Trials / Completed
CompletedNCT03508947
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 36 (actual)
- Sponsor
- Wave Life Sciences Ltd. · Industry
- Sex
- Male
- Age
- 5 Years – 18 Years
- Healthy volunteers
- Not accepted
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
| Type | Name | Description |
|---|---|---|
| DRUG | WVE-210201 | WVE-210201 is a stereopure antisense oligonucleotide (ASO) |
| DRUG | Placebo | Sodium Chloride |
Timeline
- Start date
- 2018-01-24
- Primary completion
- 2019-03-06
- Completion
- 2019-03-06
- First posted
- 2018-04-26
- Last updated
- 2019-04-08
Locations
13 sites across 7 countries: United States, Belgium, Canada, France, Italy, Netherlands, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03508947. Inclusion in this directory is not an endorsement.