A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular Dystrophy

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 32

Inclusion Criteria

1. Patient and/or parent or legal guardian must have the ability and be willing to provide written informed consent prior to any study-related procedures.
2. Diagnosis of DMD based on clinical phenotype with increased serum creatine kinase.
3. Documented mutation in the dystrophin gene associated with DMD that is amenable to exon 51 skipping.
4. Ambulatory or non-ambulatory male.
5. Age of =5 and =18 years at randomization
tests, study restrictions, and all study procedures.
7. Stable pulmonary and cardiac function, documented within the past year, as measured by:
a) Reproducible percent predicted forced vital capacity (FVC) =50%
b) 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.
8. Sexually mature males must be willing to use contraception for the duration of the study, if the patient is sexually active.

Are the trial subjects under 18? yes
Number of subjects for this age range: 32
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 28 days of Screening visit;
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 (ALT (alanine aminotransferase) and AST (aspartate aminotransferase) elevations inconsistent with age and creatine kinase [CK] level, and elevated direct or indirect bilirubin);
c) aPTT values above the upper limit of normal (ULN);
d) Platelet count 4. Severe mental retardation and/or behavioral problems that, in the opinion of the Investigator, could prohibit participation in this study.
5. Severe cardiomyopathy that, in the opinion of the Investigator, prohibits participation in this study. Cardiomyopathy that is managed by angiotensin-converting enzyme (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criteria.
6. 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.
7. 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.
8. Currently on anticoagulants or antithrombotics.
9 Received prior treatment with drisapersen.
10. Received treatment with eteplirsen or ataluren within the past 14 weeks.
11. Received any investigational drug within the past 3 months or 5 half-lives, whichever is longer.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Related Trials