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

NCT03508947 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2019-04-08

No results posted yet for this study

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.

Conditions

Interventions

DRUG

WVE-210201

WVE-210201 is a stereopure antisense oligonucleotide (ASO)

DRUG

Placebo

Sodium Chloride

Sponsors & Collaborators

  • Wave Life Sciences Ltd.

    lead INDUSTRY

Principal Investigators

  • Michael A Panzara, MD, MPH · Wave Life Sciences Ltd.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
SEQUENTIAL

Eligibility

Min Age
5 Years
Max Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-01-24
Primary Completion
2019-03-06
Completion
2019-03-06
FDA Drug
Yes

Countries

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

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03508947 on ClinicalTrials.gov