Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
- Registration Number
- NCT03508947
- Lead Sponsor
- Wave Life Sciences Ltd.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 36
-
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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description WVE-210201 (Dose B) or placebo Placebo - WVE-210201 (Dose C) or placebo Placebo - WVE-210201 (Dose A) or placebo Placebo - WVE-210201 (Dose A) or placebo WVE-210201 - WVE-210201 (Dose D) or placebo WVE-210201 - WVE-210201 (Dose D) or placebo Placebo - WVE-210201 (Dose E) or placebo Placebo - WVE-210201 (Dose E) or placebo WVE-210201 - WVE-210201 (Dose B) or placebo WVE-210201 - WVE-210201 (Dose C) or placebo WVE-210201 -
- Primary Outcome Measures
Name Time Method 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: Number of patients with adverse events (AEs) 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 Outcome Measures
Name Time Method 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
Trial Locations
- Locations (13)
Universitaire Ziekenhuizen Leuven
🇧🇪Leuven, Belgium
CHR de la Citadelle
🇧🇪Liège, Belgium
Rare Disease Research, LLC.
🇺🇸Atlanta, Georgia, United States
UZ Gent
🇧🇪Gent, Belgium
Radbound University Nijmegen Medical Care
🇳🇱Nijmegen, Netherlands
U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud
🇮🇹Messina, Italy
University Hospitals Bristol NHS Foundation Trust
🇬🇧Bristol, United Kingdom
Evelina London Children's Hospital
🇬🇧London, United Kingdom
UCL Institute of Child Health & Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom
Hôpital Armand Trousseau
🇫🇷Paris, France
London Health Sciences Centre - Hospital
🇨🇦London, Ontario, Canada
U.O. Immunologia Pediatrica
🇮🇹Milano, Italy
Alder Hey Children's Hospital
🇬🇧Liverpool, United Kingdom