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Santhera GUARDIAN
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy (GUARDIAN)
Hub Summary
This Phase 4 study aims to assess safety and effectivness of long-term treatment with vamoralone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamoralone.
Study Number: NCT06713135
Description by Santhera Pharmaceuticals
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.
Secondary Outcome Measures
- Time to first vertebral fractures (cumulative incidence)
- Number of non-vertebral fractures per 1000 person-years based on investigator reporting
- Time to first non-vertebral fractures (cumulative incidence)
- Number of cataracts per 1000 person-years based on ophthalmologist assessment
- Number of subjects not reaching Tanner stage 2 by 15 years of age
- Frequency of adverse events (AEs) and serious adverse events (SAEs)
- Change from baseline in body weight
- Number of subjects with clinically relevant laboratory abnormalities
- Change from baseline in Time to Stand (TTSTAND) velocity
- Six-minute Walk Test (6MWT)
- Change from baseline in 6MWT distance
- Age at ambulatory and non-ambulatory milestones
- North Star Ambulatory Assessment (NSAA) scores
- Change from baseline in body height
- Change from baseline in Body Mass Index (BMI)
Primary Outcome Measures
- Number of vertebral fractures per 1000 person-years based on X-ray central reading.
Can I take part?
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
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Not yet recruiting