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

April 22, 2026 updated by: Santhera Pharmaceuticals

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

80

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Ghent, Belgium, 9000
        • UZ Gent (Universitair Ziekenhuis Gent)
      • Leuven, Belgium, 3000
        • UZ Leuven (Universitair Ziekenhuis Leuven)
  • Czechia
      • Brno, Czechia
        • University Hospital Brno
      • Prague, Czechia, 150 06
        • Fakultni nemocnice Motol
  • Greece
      • Athens, Greece, 115 27
        • Children's Hospital Agia Sofia
  • Ireland
      • Dublin, Ireland, D24TN3C
        • Children's Health Ireland at Tallaght, Tallaght University Hospital
  • Israel
      • Petah Tikva, Israel, 4920235
        • Schneider Children's Medical Center
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • Leiden University Medical Center
      • Nijmegen, Netherlands, 6525 HB
        • Radboud University Nijmegen
  • New Zealand
      • Wellington, New Zealand, 6021
        • Te Wao Nui - Child Health Service, Wellington Hospital
  • Spain
      • Madrid, Spain, 28222
        • Hospital Universitario Puerta de Hierro Majadahonda
      • Valencia, Spain, 46026
        • Hospital Universitario y Politécnico de La Fe
  • United Kingdom
      • Birmingham, United Kingdom, B9 5SS
        • University Hospitals Birmingham NHS Foundation Trust
      • London, United Kingdom, WC1N 3JH
        • Great Ormond Street Hospital For Children NHS Foundation Trust
      • Newcastle, United Kingdom, NE1 3BZ
        • The John Walton Muscular Dystrophy Research Centre
    • Lanarkshire
      • Glasgow, Lanarkshire, United Kingdom
        • Queen Elizabeth University Hospital
    • Merseyside
      • Liverpool, Merseyside, United Kingdom, L14 5AB
        • Alder Hey Children's Hospital
    • West Yorkshire
      • Leeds, West Yorkshire, United Kingdom, LS1 3EX
        • Leeds Teaching Hospitals NHS Trust

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: vamorolone
On Day 1, Subjects will roll over from a previous vamorolone program and continue treatment with vamorolone under this protocol. During the study, vamorolone will be 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.
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.
Other Names:
  • vamorolone

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of vertebral fractures per 1000 person-years based on X-ray central reading.
Time Frame: At Enrolment and every 2 years during a Full visit
Lateral thoracolumbar spine X-Rays will be collected and sent to a central reader for evaluation of vertebral fractures
At Enrolment and every 2 years during a Full visit

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to first vertebral fractures (cumulative incidence)
Time Frame: From enrolment up to at least 2 years
From enrolment up to at least 2 years
Number of non-vertebral fractures per 1000 person-years based on investigator reporting
Time Frame: From enrolment until up to at least 3 years
Non-vertebral fractures will be reported by investigators and not reviewed centrally
From enrolment until up to at least 3 years
Time to first non-vertebral fractures (cumulative incidence)
Time Frame: From enrolment until up to at least 3 years
From enrolment until up to at least 3 years
Number of cataracts per 1000 person-years based on ophthalmologist assessment
Time Frame: From enrolment until up to at least 3 years
An ophthalmologist assessment including assessment of posterior capsular cataracts by slit lamp will be performed yearly
From enrolment until up to at least 3 years
Number of subjects not reaching Tanner stage 2 by 15 years of age
Time Frame: From enrolment until up to at least 3 years
Qualified personnel (eg, an individual, part of the endocrinology team and trained to assess Tanner stages) will provide an assessment of the puberty status of the subject, including testicular volume.
From enrolment until up to at least 3 years
Frequency of adverse events (AEs) and serious adverse events (SAEs)
Time Frame: From enrolment until up to at least 3 years
The occurrence of AEs should be sought by questioning of the subject and/or his caregiver at each visit or phone call during the study. All SAEs occurring from signature of the ICF up to 30 days after last dose of study medication must be reported, irrespective of severity or whether or not considered related to study medication. All SAEs must be reported within 24 hours of becoming aware of the event, whether or not the SAE is considered to be related to the study medication.
From enrolment until up to at least 3 years
Change from baseline in body weight
Time Frame: From enrolment until up to at least 3 years
Physical examination will include weight (in kg)
From enrolment until up to at least 3 years
Number of subjects with clinically relevant laboratory abnormalities
Time Frame: From enrolment until up to at least 3 years
Clinically relevant laboratory abnormalities will include HbA1c and morning cortisol measurements.
From enrolment until up to at least 3 years
Change from baseline in Time to Stand (TTSTAND) velocity
Time Frame: From enrolment until up to at least 3 years
The TTSTAND measures the time (in seconds) required for the subject to stand to an erect position from a supine position (floor)
From enrolment until up to at least 3 years
Six-minute Walk Test (6MWT)
Time Frame: From enrolment until up to at least 3 years
6MWT will be performed only in the ambulatory subjects. The total distance traveled, in meters, should be recorded along with the validity of the test as assessed by the test administrator. If a subject cannot complete 6 minutes of walking, the total meters and the time until discontinuation of the test should be recorded.
From enrolment until up to at least 3 years
Change from baseline in 6MWT distance
Time Frame: From enrolment until up to at least 3 years
From enrolment until up to at least 3 years
Age at ambulatory and non-ambulatory milestones
Time Frame: From enrolment until up to at least 3 years
Ambulatory milestones include Loss of standing from the floor, Loss of ability to climb 4-stairs, Loss of ability to walk 10 meters (loss of ambulation), Loss of ability to stand unassisted. Non-ambulatory milestones include Loss of ability to perform hand-to-mouth function, Loss of ability to use a manual wheelchair, Loss of ability to transfer independently from wheelchair, Nocturnal ventilation and Full time ventilation.
From enrolment until up to at least 3 years
North Star Ambulatory Assessment (NSAA) scores
Time Frame: At enrolment and each Full visit (Month 12, 24, 36, etc / End-of -Treatment) until End of Study
The NSAA is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with DMD and allows to monitor the progression of the disease and treatment effects.
At enrolment and each Full visit (Month 12, 24, 36, etc / End-of -Treatment) until End of Study
Change from baseline in body height
Time Frame: From enrolment until up to at least 3 years
Physical examination will include height (in cm). Ulnar length of the non-dominant arm will be used if standing height cannot be measured.
From enrolment until up to at least 3 years
Change from baseline in Body Mass Index (BMI)
Time Frame: From enrolment until up to at least 3 years
BMI will be derived based on weight and height at every study visit
From enrolment until up to at least 3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Investigators

  • Principal Investigator: A Child, MD, Leeds Teaching Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 10, 2024

Primary Completion (Estimated)

September 1, 2028

Study Completion (Estimated)

September 1, 2028

Study Registration Dates

First Submitted

November 16, 2024

First Submitted That Met QC Criteria

November 26, 2024

First Posted (Actual)

December 3, 2024

Study Record Updates

Last Update Posted (Actual)

April 23, 2026

Last Update Submitted That Met QC Criteria

April 22, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNT-IV-VAM-011

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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