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Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

W

Wave Life Sciences

Status and phase

Completed
Phase 1

Conditions

Duchenne Muscular Dystrophy

Treatments

Drug: Placebo
Drug: WVE-210201

Study type

Interventional

Funder types

Industry

Identifiers

NCT03508947
WVE-DMDX51-001

Details and patient eligibility

About

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.

Enrollment

36 patients

Sex

Male

Ages

5 to 18 years old

Volunteers

No Healthy Volunteers

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.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Sequential Assignment

Masking

Double Blind

36 participants in 5 patient groups

WVE-210201 (Dose A) or placebo
Experimental group
Treatment:
Drug: Placebo
Drug: WVE-210201
WVE-210201 (Dose B) or placebo
Experimental group
Treatment:
Drug: Placebo
Drug: WVE-210201
WVE-210201 (Dose C) or placebo
Experimental group
Treatment:
Drug: Placebo
Drug: WVE-210201
WVE-210201 (Dose D) or placebo
Experimental group
Treatment:
Drug: Placebo
Drug: WVE-210201
WVE-210201 (Dose E) or placebo
Experimental group
Treatment:
Drug: Placebo
Drug: WVE-210201

Trial contacts and locations

13

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Data sourced from clinicaltrials.gov

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