that, in the Investigator's opinion, could adversely affect the safety of the patient, make it unlikely that the course of treatment or follow up would be completed, or impair the assessment of study results. These include, but are not limited to: a. Renal insufficiency; b. Impaired hepatic function as measured by glutamate dehydrogenase (GLDH) >= 2.5x upper limit of normal (ULN) and Bilirubin >= 2x ULN (or INR >= 1.5x ULN; c. Activated partial thromboplastin time [aPTT] values above the ULN; d. Platelet count less than lower limit of normal (LLN). e. Any evidence of clinically significant structural or functional heart abnormality would prohibit participation in this study. f. Troponin I value above 2x ULN 4. Parent or legal guardian is directly or indirectly involved in the conduct and administration of this study as an Investigator, subinvestigator, study coordinator, or other study staff member, or the patient is a first-degree family member, significant other, or relative residing with one of the above persons involved directly or indirectly in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety: To assess the safety and tolerability of WVE-210201, the following will be monitored and collected: 1. Adverse events (AEs) 2. Physical examination findings, including vital signs 3. Safety laboratory tests (hematology, chemistry, coagulation, urinalysis, complement etc.) 4. Electrocardiograms 5. Echocardiograms WVE-210201 concentration in plasma and urine: 1. Blood samples will be collected for determining plasma concentration of WVE-210201. 2. Urine samples will be collected for determining concentration of WVE-210201 Immunogenicity: 1. Blood samples will be collected for assessing anti-drug and anti-dystrophin antibodies in plasma Pharmacodynamic Assessments: 1. Dystrophin protein levels and exon skipping will be assessed using muscle biopsy samples Measurements of Motor Function 1. Performance of the Upper Limb (PUL, Version 2.0) 2. Respiratory function tests (peak flow rate [PFR], cough peak flow [CPF], and forced vital capacity [FVC]) 3. Upper limb proximal strength assessed by handheld myometer 4. North Star ambulatory assessments 5. Lower limb motor function by timed function tests (including 10-meter walk/run time, sit to stand from chair, 4-stair climb time, and time to rise from the floor) | — |
Secondary
| Measure | Time frame |
|---|---|
| - | — |
Countries
Netherlands
Outcome results
None listed