A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy

NCT06713135 · tracked via the Priya Life Science UK tracker

Sponsor

Santhera Pharmaceuticals

Phase

Phase 4

Started

2024-11-10

Last updated

2026-04-23

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

vamorolone 40 mg/mL oral suspension

vamorolone 40 mg/mL oral suspension: Vamorolone is administered at a dose range between 2 mg/kg/day and 6 mg/kg/day for boys weighing \<40 kg. For boys weighing 40 kg or above, the dose range will be 80 mg to 240 mg once daily. Doses can be adjusted within the dose range as determined by the Investigator based on tolerability. The highest tolerated dose should be used.

Study summary

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.

Eligibility

Sex

MALE

Min age

—

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

Number of vertebral fractures per 1000 person-years based on X-ray central reading. — At Enrolment and every 2 years during a Full visit
Lateral thoracolumbar spine X-Rays will be collected and sent to a central reader for evaluation of vertebral fractures

Trial sites (18)

Facility	City	Region
UZ Gent (Universitair Ziekenhuis Gent)	Ghent	Belgium
UZ Leuven (Universitair Ziekenhuis Leuven)	Leuven	Belgium
University Hospital Brno	Brno	Czechia
Fakultni Nemocnice Motol	Prague	Czechia
Children's Hospital Agia Sofia	Athens	Greece
Children's Health Ireland at Tallaght, Tallaght University Hospital	Dublin	Ireland
Schneider Children's Medical Center	Petah Tikva	Israel
Leiden University Medical Center	Leiden	Netherlands
Radboud University Nijmegen	Nijmegen	Netherlands
Te Wao Nui - Child Health Service, Wellington Hospital	Wellington	New Zealand
Hospital Universitario Puerta de Hierro Majadahonda	Madrid	Spain
Hospital Universitario y Politecnico de La Fe	Valencia	Spain
Queen Elizabeth University Hospital	Glasgow	Lanarkshire
Alder Hey Children's Hospital	Liverpool	Merseyside
Leeds Teaching Hospitals NHS Trust	Leeds	West Yorkshire
University Hospitals Birmingham NHS Foundation Trust	Birmingham	United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust	London	United Kingdom
The John Walton Muscular Dystrophy Research Centre	Newcastle	United Kingdom

Other trials for the same condition

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) Active, not recruiting

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

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study Recruiting

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy Active, not recruiting

Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy Enrolling by invitation

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06713135 on ClinicalTrials.gov ↗ ← All trials in the UK