NCT06713135Active, not recruitingPhase 4
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in 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
Source: ClinicalTrials.gov · Last updated 23 Apr 2026
Brief summary (registry, in English)
This study aims to assess safety and effectivness of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamorolone.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 4
- Study type
- Interventional
- Enrollment
- 80 (estimated)
- First submitted
- 16 Nov 2024
- Start date
- 10 Nov 2024
- Primary completion date
- Sept 2028
- Lead sponsor
- Santhera Pharmaceuticals
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT06713135
What you commit to
- Study type: Interventional · Phase 4
- Planned participants: 80
- From 10 Nov 2024 to the primary completion date, Sept 2028
- 18 sites in 9 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other illness
- Any medical condition, which in the opinion of the Investigator, would affect study participation, performance or interpretation of study assessments
- Severe hepatic impairment
Who the study is looking for
- 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
Sex: Male · Does not accept healthy volunteers
Sites (18)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| UZ GENT (Universitair Ziekenhuis Gent) Coordinator of this site? Confirm its status | Ghent | Belgium | Unknown status |
| UZ Leuven (Universitair Ziekenhuis Leuven) Coordinator of this site? Confirm its status | Leuven | Belgium | Unknown status |
| University Hospital Brno Coordinator of this site? Confirm its status | Brno | Czechia | Unknown status |
| Fakultni nemocnice Motol Coordinator of this site? Confirm its status | Prague | Czechia | Unknown status |
| Hospital Universitario Puerta de Hierro - Majadahonda Coordinator of this site? Confirm its status | Madrid | Spain | Unknown status |
| Hospital Universitario y Politécnico de La Fe Coordinator of this site? Confirm its status | Valencia | Spain | Unknown status |
| University Hospitals Birmingham NHS Foundation Trust Coordinator of this site? Confirm its status | Birmingham | United Kingdom | Unknown status |
| Queen Elizabeth University Hospital Coordinator of this site? Confirm its status | Glasgow | United Kingdom | Unknown status |
| Leeds Teaching Hospitals NHS Trust Coordinator of this site? Confirm its status | Leeds | United Kingdom | Unknown status |
| Alder Hey Children's Hospital Coordinator of this site? Confirm its status | Liverpool | United Kingdom | Unknown status |
| Great Ormond Street Hospital For Children NHS Foundation Trust Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| The John Walton Muscular Dystrophy Research Centre Coordinator of this site? Confirm its status | Newcastle | United Kingdom | Unknown status |
| Children's Hospital Agia Sofia Coordinator of this site? Confirm its status | Athens | Greece | Unknown status |
| Children's Health Ireland at Tallaght, Tallaght University Hospital Coordinator of this site? Confirm its status | Dublin | Ireland | Unknown status |
| Schneider Children's Medical Center Coordinator of this site? Confirm its status | Petah Tikva | Israel | Unknown status |
| Leiden University Medical Center Coordinator of this site? Confirm its status | Leiden | Netherlands | Unknown status |
| Radboud University Nijmegen Coordinator of this site? Confirm its status | Nijmegen | Netherlands | Unknown status |
| Te Wao Nui - Child Health Service, Wellington Hospital Coordinator of this site? Confirm its status | Wellington | New Zealand | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.