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New treatment option for Duchenne Muscular Dystrophy
Official title Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
ClinicalTrials.gov ID: NCT04906460
What this study is testing
What is WVE-N531?
WVE-N531 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 1 year
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 18, men only
You may be able to join if
- 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...
- 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 ).
You likely can't join if
- Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient...
- 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...
See the full eligibility criteria
- 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
- 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 \ 45% in...
- 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...
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- Atlanta, Georgia, United States
- Amman, Jordan
- Headington, Oxford, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 1 year per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling male, 4 years to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; Atlanta, Georgia, United States; Amman, Jordan; Headington, Oxford, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.