Long-term treatment with vamorolone for boys with Duchenne Muscular Dystrophy
An Open-label Study to Collect Safety and Effectiveness Information on Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy Who Have Completed Prior Studies With Vamorolone
This study is testing if long-term treatment with vamorolone is safe and effective for boys with Duchenne Muscular Dystrophy who have already tried this medication before.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 80 (estimated) |
| Sex | Male |
| Sponsor | Santhera Pharmaceuticals Industry-sponsored |
| Locations | 12 sites (Gent and 11 other locations) |
| Trial ID | NCT06713135 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and effectiveness of long-term treatment with vamorolone in boys diagnosed with Duchenne Muscular Dystrophy (DMD) who have previously participated in studies involving vamorolone. Participants will continue their treatment under a new protocol after having been part of compassionate use programs. The primary focus is on assessing the safety concerning vertebral fractures, while secondary objectives include evaluating non-vertebral fractures, cataracts, delayed puberty, and overall safety, as well as both ambulatory and non-ambulatory functions.
Who should consider this trial
Good fit: Ideal candidates are boys with Duchenne Muscular Dystrophy who have previously completed studies with vamorolone and are currently receiving this treatment.
Not a fit: Patients who have discontinued vamorolone treatment for safety reasons or have severe hepatic impairment may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve the long-term health and quality of life for boys with Duchenne Muscular Dystrophy.
How similar studies have performed: Other studies involving vamorolone have shown promising results, indicating a potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Subject and/or subject's parent(s) or legal guardian has provided written informed consent * Subject has previously completed either the VBP15-LTE or VBP15-004 study, and transitioned through the Compassionate Use Program, Named Patient Program or Expanded Acess Protocol * Subject is on vamorolone on day of enrolment * Subject and parent / legal guardian are willing and able to comply with the protocol schedule, assessments and requirements Exclusion Criteria: * Any medical condition, which in the opinion of the Investigator, would affect study participation, performance or interpretation of study assessments * Vamorolone treatment discontinued for ≥ 6 months within the year prior to enrolment for a non-safety reason, or vamorolone treatment previously discontinued at any time for a safety reason * Severe hepatic impairment
Where this trial is running
Gent and 11 other locations
- UZ Gent (Universitair Ziekenhuis Gent) — Gent, Belgium (Not_yet_recruiting)
- UZ Leuven (Universitair Ziekenhuis Leuven) — Leuven, Belgium (Not_yet_recruiting)
- University Hospital Brno — Brno, Czechia (Not_yet_recruiting)
- Fakultni Nemocnice Motol — Prague, Czechia (Recruiting)
- Hospital Universitario Puerta de Hierro Majadahonda — Madrid, Spain (Not_yet_recruiting)
- Hospital Universitario y Politecnico de La Fe — Valencia, Spain (Not_yet_recruiting)
- Queen Elizabeth University Hospital — Glasgow, Lanarkshire, United Kingdom (Not_yet_recruiting)
- Alder Hey Children's Hospital — Liverpool, Merseyside, United Kingdom (Not_yet_recruiting)
- Leeds Teaching Hospitals NHS Trust — Leeds, West Yorkshire, United Kingdom (Not_yet_recruiting)
- University Hospitals Birmingham NHS Foundation Trust — Birmingham, United Kingdom (Not_yet_recruiting)
- Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom (Not_yet_recruiting)
- The John Walton Muscular Dystrophy Research Centre — Newcastle, United Kingdom (Not_yet_recruiting)
Study contacts
- Principal investigator: A Child, MD — Leeds Teaching Hospital
- Study coordinator: Clinical Trial Leader
- Email: caroline.waldy@santhera.com
- Phone: +41 79 476 08 26
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.