*The GUARDIAN Study
Research type
Research Study
Full title
*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
IRAS ID
1010157
Contact name
Waldy Caroline
Contact email
Sponsor organisation
Santhera Pharmaceuticals (Switzerland) Ltd.
Clinicaltrials.gov Identifier
Research summary
This trial is a Phase 4, open-label, multi-center trial designed to collect long-term safety and effectiveness data from boys with Duchenne Muscular Dystrophy (DMD) who have participated in studies VBP15-002/003/LTE and VBP15-004 of vamorolone clinical development program.
The primary aim of the study is to evaluate the safety of long-term treatment with vamorolone in boys with DMD regarding vertebral fractures.
The study is divided in 3 periods:1. Enrolment period: between informed consent form (ICF) sign off and 1st dose of study treatment (Day 1)
2. Treatment period of at least 3 years: between Day 1 and End-of-Treatment (EOT), the subject visits the study site every 6-months
3. Follow-up period lasting at least 12 weeks: between EOT and End-of-Study (EOS). In cases where a subject discontinues study medication but switches to Agamree® (commercially available vamorolone), there is no Follow-up period (EOT and EOS are combined).REC name
Wales REC 2
REC reference
24/WA/0221
Date of REC Opinion
20 Aug 2024
REC opinion
Further Information Favourable Opinion