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Duchenne Muscular DystrophyPhase 1/2NCT04906460

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

A Phase 1/2 study testing WVE-N531 in people with Duchenne Muscular Dystrophy, run by Wave Life Sciences USA, Inc. at 6 sites in 3 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). Read more about this study

Who is it for?
Ages 4 to 18 years, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
WVE-N531
Could I receive a placebo?
No, everyone receives an active treatment
How many people
25 participants
Where
6 sitesin 3 countries
Study dates
28 Sep 2021to 24 Apr 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 to 18 years
  • Men only
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Part A and Part B:
  • Part A patients may be screened for Part B upon completion of a washout period of ≥18 weeks from last dose in Part A. New patients may also be screened for Part B
  • Diagnosis of DMD based on clinical phenotype.
  • Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention
  • Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) (Part B ).
  • Ambulatory or non-ambulatory male
Show all 20Show fewer
  • Stable pulmonary and cardiac function, as measured by the following: (Part B):
  • 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 (ECHO) and/or cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study.
  • 7.Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid.
  • 8. Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit (Part B ).
  • Part C
  • New patients to be screened for Part C.
  • Diagnosis of DMD based on clinical phenotype.
  • Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention
  • Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) .
  • Ambulatory male
  • Stable pulmonary and cardiac function, as measured by the following:
  • 1. Reproducible percent predicted forced vital capacity (FVC) ≥50%; 2. Left ventricular ejection fraction (LVEF) >55% in patients as measured (and documented) by echocardiogram (ECHO) and/or cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study.
  • 7. Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid.
  • 8. Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit .

Who cannot join

  • Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the study procedures.
  • Part B and Part C: Major surgery within 3 months prior to Day 1 or planned major surgery for any time during the study.
  • Part B: Diagnosis of active alcohol, cannabinoid, or other substance use disorder (except nicotine) within 6 months prior to the Screening visit
  • Part C: Any recreational substance use (including prescribed cannabinoids), with the exception of nicotine, irrespective of legality, within 2 months prior to Screening and/or unwilling to refrain from such use for the duration of the study.

Trial locations and contacts

6 sites in 3 countries.

6 sites in 3 countries

Jordan3 sites

  • Istiklal Hospital/ Clinical Research Unit

    Amman

  • The Specialty Hospital (TSH)/ Advanced Clinical Center

    Amman

  • King Abdullah University Hospital (KAUH)

    Irbid

Other trials in Jordan

United States2 sites

  • Rare Disease Research LLC

    Atlanta, Georgia, 30329

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202-3500

Other trials in United States

United Kingdom1 site

  • Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust

    Headington, Oxford, OX3 9DU

Other trials in United Kingdom

About this study

Official title: An Open-label Phase 1b/2 Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy

This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 3 parts, Part A, Part B, including Part B Extension Arm, and Part C. Part A is completed. Part B is completed. Following completion of Part B, all patients elected to continue to receive study drug in the optional Part B open-label Extension Arm. Part C has been added to the study and will enroll new patients.

Read the detailed description

Following completion of Part A, eligible patients rolled over into Part B to continue to receive treatment. In addition, new patients were enrolled up to a total of 11 patients in Part B. All patients received WVE-N531 at 10 mg/kg every other week (Q2W) until competent authority approval of a protocol update, when all patients were switched to Q4W dosing. Muscle biopsies were performed following 24 weeks and for new Part B patients (those that did not take part in Part A) following 48 weeks of treatment. Following completion of Part B, all patients elected to continue to receive study drug at Q4W for up to 1 year in an optional Part B Extension Arm.

In part C of the study, 13 new patients have been enrolled . All patients will undergo an open muscle biopsy, at baseline and following 24 weeks of treatment.

The primary endpoint for Part B is the measurement of dystrophin protein levels. Participants will also be evaluated for safety, tolerability, digital and functional endpoints.

The primary endpoint for Part C is the measurement of dystrophin protein levels. Participants will also be evaluated for safety, tolerability, digital and functional endpoints. Safety monitoring will occur through 10 months after the last dose.

Being tested
WVE-N531
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • WVE-N531Drug

    WVE-N531 is an antisense oligonucleotide (ASO)

Study groups in NCT04906460
Study groupRoleWhat they receive
WVE-N531ExperimentalNot described
Receives: Drug: WVE-N531

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part A: Safety: Proportion of patients with adverse events (AEs)

    Measured: Day 1 (initial dose) up to 24 weeks after the last dose of Part A

  2. Part B: Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531

    Measured: At Week 26 and at Week 50 of Part B

  3. 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

    Measured: At Baseline and following 24 weeks of treatment in Part C

Secondary outcomes (8)

  1. Part A: Pharmacokinetics: Concentration of WVE-N531 in muscle tissue

    Measured: Day 1 (initial dose) through 2 weeks after the last dose of Part A

  2. Part A: Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531

    Measured: Day 1 (initial dose) through 2 weeks after the last dose of Part A

  3. 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.

    Measured: Collected at baseline, Weeks 24 and 48 of Part B, at baseline and Weeks 26 and 50 of the Extension Arm

  4. 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.

    Measured: Collected at baseline, Weeks 24 and 48 of Part B, at baseline and Weeks 26 and 50 of the Extension Arm

  5. Part B: Stride Velocity 95th Centile (SV95C)/upper limb outcome (non-ambulatory patients)

    Measured: Collected at baseline, Weeks 24 and 48 of Part B, at baseline and Weeks 26 and 50 of the Extension Arm

  6. 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.

    Measured: Collected at baseline and Week 24 of Part C

  7. 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.

    Measured: Collected at baseline and Week 24 of Part C

  8. Part C: Stride Velocity 95th Centile (SV95C)/upper limb outcome (non-ambulatory patients)

    Measured: Collected at baseline and Week 24 of Part C

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 May 2021
  2. Study start28 Sep 2021
  3. Primary completionOct 2026 (estimated)
  4. Study completion24 Apr 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
25 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04906460
Sponsor's protocol ID
WVE-N531-001
Status last verified
Oct 2026
First submitted
24 May 2021
Last updated
19 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing WVE-N531 in people with Duchenne Muscular Dystrophy, run by Wave Life Sciences USA, Inc. at 6 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 20 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in Jordan, United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part A: Safety: Proportion of patients with adverse events (AEs) (time frame: Day 1 (initial dose) up to 24 weeks after the last dose of Part A). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04906460.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026; AzTrials last refreshed its copy on 26 Oct 2026.

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