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NCT04906460

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

StatusActive, Not Recruiting
PhasePhase 1/2
Primary completion (estimated)

At risk: Primary completion moved at least 3 months later.

See at-risk trials from Wave Life Sciences USA, Inc.

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Record History

All 11 versions: 11 with changes below. See every version on ClinicalTrials.gov ↗

  1. Enrollment closed, study ongoing Show 6 more changesShow less
    • Primary completion pushed from Jun 27, 2026 to Oct 2026
    • Enrollment target: estimated 26 to confirmed 25
    • Study sites: 5 to 6
    • Eligibility criteria expanded, about 11 words
    • Secondary endpoints: details revised at 4 of 8 entries
    • Study description shortened, about 7 words
  2. Study sites: 3 to 5v10
  3. Trial status: Active, Not Recruiting to Recruiting
    • Site details revised at 3 of 3 sites
    v9
  4. Enrollment target: 11 to 26v8 Show 6 more changesShow less
    • Primary completion date: Jan 16, 2025 to Jun 27, 2026
    • Primary endpoints: 1 to 3
    • Study completion date: Oct 2026 to Apr 24, 2027
    • Eligibility criteria expanded, about 195 words
    • Secondary endpoints: 3 to 8
    • Study description expanded, about 67 words
  5. Primary completion date changed from estimate of Jan 2025 to Jan 16, 2025v7 Show 4 more changesShow less
    • Study completion date: May 2025 to Oct 2026
    • Eligibility criteria expanded, about 17 words
    • Secondary endpoints: details revised at 3 of 3 entries
    • Study description expanded, about 77 words
Show 5 earlier versionsHide 5 earlier versions
  1. Primary completion date: Dec 2022 to Jan 2025v6 Show 9 more changesShow less
    • Primary endpoint revised
    • Trial status: Recruiting to Active, Not Recruiting
    • Enrollment target: estimated 15 to confirmed 11
    • Study sites: 2 to 3
    • Study completion date: Dec 2022 to May 2025
    • Eligibility criteria shortened, about 29 words
    • Secondary endpoints: 2 to 3
    • Study description shortened, about 22 words
    • Study title expanded, about 1 word
  2. Primary and study completion dates: Sep 2022 to Dec 2022v5
  3. Eligibility criteria shortened, about 19 words
    • Site contacts updated at 2 of 2 sites
    • Study description shortened, about 2 words
    v4
  4. Trial status: Not Yet Recruiting to Recruiting
    • Start date changed from estimate of Jun 2021 to Sep 28, 2021
    • Study sites: 0 to 2
    v3
  5. Study title shortened, about 2 wordsv2
  1. Study First Submitted
  • Notable: the plan changed
  • Minor: routine updates

Endpoints

Primary

Part A: Safety: Proportion of patients with adverse events (AEs)
Timeframe: Day 1 (initial dose) up to 24 weeks after the last dose of Part A
Part B: Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531
Timeframe: At Week 26 and at Week 50 of Part B
Part C: Pharmacodynamics: Change from baseline dystrophin level (% normal dystrophin) as assessed by a validated assay analysis in muscle tissue following multiple doses of WVE-N531
Timeframe: At Baseline and following 24 weeks of treatment in Part C

Secondary (8)

Part A: Pharmacokinetics: Concentration of WVE-N531 in muscle tissue
Part A: Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531
Part B: North Star Ambulatory Assessment (NSAA) (Version 2.0), including time to stand and a timed 10-meter walk/run, with a range of 0 to 34 where higher scores indicate better outcome.
Part B: Performance of the Upper Limb (PUL) (Version 2.0) with a range of 0 to 64 where higher scores indicate a better outcome.
Part B: Stride Velocity 95th Centile (SV95C)/upper limb outcome (non-ambulatory patients)
Show 3 moreShow less
Part C: North Star Ambulatory Assessment (NSAA) (Version 2.0), including time to stand and a timed 10-meter walk/run, with a range of 0 to 34 where higher scores indicate better outcome.
Part C: Performance of the Upper Limb (PUL) (Version 2.0) with a range of 0 to 64 where higher scores indicate a better outcome.
Part C: Stride Velocity 95th Centile (SV95C)/upper limb outcome (non-ambulatory patients)

Arms and interventions

WVE-N531Experimental

Drugs: WVE-N531

Sites

6 sites in 3 countries

Jordan 3, United States 2, United Kingdom 1