Duchenne Muscular Dystrophy
Conditions
Brief summary
This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.
Interventions
DRUGWVE-210201
WVE-210201 is a stereopure antisense oligonucleotide (ASO)
DRUGPlacebo
Sodium Chloride
Sponsors
Wave Life Sciences Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
MALE
Age
5 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of Duchenne muscular dystrophy (DMD) based on clinical phenotype with increased serum creatine kinase * Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping * Ambulatory or non-ambulatory male patients aged ≥5 - ≤18 years * Stable pulmonary and cardiac function as measured by: 1. Reproducible percent predicted forced vital capacity (FVC) ≥50% 2. Left ventricular ejection fraction (LVEF) \>55% in patients \<10 years of age and \>45% in patients ≥10 years of age, as measured (and documented) by echocardiogram within one year prior to enrollment into the study.
Exclusion criteria
* Severe cardiomyopathy; cardiomyopathy that is managed by angiotensin-converting enzyme (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criteria. * Need for mechanical or non-invasive ventilation OR anticipated need for mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator. * Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify dose or regimen during the study. * Currently on anticoagulants or antithrombotics. * Received treatment with eteplirsen or ataluren within the past 14 weeks. * Received prior treatment with drisapersen. * Received any investigational drug within the past 3 months or 5 half-lives, whichever is longer.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety: Number of patients with adverse events (AEs) | Day 1 to Day 85 (end of study) |
| Safety: Severity of AEs | Day 1 to Day 85 (end of study) |
| Safety: Number of patients with serious AEs (SAEs) | Day 1 to Day 85 (end of study) |
| Safety and Tolerability: Number of patients who withdraw due to AEs | Day 1 to Day 85 (end of study) |
Secondary
| Measure | Time frame |
|---|---|
| PK: Time of occurrence of Cmax (tmax) | Day 1, Day 2, and Day 8 |
| PK: Area under the plasma concentration-time curve (AUC 0-t) | Day 1, Day 2, and Day 8 |
| Pharmacokinetics (PK): Maximum observed concentration (Cmax) | Day 1, Day 2, and Day 8 |
Countries
Belgium, Canada, France, Italy, Netherlands, United Kingdom, United States
Outcome results
None listed