NCT03508947
Completed
Phase 1
A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy
Overview
- Phase
- Phase 1
- Intervention
- Placebo
- Conditions
- Duchenne Muscular Dystrophy
- Sponsor
- Wave Life Sciences Ltd.
- Enrollment
- 36
- Locations
- 13
- Primary Endpoint
- Safety: Severity of AEs
- Status
- Completed
- Last Updated
- 7 years ago
Overview
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.
Investigators
Eligibility Criteria
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:
- •Reproducible percent predicted forced vital capacity (FVC) ≥50%
- •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.
Arms & Interventions
WVE-210201 (Dose B) or placebo
Intervention: Placebo
WVE-210201 (Dose C) or placebo
Intervention: WVE-210201
WVE-210201 (Dose C) or placebo
Intervention: Placebo
WVE-210201 (Dose D) or placebo
Intervention: WVE-210201
WVE-210201 (Dose D) or placebo
Intervention: Placebo
WVE-210201 (Dose E) or placebo
Intervention: WVE-210201
WVE-210201 (Dose E) or placebo
Intervention: Placebo
WVE-210201 (Dose A) or placebo
Intervention: WVE-210201
WVE-210201 (Dose A) or placebo
Intervention: Placebo
WVE-210201 (Dose B) or placebo
Intervention: WVE-210201
Outcomes
Primary Outcomes
Safety: Severity of AEs
Time Frame: Day 1 to Day 85 (end of study)
Safety: Number of patients with serious AEs (SAEs)
Time Frame: Day 1 to Day 85 (end of study)
Safety: Number of patients with adverse events (AEs)
Time Frame: Day 1 to Day 85 (end of study)
Safety and Tolerability: Number of patients who withdraw due to AEs
Time Frame: Day 1 to Day 85 (end of study)
Secondary Outcomes
- 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)
- PK: Time of occurrence of Cmax (tmax)(Day 1, Day 2, and Day 8)
Study Sites (13)
Loading locations...
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