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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03508947
• Other study ID #: WVE-DMDX51-001
• Status: Completed
• Phase: Phase 1
• Start date: January 24, 2018
• Est. completion date: March 6, 2019

Study information

• Verified date: April 2019
• Source: Wave Life Sciences Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 36
• Est. completion date: March 6, 2019
• Est. primary completion date: March 6, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• WVE-210201
WVE-210201 is a stereopure antisense oligonucleotide (ASO)
• Placebo
Sodium Chloride

Locations

Country	Name	City	State
Belgium	UZ Gent	Gent
Belgium	Universitaire Ziekenhuizen Leuven	Leuven
Belgium	CHR de la Citadelle	Liège
Canada	London Health Sciences Centre - Hospital	London	Ontario
France	Hôpital Armand Trousseau	Paris
Italy	U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud	Messina
Italy	U.O. Immunologia Pediatrica	Milano
Netherlands	Radbound University Nijmegen Medical Care	Nijmegen
United Kingdom	University Hospitals Bristol NHS Foundation Trust	Bristol
United Kingdom	Alder Hey Children's Hospital	Liverpool
United Kingdom	Evelina London Children's Hospital	London
United Kingdom	UCL Institute of Child Health & Great Ormond Street Hospital for Children	London
United States	Rare Disease Research, LLC.	Atlanta	Georgia

Sponsors (1)

Lead Sponsor	Collaborator
Wave Life Sciences Ltd.

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety: Number of patients with adverse events (AEs)		Day 1 to Day 85 (end of study)
Primary	Safety: Severity of AEs		Day 1 to Day 85 (end of study)
Primary	Safety: Number of patients with serious AEs (SAEs)		Day 1 to Day 85 (end of study)
Primary	Safety and Tolerability: Number of patients who withdraw due to AEs		Day 1 to Day 85 (end of study)
Secondary	Pharmacokinetics (PK): Maximum observed concentration (Cmax)		Day 1, Day 2, and Day 8
Secondary	PK: Time of occurrence of Cmax (tmax)		Day 1, Day 2, and Day 8
Secondary	PK: Area under the plasma concentration-time curve (AUC 0-t)		Day 1, Day 2, and Day 8