NCT06713135

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

• Status: Active Not Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 80 (estimated)
• Sponsor: Santhera Pharmaceuticals · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed description

All subjects in this study have completed previous studies with vamorolone and continued to receive vamorolone under special programs: Compassionate Use Program \[CUP\], Named Patient Program \[NPP\] or Expanded Access Protocol \[EAP\]. All subjects will continue treatment with vamorolone under Guardian protocol instead. The primary objective of this study is to evaluate the safety of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy regarding vertebral fractures. Secondary study objectives will evaluate the safety of long-term treatment with vamorolone on non-vertebral fractures, cataracts, delayed puberty, overall safety as well as ambulatory and non-ambulatory function.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2024-11-10
• Primary completion: 2028-09-01
• Completion: 2028-09-01
• First posted: 2024-12-03
• Last updated: 2026-01-09

Locations

18 sites across 9 countries: Belgium, Czechia, Greece, Ireland, Israel, Netherlands, New Zealand, Spain, United Kingdom