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

PHASE4RecruitingINTERVENTIONAL
Enrollment

80

Participants

Timeline

Start Date

November 10, 2024

Primary Completion Date

September 30, 2028

Study Completion Date

September 30, 2028

Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG

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.

Trial Locations (12)

3000

NOT_YET_RECRUITING

UZ Leuven (Universitair Ziekenhuis Leuven), Leuven

9000

NOT_YET_RECRUITING

UZ Gent (Universitair Ziekenhuis Gent), Ghent

28222

NOT_YET_RECRUITING

Hospital Universitario Puerta de Hierro Majadahonda, Madrid

46026

NOT_YET_RECRUITING

Hospital Universitario y Politecnico de La Fe, Valencia

613 00

NOT_YET_RECRUITING

University Hospital Brno, Brno

150 06

RECRUITING

Fakultni Nemocnice Motol, Prague

Unknown

NOT_YET_RECRUITING

Queen Elizabeth University Hospital, Glasgow

L14 5AB

NOT_YET_RECRUITING

Alder Hey Children's Hospital, Liverpool

LS1 3EX

NOT_YET_RECRUITING

Leeds Teaching Hospitals NHS Trust, Leeds

B9 5SS

NOT_YET_RECRUITING

University Hospitals Birmingham NHS Foundation Trust, Birmingham

WC1N 3JH

NOT_YET_RECRUITING

Great Ormond Street Hospital for Children NHS Foundation Trust, London

NE1 3BZ

NOT_YET_RECRUITING

The John Walton Muscular Dystrophy Research Centre, Newcastle

Sponsors
All Listed Sponsors
lead

Santhera Pharmaceuticals

INDUSTRY