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

Last updated: January 7, 2026
Sponsor: Santhera Pharmaceuticals
Overall Status: Active - Not Recruiting

Phase

4

Condition

Muscular Dystrophy

Treatment

vamorolone 40 mg/mL oral suspension

Clinical Study ID

NCT06713135
SNT-IV-VAM-011
  • Male

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 Criteria

Inclusion

Inclusion Criteria:

  • Subject and/or subject's parent(s) or legal guardian has provided written informedconsent

  • Subject has previously completed either the VBP15-LTE or VBP15-004 study, andtransitioned through the Compassionate Use Program, Named Patient Program orExpanded Acess Protocol

  • Subject is on vamorolone on day of enrolment

  • Subject and parent / legal guardian are willing and able to comply with the protocolschedule, assessments and requirements

Exclusion

Exclusion Criteria:

  • Any medical condition, which in the opinion of the Investigator, would affect studyparticipation, performance or interpretation of study assessments

  • Vamorolone treatment discontinued for ≥ 6 months within the year prior to enrolmentfor a non-safety reason, or vamorolone treatment previously discontinued at any timefor a safety reason

  • Severe hepatic impairment

Study Design

Total Participants: 80
Treatment Group(s): 1
Primary Treatment: vamorolone 40 mg/mL oral suspension
Phase: 4
Study Start date:
November 10, 2024
Estimated Completion Date:
September 30, 2028

Study 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.

Connect with a study center

  • UZ Gent (Universitair Ziekenhuis Gent)

    Gent, 9000
    Belgium

    Site Not Available

  • UZ Gent (Universitair Ziekenhuis Gent)

    Ghent 2797656, 9000
    Belgium

    Site Not Available

  • UZ Leuven (Universitair Ziekenhuis Leuven)

    Leuven, 3000
    Belgium

    Site Not Available

  • UZ Leuven (Universitair Ziekenhuis Leuven)

    Leuven 2792482, 3000
    Belgium

    Site Not Available

  • University Hospital Brno

    Brno, 613 00
    Czechia

    Site Not Available

  • University Hospital Brno

    Brno 3078610,
    Czechia

    Site Not Available

  • Fakultni Nemocnice Motol

    Prague, 150 06
    Czechia

    Site Not Available

  • Fakultni Nemocnice Motol

    Prague 3067696, 150 06
    Czechia

    Site Not Available

  • Children's Hospital Agia Sofia

    Athens 264371, 115 27
    Greece

    Site Not Available

  • Children's Health Ireland at Tallaght, Tallaght University Hospital

    Dublin 2964574, D24TN3C
    Ireland

    Site Not Available

  • Schneider Children's Medical Center

    Petah Tikva 293918, 4920235
    Israel

    Site Not Available

  • Leiden University Medical Center

    Leiden 2751773, 2333 ZA
    Netherlands

    Site Not Available

  • Radboud University Nijmegen

    Nijmegen 2750053, 6525 HB
    Netherlands

    Site Not Available

  • Te Wao Nui - Child Health Service, Wellington Hospital

    Wellington 2179537, 6021
    New Zealand

    Site Not Available

  • Hospital Universitario Puerta de Hierro Majadahonda

    Madrid, 28222
    Spain

    Site Not Available

  • Hospital Universitario Puerta de Hierro Majadahonda

    Madrid 3117735, 28222
    Spain

    Site Not Available

  • Hospital Universitario y Politecnico de La Fe

    Valencia, 46026
    Spain

    Site Not Available

  • Hospital Universitario y Politecnico de La Fe

    Valencia 2509954, 46026
    Spain

    Site Not Available

  • Queen Elizabeth University Hospital

    Glasgow, Lanarkshire
    United Kingdom

    Site Not Available

  • Queen Elizabeth University Hospital

    Glasgow 2648579, Lanarkshire
    United Kingdom

    Site Not Available

  • Alder Hey Children's Hospital

    Liverpool, Merseyside L14 5AB
    United Kingdom

    Site Not Available

  • Alder Hey Children's Hospital

    Liverpool 2644210, Merseyside L14 5AB
    United Kingdom

    Site Not Available

  • Leeds Teaching Hospitals NHS Trust

    Leeds, West Yorkshire LS1 3EX
    United Kingdom

    Site Not Available

  • Leeds Teaching Hospitals NHS Trust

    Leeds 2644688, West Yorkshire LS1 3EX
    United Kingdom

    Site Not Available

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham, B9 5SS
    United Kingdom

    Site Not Available

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham 2655603, B9 5SS
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, WC1N 3JH
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London 2643743, WC1N 3JH
    United Kingdom

    Site Not Available

  • The John Walton Muscular Dystrophy Research Centre

    Newcastle, NE1 3BZ
    United Kingdom

    Site Not Available

  • The John Walton Muscular Dystrophy Research Centre

    Newcastle 6695976, NE1 3BZ
    United Kingdom

    Site Not Available

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