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NCT Number: NCT06713135

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Sex eligibility

Male

Study type

Interventional

Phase

Phase 4

Primary location

UZ Gent (Universitair Ziekenhuis Gent), Ghent, Belgium

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

vamorolone 40 mg/mL oral suspension

Drug

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

Primary outcomes

  1. 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

Secondary outcomes

  1. Time to first vertebral fractures (cumulative incidence)

    Time frame: From enrolment up to at least 2 years

  2. 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

  3. Time to first non-vertebral fractures (cumulative incidence)

    Time frame: From enrolment until up to at least 3 years

  4. 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

  5. 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.

  6. 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.

  7. Change from baseline in body weight

    Time frame: From enrolment until up to at least 3 years

    Physical examination will include weight (in kg)

  8. 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.

  9. 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)

  10. 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.

  11. Change from baseline in 6MWT distance

    Time frame: From enrolment until up to at least 3 years

  12. 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.

  13. 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.

  14. 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.

  15. 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

Sponsors and collaborators

Lead sponsor

Santhera Pharmaceuticals

Industry

Registry information

Official study 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

Acronym: GUARDIAN

Important dates

Study start
2024
Primary completion
2028
Study completion
2028
First posted
Dec 3, 2024
Registry last updated
Apr 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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