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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03508947
• Date of registration: 16/04/2018
• Prospective Registration: No
• Primary sponsor: Wave Life Sciences Ltd.
• Public title: Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
• Scientific title: A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy
• Date of first enrolment: January 24, 2018
• Target sample size: 36
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03508947
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 1

Countries of recruitment

Belgium, Canada, France, Italy, Netherlands, United Kingdom, United States

Contacts

• Name: Michael A Panzara, MD, MPH
• Affiliation: Wave Life Sciences Ltd.

Key inclusion & exclusion 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:

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.



Age minimum: 5 Years
Age maximum: 18 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Placebo

Drug: WVE-210201

Primary Outcome(s)

Safety and Tolerability: Number of patients who withdraw due to AEs [Time Frame: Day 1 to Day 85 (end of study)]

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)]

Secondary Outcome(s)

Pharmacokinetics (PK): Maximum observed concentration (Cmax) [Time Frame: Day 1, Day 2, and Day 8]

PK: Area under the plasma concentration-time curve (AUC 0-t) [Time Frame: Day 1, Day 2, and Day 8]

PK: Time of occurrence of Cmax (tmax) [Time Frame: Day 1, Day 2, and Day 8]

Secondary ID(s)

WVE-DMDX51-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors