A Phase 4 interventional study of vamorolone 40 mg/mL oral suspension in Duchenne Muscular Dystrophy, sponsored by Santhera Pharmaceuticals. Active, not recruiting at 18 sites in 9 countries. Open to male participants. Per ClinicalTrials.gov, last updated 2026-04-23.
Sponsored by Santhera Pharmaceuticals · Phase 4, Interventional, and Treatment
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Among 84 studies in Muscular Dystrophy, Duchenne with a site in United Kingdom — 15 open to participants now, 25 under way, 7 of them phase 3. Muscular Dystrophy, Duchenne · United Kingdom.
Among the 84 studies in Muscular Dystrophy, Duchenne with a site in United Kingdom, this one is the 33rd largest by planned enrollment of the 72 interventional ones that give a count, is one of 5 that started in 2024, is the only one in phase 4, reaches primary completion later than 12 of the 24 other live ones, and was the 75th registered.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.
Browse Muscular Dystrophy, Duchenne studies →15 of the 84 studies in Muscular Dystrophy, Duchenne with a site in United Kingdom are open now. Recruiting first, the newest first:
Avidity Biosciences, Inc. · Sites in United Kingdom, Australia, Belgium and 7 more countries
Ages 7–16 · Men only
University of Oxford · Sites in United Kingdom
Ages 1–3 · Men only · Healthy volunteers
Entrada Therapeutics, Inc. · Sites in United Kingdom, Belgium, Italy and 2 more countries
Ages 4–20 · Men only
Entrada Therapeutics, Inc. · Sites in United Kingdom, Belgium, Italy and 1 more country
Ages 4–20 · Men only
Italfarmaco · Sites in United Kingdom, Belgium, Italy and 1 more country
Ages 2–6 · Men only
A study can be open while its sites in United Kingdom have not opened yet or have stopped. Only those recruiting at a site in United Kingdom →
Of the 66 Muscular Dystrophy, Duchenne studies recruiting now, 30 (45%) set no upper age limit and 34 (52%) stop at 75 or younger; 59 take children under 18; 12 take healthy volunteers.
This study: male · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
84 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 15 · Phase 2 37 · Phase 3 30 · Phase 4 1
First in 2007; this study’s year is marked. 5 started in 2024.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 80 is above the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Median enrollment 74 across the 72 interventional studies, 85 across the 11 observational. Of the 56 that completed or were terminated, 32 (57%) have results posted.
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67 of 84 have an industry lead sponsor. The 10 most frequent:
Santhera Pharmaceuticals is the lead sponsor of 27 studies on the registry; 1 is open to participants now.
Santhera Pharmaceuticals has 9 studies filed under Muscular Dystrophy, Duchenne — fewer than ten, so there is no comparison to draw.
5 of the studies in Muscular Dystrophy, Duchenne with a site in United Kingdom that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05881408Phase 3Sarepta Therapeutics, Inc.A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) | 2027-05-31 |
| NCT07287189Satellos Bioscience, Inc.Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients | 2027-03-31 |
| NCT06280209BioMarin PharmaceuticalA Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy | 2027-04-14 |
| NCT06138639Solid Biosciences Inc.A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) | 2027-05-06 |
| NCT07423026University of OxfordA Remote Study Using Technology to Assess Outcomes in DMD | 2027-10-01 |
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Among the 84 studies in Muscular Dystrophy, Duchenne with a site in United Kingdom, from their registry updates:
This tab as a report to print or save as PDF → With an account.
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.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Drug page: Vamorolone
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.
Also known as: vamorolone
Number of vertebral fractures per 1000 person-years based on X-ray central reading.
Lateral thoracolumbar spine X-Rays will be collected and sent to a central reader for evaluation of vertebral fractures
Time frame: At Enrolment and every 2 years during a Full visit
Time to first vertebral fractures (cumulative incidence)
Time frame: From enrolment up to at least 2 years
Number of non-vertebral fractures per 1000 person-years based on investigator reporting
Non-vertebral fractures will be reported by investigators and not reviewed centrally
Time frame: 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
Number of cataracts per 1000 person-years based on ophthalmologist assessment
An ophthalmologist assessment including assessment of posterior capsular cataracts by slit lamp will be performed yearly
Time frame: From enrolment until up to at least 3 years
Number of subjects not reaching Tanner stage 2 by 15 years of age
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.
Time frame: From enrolment until up to at least 3 years
Frequency of adverse events (AEs) and serious adverse events (SAEs)
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.
Time frame: From enrolment until up to at least 3 years
Change from baseline in body weight
Physical examination will include weight (in kg)
Time frame: From enrolment until up to at least 3 years
Number of subjects with clinically relevant laboratory abnormalities
Clinically relevant laboratory abnormalities will include HbA1c and morning cortisol measurements.
Time frame: From enrolment until up to at least 3 years
Change from baseline in Time to Stand (TTSTAND) velocity
The TTSTAND measures the time (in seconds) required for the subject to stand to an erect position from a supine position (floor)
Time frame: From enrolment until up to at least 3 years
Six-minute Walk Test (6MWT)
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.
Time frame: 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
Age at ambulatory and non-ambulatory milestones
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.
Time frame: From enrolment until up to at least 3 years
North Star Ambulatory Assessment (NSAA) scores
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.
Time frame: At enrolment and each Full visit (Month 12, 24, 36, etc / End-of -Treatment) until End of Study
Change from baseline in body height
Physical examination will include height (in cm). Ulnar length of the non-dominant arm will be used if standing height cannot be measured.
Time frame: From enrolment until up to at least 3 years
Change from baseline in Body Mass Index (BMI)
BMI will be derived based on weight and height at every study visit
Time frame: From enrolment until up to at least 3 years
Started Nov 2024 (actual). Primary completion is estimated for Sep 2028, 46 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Vamorolone
Muscular Dystrophy, Duchenne →