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CompletedNCT05185622Updated Oct 24, 2025Results posted

A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

A Phase 2 interventional study of Vamorolone in Duchenne Muscular Dystrophy, sponsored by Santhera Pharmaceuticals. Completed at 5 sites in Canada. Open to male participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2025-10-24.

Sponsored by Santhera Pharmaceuticals · Phase 2, Interventional, and Treatment

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Phase
Phase 2
Study type
Interventional
Enrollment
54
Allocation
Non-randomized
Ages
2 Years to 17 Years
Sex
Male
01

Where this study stands

Counted across the records on this site · 2026-11-14

Among 65 studies in Muscular Dystrophy, Duchenne with a site in Canada — 5 open to participants now, 14 under way, 8 of them phase 3. Muscular Dystrophy, Duchenne · Canada.

Enrollment
38th of 61
54 · median 70
Started
2022
with 1 other
Phase 2
17 of 65
none open now
Registered
54th of 65
by date

Among the 65 studies in Muscular Dystrophy, Duchenne with a site in Canada, this one is the 38th largest by enrollment of the 61 interventional ones that give a count, is one of 2 that started in 2022, is one of 17 in phase 2, and was the 54th registered.

Muscular Dystrophy, Duchenne

481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.

Browse Muscular Dystrophy, Duchenne studies →

Open to participants now

5 of the 65 studies in Muscular Dystrophy, Duchenne with a site in Canada are open now. Recruiting first, the newest first:

All 5 open studies →

A study can be open while its sites in Canada have not opened yet or have stopped. Only those recruiting at a site in Canada →

Who these studies take

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: 2 Years to 17 Years · male · no healthy volunteers.

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02

Study summary

This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in steroid-naïve boys ages 2 to \<4 years, and glucocorticoid-treated and currently untreated boys ages 7 to \<18 years with DMD.

Read the detailed description

This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg over a treatment period of 3 months in steroid-naïve boys ages 2 to \<4, and glucocorticoid-treated and currently untreated boys ages 7 to \<18 years with DMD.

The study is comprised of a 5-week Pretreatment Screening Period; a 1-day Pretreatment Baseline Period; a 3-month open-label Treatment Period (Weeks 1-12); and a 4-8 week open-label Dose-tapering Period (starting from Weeks 13) for subjects who will not transition directly to further vamorolone or standard of care (SoC) glucocorticoid treatment at the end of the study.

Subjects will be enrolled into the study at the Screening Visit, at the time written informed consent is obtained.

Within the 2 to \<4 years age group, the initial 10 eligible subjects will be assigned to the 2.0 mg/kg/day treatment group at the Baseline Day -1 Visit. The subsequent 10 eligible subjects will be assigned to the 6.0 mg/kg/day treatment group at the Baseline Day -1 Visit.

Within the 7 to \<18 years age group, both corticosteroid-treated and untreated, the initial 12 eligible subjects will be assigned to the 2.0 mg/kg/day treatment group at the Baseline Day -1 Visit. The subsequent 12 eligible subjects will be assigned to the 6.0 mg/kg/day treatment group at the Baseline Day -1 Visit.

The first 6 subjects in each age group at 2 mg/kg will serve as the PK/safety run-in cohorts. PK assessments will be performed at week 2 and together with the safety assessment during the first 4 weeks of treatment this will be the basis to confirm whether 2 and 6 mg/kg/day will be used in the subsequent patients or if a dose adjustment is needed to avoid over or under-exposure in patients for any of the two age groups.

Glucocorticoid-treated subjects in the 7 to \<18 years age group will take their final dose of SoC glucocorticoid therapy for DMD on Baseline Day -1, within 24 hours prior to administration of the first dose of vamorolone study medication.

All subjects in both age groups will begin their assigned vamorolone treatment on Treatment Period Day 1, and will continue to receive their assigned vamorolone treatment throughout the duration of the 3 month Treatment Period (Weeks 1-12).

At the end of the 3-month Treatment Period (Week 12), subjects will be given the option to receive vamorolone in an expanded access or compassionate use program, if possible, or to transition to SoC treatment for DMD (may include glucocorticoids). Subjects completing VBP15-006 and enrolling directly into the expanded access or compassionate use program or transitioning directly to SoC glucocorticoid treatment will not need to taper their vamorolone dose prior to participation in the expanded access or compassionate use program or initiation of SoC glucocorticoid treatment. All subjects who will not transition directly to further vamorolone or SoC glucocorticoid treatment will begin a 4 -8 week open label Dose tapering Period during which the dose of study medication will be progressively reduced and discontinued.

03

Conditions studied

  • Duchenne Muscular Dystrophy

Keywords

  • Duchenne Muscular Dystrophy
  • Becker Muscular Dystrophy
  • Duchenne and Becker Muscular Dystrophy
  • Muscular Dystrophy
04

Who can participate

Ages eligible
2 Years to 17 Years
Sexes eligible
Male
Accepts healthy volunteers
No

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.

Inclusion criteria

  1. Subject's parent(s) or legal guardian(s) has (have) provided written and Health Insurance Portability and Accountability Act (HIPAA) authorization, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to local requirements;

  2. Subject has a centrally confirmed (by TRiNDS central genetic counselor[s]) diagnosis of DMD, defined as:

    1. Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
    2. Identifiable mutation within the DMD gene (deletion/duplication of one or more exons), where reading frame can be predicted as 'out-of-frame,' and clinical picture consistent with typical DMD, OR
    3. Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e., nonsense mutation, deletion/duplication leading to a downstream stop codon), with a clinical picture consistent with typical DMD;
  3. Subject is male, 2 to \<4 years or 7 to \<18 years of age at time of enrollment in the study;
  4. If 7 to \<18 years of age and currently taking standard of care for treatment of DMD, subject has been taking standard of care glucocorticoids at stable dose for at least 3 months prior to enrollment in the study, and will continue the same stable dose regimen through the date of the Baseline Day -1 Visit. [Note: Inhaled and/or topical glucocorticoids are permitted if last use is at least 4 weeks prior to enrollment or if administered at stable dose beginning at least 4 weeks prior to enrollment and anticipated to be used at the stable dose regimen for the duration of the study];
  5. If 7 to \<18 years of age, and not currently glucocorticoid-treated, subject has not received oral glucocorticoids or other oral agents for at least 3 months prior to enrollment. [Note: Inhaled and/or topical glucocorticoids are permitted if last use is at least 4 weeks prior to enrollment or if administered at stable dose beginning at least 4 weeks prior to enrollment and anticipated to be used at the stable dose regimen for the duration of the study];
  6. Clinical laboratory test results are within the normal range at the Screening Visit, or if abnormal, are not , in the opinion of the Investigator. [Notes: Serum gamma glutamyl transferase (GGT), creatinine, and total all must be ≤ at the Screening Visit. An abnormal vitamin D level that is considered clinically significant will not exclude a subject from participating];

  7. Subject has evidence of chicken pox immunity as determined by:

    • Presence of IgG antibodies to varicella, as documented by a positive test result from the local laboratory from blood collected during the Screening Period; OR
    • Documentation, provided at the Screening Visit, that the subject has had 2 doses of varicella vaccine, with or without serologic evidence of immunity; the second of the 2 immunizations must have been given at least 14 days prior to assignment to a dose group;
  8. Subject and parent(s)/guardian(s) are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

Exclusion criteria

Exclusion Criteria:

  1. Subject has current or history of major renal or hepatic impairment, diabetes mellitus or ;
  2. Subject has current or history of chronic systemic fungal or viral infections;
  3. Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), or mexrenone (mexrenoate potassium) within 4 weeks prior to enrollment;
  4. Subject has a history of primary hyperaldosteronism;
  5. Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
  6. If 2 to \<4 years of age, subject is currently being treated or has received previous treatment with oral or other immunosuppressive agents [Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 1 month cumulative, with last use at least 3 months prior to enrollment, will be considered for eligibility on a case-by-case basis, unless discontinued for intolerance. Inhaled and/or topical glucocorticoids are permitted if last use is at least 4 weeks prior to enrollment or if administered at stable dose beginning at least 4 weeks prior to enrollment and anticipated to be used at the stable dose regimen for the duration of the study];
  7. Subject has an allergy or to the study medication or to any of its constituents;
  8. Subject has used idebenone within 4 weeks prior to enrollment;
  9. Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
  10. Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;
  11. Subject is taking (or has taken within 4 weeks prior to enrollment) herbal remedies and supplements which can impact muscle strength and function (e.g., Co-enzyme Q10, creatine, etc);
  12. Subject is taking (or has taken within 3 months prior to enrollment) any medication indicated for DMD, including Exondys51, Exondys53, Exondys45, Viltepso and Translarna;
  13. Subject has been administered a within 14 days prior to the first dose of study medication;
  14. Subject is currently taking any other drug or has taken any other investigational drug within 3 months prior to enrollment;
  15. Subject has previously been enrolled in the VBP15-006 study or any other vamorolone study.
Terms in these criteria, in plain language(9)

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.

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.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Live vaccines

Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
54 participants (actual)

Study arms

  • Experimental
    Treatment Group 1

    Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2.

    Drug: Vamorolone

  • Experimental
    Treatment Group 2

    Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1.

    Drug: Vamorolone

  • Experimental
    Treatment Group 3

    Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study.

    Drug: Vamorolone

  • Experimental
    Treatment Group 4

    Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study.

    Drug: Vamorolone

  • Experimental
    Treatment Group 5

    Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study.

    Drug: Vamorolone

  • Experimental
    Treatment Group 6

    Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study.

    Drug: Vamorolone

  • Experimental
    Treatment Sub-Group 7

    Patients in Treatment Sub-Group 7 must be ages 12-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 7 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study.

    Drug: Vamorolone

Interventions

  • DrugVamorolone

    Oral administration of vamorolone for 12 weeks.

    Also known as: VBP15

06

What researchers measure

Primary outcomes

  1. Number of Participants With Any Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)

    An Adverse Event is any untoward medical occurrence in a subject and does not necessarily have to have a causal relationship with the intervention. Pre-existing conditions that worsen during the study are to be reported as AEs.

    Time frame: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed

  2. Number of Participants With Drug Related Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)

    Drug related Adverse Events are TEAEs whose Causality were labeled as 'DEFINITE', 'POSSIBLE' or 'PROBABLE

    Time frame: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed

  3. Number of Participants With Severe Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)

    Severe or medically significant but not immediately life -threatening: hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living; incapacitating with inability to work or perform normal daily activity.

    Time frame: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed

  4. Number of Participants With Serious Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)

    A Serious Adverse Event (SAE) is defined as any AE regardless of causality that meets any of the following criteria: * Results in death * Is life-threatening * Requires inpatient hospitalization or prolongation of an existing hospitalization * Results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions * Results in a congenital anomaly/birth defect * Is an important medical event that may jeopardize the subject and may require medical or surgical intervention to prevent one of the outcomes listed above

    Time frame: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose of study drug)

  5. Number of Participants With Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03) Leading to Study Treatment Discontinuation

    Adverse Events leading to Study treatment discontinuation

    Time frame: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed

  6. Change in Height (Absolute) From Baseline to Week 12

    Standing height will be assessed for subjects ages 2-\<4 years; height calculated from ulnar length in subjects ages 7-\<18.

    Time frame: Baseline, 12 weeks

  7. Change in Height (Percentile) From Baseline to Week 12

    Standing height will be assessed for subjects ages 2-\<4 years; height calculated from ulnar length in subjects ages 7-\<18.

    Time frame: Baseline, 12 weeks

  8. Change in Height (Z-score) From Baseline to Week 12

    Standing height will be assessed for subjects ages 2-\<4 years; height calculated from ulnar length in subjects ages 7-\<18. The z-score (standard score) is the number of standard deviations by which the outcome measure is above or below the value in the general population. A Z-score of 0 represents the population mean. A measure above the normal value has a positive z-score (higher height).

    Time frame: Baseline, 12 weeks

  9. Change in Weight (Absolute) From Baseline to Week 12

    Body weight will be assessed at each of the scheduled time points.

    Time frame: Baseline, 12 weeks

  10. Change in Weight (Percentile) From Baseline to Week 12

    Body weight will be assessed at each of the scheduled time points.

    Time frame: Baseline, 12 weeks

  11. Change in Weight (Z-score) From Baseline to Week 12

    Body weight will be assessed at each of the scheduled time points. The z-score (standard score) is the number of standard deviations by which the outcome measure is above or below the value in the general population. A Z-score of 0 represents the population mean. A measure above the normal value has a positive z-score (higher weight).

    Time frame: Baseline, 12 weeks

  12. Change in Body Mass Index (BMI) (Absolute) From Baseline to Week 12

    Body Mass Index is a measure of weight adjusted for height.

    Time frame: Baseline, Week 12

  13. Change in Body Mass Index (BMI) (Percentile) From Baseline to Week 12

    Body Mass Index is a measure of weight adjusted for height.

    Time frame: Baseline, Week 12

  14. Change in Body Mass Index (BMI) (Z-score) From Baseline to Week 12

    Body Mass Index is a measure of weight adjusted for height. The z-score (standard score) is the number of standard deviations by which the outcome measure is above or below the value in the general population. A Z-score of 0 represents the population mean. A measure above the normal value has a positive z-score (higher BMI).

    Time frame: Baseline, Week 12

  15. Change in Diastolic Blood Pressure

    Change from Baseline to Week 12 in diastolic sitting blood pressure.

    Time frame: Day 1, Week 2, Week 6, Week 12, Week 16

  16. Change in Systolic Blood Pressure

    Change from Baseline to Week 12 in systolic sitting blood pressure.

    Time frame: Day 1, Week 2, Week 6, Week 12, Week 16

  17. Number of Participants With Treatment Emergent Cushingoid Features

    Treatment emergent cushingoid features based on physical examination at all baseline, on-treatment and post-treatment assessments

    Time frame: Baseline through Week 16

  18. Number of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result

    Each subject had blood drawn and urine collected for the standard hematology, chemistry and lipids clinical laboratory tests. In addition, fasting glucose and insulin, morning cortisol, as well as, in the additional 12 to \<18 years age group, LH, FSH, TSH, FT4 were collected. HbA1c determination had also to be performed if urine glucose was positive and/or fasted glucose levels was above normal limits. Any treatment-emergent clinically significant abnormal laboratory test result was reporte

    Time frame: Day 1, Week 6, Week 12, Week 16

  19. Categorical Analysis of QTcF at Week 12

    12-lead 1electrocardiogram (ECG) as recorded after subject has rested quietly in a supine position for at least 5 minutes. ECG components are QRS duration, PR \[PQ\] interval, RR interval, QT interval and QTc.

    Time frame: Baseline, Week 12

  20. Number of Eyes With Cataract

    Cataract was diagnosed by the presence of partial or complete opacity of the crystalline lens at Baseline and Week 12.

    Time frame: Baseline - Week 12

  21. Number of Eyes With Glaucoma

    Glaucoma was diagnosed by ocular pressure at Baseline and Week 12.

    Time frame: Baseline - Week 12

Secondary outcomes

  1. Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2

    The plasma concentration of vamorolone was measured on Day 1 and Week 2 predose, and 1h, 2h and 6h postdose and also 4h and 8h post in the 7-18 year groups.

    Time frame: Day 1, Week 2

  2. Descriptive Statistics of PK Parameters - Tmax

    Tmax is the time to reach the maximum observed concentration collected during a dosing interval

    Time frame: Day 1, Week 2

  3. Descriptive Statistics of PK Parameters - Cmax

    Cmax is the maximum observed concentration

    Time frame: Day 1, Week 2

  4. Descriptive Statistics of PK Parameters in Subjects Aged 2 to 4 Years - AUC 0-6

    AUC 0-6 is the area under the concentration-time curve during the first 6 hours after dosing

    Time frame: Day 1, Week 2

  5. Descriptive Statistics of PK Parameters in Subjects Aged 7 to 18 Years - AUC 0-inf

    AUC 0-8 is the area under the concentration-time curve after dosing extrapolated to infinity

    Time frame: Day 1, Week 2

Other outcomes

  1. Change From Baseline to Week 12 in Bayley-III Gross Motor Scale (Ages 2 to <4 Years Only)

    The Bayley-III Gross Motor scale is a functional assessment, an accurate reflection of muscle strength for subjects with DMD ages 2 to \<4 years. The minimum score value is 0 and the maximum score value is 72. Higher scores mean a better outcome.

    Time frame: Baseline, Week 12

  2. Change From Baseline to Week 12 in Morning Cortisol Concentration

    Morning cortisol \[adrenal suppression\] samples were collected after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication at Day 1 and Week 12 Visits, before 10 AM local time

    Time frame: Baseline, Week 12

  3. Change From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Type 1 Collagen C-telopeptide [CTX1])

    Samples for CTX1, osteocalcin and P1NP were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication

    Time frame: Baseline, Week 12

  4. Change From Baseline to Week 12 in Bone Turnover Biomarkers (Osteocalcin)

    Samples for CTX1, osteocalcin and P1NP were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication

    Time frame: Baseline, Week 12

  5. Change From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Aminoterminal Propeptide of Type I Collagen [P1NP] )

    Samples for CTX1, osteocalcin and P1NP were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication

    Time frame: Baseline, Week 12

  6. Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Glucose)

    Glucose, HbA1c and insulin were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication.

    Time frame: Baseline, Week 12

  7. Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Hemoglobin A1c [HbA1c])

    Glucose, HbA1c and insulin were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication. The Baseline sample for HbA1c measurement may have been collected non-fasting.

    Time frame: Baseline, Week 12

  8. Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Insulin)

    Glucose, HbA1c and insulin were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication.

    Time frame: Baseline, Week 12

07

Results

Posted Oct 24, 2025

Participant flow

Participant flow — Overall Study
MilestoneTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Started101066616
Completed101066616
Not completed000000

Outcome measures

PrimaryNumber of Participants With Any Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)

An Adverse Event is any untoward medical occurrence in a subject and does not necessarily have to have a causal relationship with the intervention. Pre-existing conditions that worsen during the study are to be reported as AEs.

Time frame:
From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed
Reported as:
Count of participants · Participants
Number of Participants With Any Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)
ParticipantsTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Number of Participants With Any Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)7964312
PrimaryNumber of Participants With Drug Related Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)

Drug related Adverse Events are TEAEs whose Causality were labeled as 'DEFINITE', 'POSSIBLE' or 'PROBABLE

Time frame:
From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed
Reported as:
Count of participants · Participants
Number of Participants With Drug Related Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)
ParticipantsTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Number of Participants With Drug Related Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)173418
PrimaryNumber of Participants With Severe Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)

Severe or medically significant but not immediately life -threatening: hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living; incapacitating with inability to work or perform normal daily activity.

Time frame:
From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed
Reported as:
Count of participants · Participants
Number of Participants With Severe Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)
ParticipantsTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Number of Participants With Severe Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)000101
PrimaryNumber of Participants With Serious Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)

A Serious Adverse Event (SAE) is defined as any AE regardless of causality that meets any of the following criteria: * Results in death * Is life-threatening * Requires inpatient hospitalization or prolongation of an existing hospitalization * Results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions * Results in a congenital anomaly/birth defect * Is an important medical event that may jeopardize the subject and may require medical or surgical intervention to prevent one of the outcomes listed above

Time frame:
From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose of study drug)
Reported as:
Count of participants · Participants
Number of Participants With Serious Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)
ParticipantsTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Number of Participants With Serious Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)000101
PrimaryNumber of Participants With Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03) Leading to Study Treatment Discontinuation

Adverse Events leading to Study treatment discontinuation

Time frame:
From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed
Reported as:
Count of participants · Participants
Number of Participants With Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03) Leading to Study Treatment Discontinuation
ParticipantsTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Number of Participants With Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03) Leading to Study Treatment Discontinuation000000
PrimaryChange in Height (Absolute) From Baseline to Week 12

Standing height will be assessed for subjects ages 2-\<4 years; height calculated from ulnar length in subjects ages 7-\<18.

Time frame:
Baseline, 12 weeks
Reported as:
Mean · cm
Change in Height (Absolute) From Baseline to Week 12
cmTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Height (Absolute) From Baseline to Week 121.98 ± 1.1462.35 ± 0.5680.55 ± 0.5572.29 ± 1.6090.60 ± 0.5941.27 ± 1.313
PrimaryChange in Height (Percentile) From Baseline to Week 12

Standing height will be assessed for subjects ages 2-\<4 years; height calculated from ulnar length in subjects ages 7-\<18.

Time frame:
Baseline, 12 weeks
Reported as:
Mean · percentile
Change in Height (Percentile) From Baseline to Week 12
percentileTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Height (Percentile) From Baseline to Week 121.17 ± 11.5382.09 ± 5.634-3.89 ± 3.9072.02 ± 8.338-3.69 ± 3.408-0.30 ± 1.390
PrimaryChange in Height (Z-score) From Baseline to Week 12

Standing height will be assessed for subjects ages 2-\<4 years; height calculated from ulnar length in subjects ages 7-\<18. The z-score (standard score) is the number of standard deviations by which the outcome measure is above or below the value in the general population. A Z-score of 0 represents the population mean. A measure above the normal value has a positive z-score (higher height).

Time frame:
Baseline, 12 weeks
Reported as:
Mean · Z-score (score on a scale)
Change in Height (Z-score) From Baseline to Week 12
Z-score (score on a scale)Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Height (Z-score) From Baseline to Week 120.04 ± 0.3610.14 ± 0.201-0.12 ± 0.1110.07 ± 0.253-0.13 ± 0.1590.00 ± 0.194
PrimaryChange in Weight (Absolute) From Baseline to Week 12

Body weight will be assessed at each of the scheduled time points.

Time frame:
Baseline, 12 weeks
Reported as:
Mean · kg
Change in Weight (Absolute) From Baseline to Week 12
kgTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Weight (Absolute) From Baseline to Week 120.43 ± 0.7760.65 ± 0.911-0.18 ± 3.2593.47 ± 3.1331.53 ± 1.1881.94 ± 2.598
PrimaryChange in Weight (Percentile) From Baseline to Week 12

Body weight will be assessed at each of the scheduled time points.

Time frame:
Baseline, 12 weeks
Reported as:
Mean · percentile
Change in Weight (Percentile) From Baseline to Week 12
percentileTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Weight (Percentile) From Baseline to Week 12-1.44 ± 12.835-1.97 ± 12.504-4.00 ± 11.67215.20 ± 2.0832.12 ± 4.2354.22 ± 8.566
PrimaryChange in Weight (Z-score) From Baseline to Week 12

Body weight will be assessed at each of the scheduled time points. The z-score (standard score) is the number of standard deviations by which the outcome measure is above or below the value in the general population. A Z-score of 0 represents the population mean. A measure above the normal value has a positive z-score (higher weight).

Time frame:
Baseline, 12 weeks
Reported as:
Mean · Z-score (score on a scale)
Change in Weight (Z-score) From Baseline to Week 12
Z-score (score on a scale)Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Weight (Z-score) From Baseline to Week 12-0.01 ± 0.4060.05 ± 0.430-0.03 ± 0.4380.55 ± 0.1590.11 ± 0.1470.22 ± 0.353
PrimaryChange in Body Mass Index (BMI) (Absolute) From Baseline to Week 12

Body Mass Index is a measure of weight adjusted for height.

Time frame:
Baseline, Week 12
Reported as:
Mean · kg/m2
Change in Body Mass Index (BMI) (Absolute) From Baseline to Week 12
kg/m2Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Body Mass Index (BMI) (Absolute) From Baseline to Week 12-0.09 ± 0.933-0.04 ± 0.6650.05 ± 1.3941.53 ± 1.3850.82 ± 0.7840.60 ± 1.471
PrimaryChange in Body Mass Index (BMI) (Percentile) From Baseline to Week 12

Body Mass Index is a measure of weight adjusted for height.

Time frame:
Baseline, Week 12
Reported as:
Mean · percentile
Change in Body Mass Index (BMI) (Percentile) From Baseline to Week 12
percentileTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Body Mass Index (BMI) (Percentile) From Baseline to Week 12-2.85 ± 24.623-1.61 ± 12.509-3.00 ± 15.78110.78 ± 7.3126.22 ± 6.8494.44 ± 11.498
PrimaryChange in Body Mass Index (BMI) (Z-score) From Baseline to Week 12

Body Mass Index is a measure of weight adjusted for height. The z-score (standard score) is the number of standard deviations by which the outcome measure is above or below the value in the general population. A Z-score of 0 represents the population mean. A measure above the normal value has a positive z-score (higher BMI).

Time frame:
Baseline, Week 12
Reported as:
Mean · Z-score (score on a scale)
Change in Body Mass Index (BMI) (Z-score) From Baseline to Week 12
Z-score (score on a scale)Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Body Mass Index (BMI) (Z-score) From Baseline to Week 12-0.05 ± 0.764-0.02 ± 0.4950.27 ± 0.7711.10 ± 0.5840.29 ± 0.3170.29 ± 0.483
PrimaryChange in Diastolic Blood Pressure

Change from Baseline to Week 12 in diastolic sitting blood pressure.

Time frame:
Day 1, Week 2, Week 6, Week 12, Week 16
Reported as:
Mean · mmHg
Change in Diastolic Blood Pressure
mmHgTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Diastolic Blood Pressure-0.44 ± 8.2482.00 ± 8.179-3.17 ± 7.3335.80 ± 6.9791.50 ± 6.950-2.40 ± 11.915
PrimaryChange in Systolic Blood Pressure

Change from Baseline to Week 12 in systolic sitting blood pressure.

Time frame:
Day 1, Week 2, Week 6, Week 12, Week 16
Reported as:
Mean · mmHg
Change in Systolic Blood Pressure
mmHgTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change in Systolic Blood Pressure-2.33 ± 12.923-2.20 ± 11.233-2.50 ± 6.5045.00 ± 13.2662.50 ± 5.9580.07 ± 11.591
PrimaryNumber of Participants With Treatment Emergent Cushingoid Features

Treatment emergent cushingoid features based on physical examination at all baseline, on-treatment and post-treatment assessments

Time frame:
Baseline through Week 16
Reported as:
Count of participants · Participants
Number of Participants With Treatment Emergent Cushingoid Features
ParticipantsTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Number of Participants With Treatment Emergent Cushingoid Features000100
PrimaryNumber of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result

Each subject had blood drawn and urine collected for the standard hematology, chemistry and lipids clinical laboratory tests. In addition, fasting glucose and insulin, morning cortisol, as well as, in the additional 12 to \<18 years age group, LH, FSH, TSH, FT4 were collected. HbA1c determination had also to be performed if urine glucose was positive and/or fasted glucose levels was above normal limits. Any treatment-emergent clinically significant abnormal laboratory test result was reporte

Time frame:
Day 1, Week 6, Week 12, Week 16
Reported as:
Count of participants · Participants
Number of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result
ParticipantsTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Cortisol decreased012002
Blood glucose decreased000001
Blood insulin increased000001
Blood TSH increased000001
Tyroxin free increased000001
PrimaryCategorical Analysis of QTcF at Week 12

12-lead 1electrocardiogram (ECG) as recorded after subject has rested quietly in a supine position for at least 5 minutes. ECG components are QRS duration, PR \[PQ\] interval, RR interval, QT interval and QTc.

Time frame:
Baseline, Week 12
Reported as:
Count of participants · Participants
Categorical Analysis of QTcF at Week 12
ParticipantsTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
=< 450 msec101066515
> 450 msec000000
PrimaryNumber of Eyes With Cataract

Cataract was diagnosed by the presence of partial or complete opacity of the crystalline lens at Baseline and Week 12.

Time frame:
Baseline - Week 12
Reported as:
Number · eyes
Number of Eyes With Cataract
eyesTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
No Cataract : Baseline18191212817
No Cataract : Week 121881210815
Cataract : Baseline0000415
Cataract : Week 120000213
PrimaryNumber of Eyes With Glaucoma

Glaucoma was diagnosed by ocular pressure at Baseline and Week 12.

Time frame:
Baseline - Week 12
Reported as:
Number · eyes
Number of Eyes With Glaucoma
eyesTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
No Glaucoma: Baseline18191212817
No Glaucoma: Week 121881210815
Suspected Glaucoma: Baseline000002
Suspected Glaucoma: Week 12000000
Glaucoma: Baseline000000
Glaucoma: Week 12000000
SecondaryPre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2

The plasma concentration of vamorolone was measured on Day 1 and Week 2 predose, and 1h, 2h and 6h postdose and also 4h and 8h post in the 7-18 year groups.

Time frame:
Day 1, Week 2
Reported as:
Mean · ng / mL
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2
ng / mLTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Day 1 - predose0.0 ± 0.000.0 ± 0.000.0 ± 0.000.0 ± 0.000.0 ± 0.000.0 ± 0.00
Day 1 - 1h198.3 ± 196.67471.3 ± 333.61142.5 ± 225.74519.1 ± 437.26142.5 ± 225.74519.1 ± 437.26
Day 1 - 2h236.7 ± 153.58769.3 ± 527.67216.4 ± 181.19780.5 ± 457.91216.4 ± 181.19780.5 ± 457.91
Day 1 - 4h——163.8 ± 103.74711.1 ± 353.60163.8 ± 103.74711.1 ± 353.60
Day 1 - 6h125.7 ± 84.83280.0 ± 249.99148.0 ± 109.33389.1 ± 307.57148.0 ± 109.33389.1 ± 307.57
Day 1 - 8h——78.5 ± 41.66170.6 ± 183.6778.5 ± 41.66170.6 ± 183.67
Week 2 - predose0.0 ± 0.000.0 ± 0.000.7 ± 2.062.9 ± 5.220.7 ± 2.062.9 ± 5.22
Week 2 - 1h346.0 ± 329.08409.8 ± 385.65192.0 ± 199.92463.7 ± 348.19192.0 ± 199.92463.7 ± 348.19
Week 2 - 2h360.4 ± 222.93829.4 ± 740.83217.8 ± 149.31686.3 ± 369.69217.8 ± 149.31686.3 ± 369.69
Week 2 - 4h——207.6 ± 167.99603.0 ± 346.88207.6 ± 167.99603.0 ± 346.88
Week 2 - 6h134.5 ± 110.99272.0 ± 109.71158.9 ± 105.34300.2 ± 206.18158.9 ± 105.34300.2 ± 206.18
Week 2 - 8h——55.8 ± 31.81174.7 ± 137.7855.8 ± 31.81174.7 ± 137.78
SecondaryDescriptive Statistics of PK Parameters - Tmax

Tmax is the time to reach the maximum observed concentration collected during a dosing interval

Time frame:
Day 1, Week 2
Reported as:
Median · h
Descriptive Statistics of PK Parameters - Tmax
hGroup 1Group 2Group 3Group 4
Day 12.0 (1.0 to 6.0)2.0 (1.0 to 6.0)2.0 (1.0 to 6.0)2.0 (1.0 to 6.0)
Week 21.9 (1.0 to 6.3)2.0 (1.1 to 6.0)2.9 (1.0 to 8.0)2.0 (1.0 to 6.0)
SecondaryDescriptive Statistics of PK Parameters - Cmax

Cmax is the maximum observed concentration

Time frame:
Day 1, Week 2
Reported as:
Geometric mean · ng/mL
Descriptive Statistics of PK Parameters - Cmax
ng/mLGroup 1Group 2Group 3Group 4
Day 1246.8 ± 2.01781.7 ± 1.77254.5 ± 2.04922.2 ± 1.60
Week 2349.3 ± 2.06719.5 ± 2.23334.5 ± 1.67765.5 ± 1.59
SecondaryDescriptive Statistics of PK Parameters in Subjects Aged 2 to 4 Years - AUC 0-6

AUC 0-6 is the area under the concentration-time curve during the first 6 hours after dosing

Time frame:
Day 1, Week 2
Reported as:
Geometric mean · ng.h/mL
Descriptive Statistics of PK Parameters in Subjects Aged 2 to 4 Years - AUC 0-6
ng.h/mLGroup 1Group 2
Day 1816.3 ± 1.722216.2 ± 1.47
Week 21448.4 ± 1.382571.6 ± 2.02
SecondaryDescriptive Statistics of PK Parameters in Subjects Aged 7 to 18 Years - AUC 0-inf

AUC 0-8 is the area under the concentration-time curve after dosing extrapolated to infinity

Time frame:
Day 1, Week 2
Reported as:
Geometric mean · ng.h/mL
Descriptive Statistics of PK Parameters in Subjects Aged 7 to 18 Years - AUC 0-inf
ng.h/mLGroup 3Group 4
Day 11253.0 ± 1.654119.8 ± 1.53
Week 21528.6 ± 1.153587.7 ± 1.72
Other pre-specifiedChange From Baseline to Week 12 in Bayley-III Gross Motor Scale (Ages 2 to <4 Years Only)

The Bayley-III Gross Motor scale is a functional assessment, an accurate reflection of muscle strength for subjects with DMD ages 2 to \<4 years. The minimum score value is 0 and the maximum score value is 72. Higher scores mean a better outcome.

Time frame:
Baseline, Week 12
Reported as:
Mean · score on a scale
Change From Baseline to Week 12 in Bayley-III Gross Motor Scale (Ages 2 to <4 Years Only)
score on a scaleTreatment Group 1Treatment Group 2
Change From Baseline to Week 12 in Bayley-III Gross Motor Scale (Ages 2 to <4 Years Only)0.44 ± 1.1302.50 ± 1.716
Other pre-specifiedChange From Baseline to Week 12 in Morning Cortisol Concentration

Morning cortisol \[adrenal suppression\] samples were collected after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication at Day 1 and Week 12 Visits, before 10 AM local time

Time frame:
Baseline, Week 12
Reported as:
Mean · nmol/L
Change From Baseline to Week 12 in Morning Cortisol Concentration
nmol/LTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change From Baseline to Week 12 in Morning Cortisol Concentration-184.700 ± 138.2020-217.750 ± 103.9832-110.667 ± 85.8596-248.667 ± 186.906012.000 ± 49.3356-34.933 ± 42.1485
Other pre-specifiedChange From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Type 1 Collagen C-telopeptide [CTX1])

Samples for CTX1, osteocalcin and P1NP were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication

Time frame:
Baseline, Week 12
Reported as:
Mean · ng/L
Change From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Type 1 Collagen C-telopeptide [CTX1])
ng/LTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Type 1 Collagen C-telopeptide [CTX1])28.300 ± 387.1423138.000 ± 257.664093.500 ± 235.7446101.800 ± 365.3542631.833 ± 155.3621273.000 ± 335.4937
Other pre-specifiedChange From Baseline to Week 12 in Bone Turnover Biomarkers (Osteocalcin)

Samples for CTX1, osteocalcin and P1NP were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication

Time frame:
Baseline, Week 12
Reported as:
Mean · ug/L
Change From Baseline to Week 12 in Bone Turnover Biomarkers (Osteocalcin)
ug/LTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change From Baseline to Week 12 in Bone Turnover Biomarkers (Osteocalcin)-3.340 ± 21.05502.422 ± 10.696014.817 ± 18.7328-1.120 ± 15.852831.700 ± 11.691516.546 ± 9.1486
Other pre-specifiedChange From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Aminoterminal Propeptide of Type I Collagen [P1NP] )

Samples for CTX1, osteocalcin and P1NP were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication

Time frame:
Baseline, Week 12
Reported as:
Mean · ug/L
Change From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Aminoterminal Propeptide of Type I Collagen [P1NP] )
ug/LTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Aminoterminal Propeptide of Type I Collagen [P1NP] )-39.870 ± 188.778714.478 ± 117.570419.067 ± 78.6139-82.840 ± 178.0726318.850 ± 99.0038149.671 ± 106.2874
Other pre-specifiedChange From Baseline to Week 12 in Insulin Resistance Biomarkers (Glucose)

Glucose, HbA1c and insulin were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication.

Time frame:
Baseline, Week 12
Reported as:
Mean · mmol/L
Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Glucose)
mmol/LTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Glucose)-0.160 ± 0.2912-0.243 ± 0.6309-0.063 ± 0.3524-0.335 ± 0.38150.195 ± 0.5727-0.254 ± 0.3909
Other pre-specifiedChange From Baseline to Week 12 in Insulin Resistance Biomarkers (Hemoglobin A1c [HbA1c])

Glucose, HbA1c and insulin were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication. The Baseline sample for HbA1c measurement may have been collected non-fasting.

Time frame:
Baseline, Week 12
Reported as:
Mean · percent
Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Hemoglobin A1c [HbA1c])
percentTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Hemoglobin A1c [HbA1c])0.1 ± 0.260.0 ± 0.150.0 ± 0.160.0 ± 0.100.0 ± 0.11-0.1 ± 0.12
Other pre-specifiedChange From Baseline to Week 12 in Insulin Resistance Biomarkers (Insulin)

Glucose, HbA1c and insulin were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication.

Time frame:
Baseline, Week 12
Reported as:
Mean · pmol/L
Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Insulin)
pmol/LTreatment Group 2Treatment Group 3Treatment Group 4Treatment Group 6
Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Insulin)6.333 ± 20.51830.667 ± 33.005146.333 ± 51.9256-26.333 ± 51.1493

Adverse events

Collected over AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment Group 10/10 (0%)0/10 (0%)7/10 (70%)
Treatment Group 20/10 (0%)0/10 (0%)9/10 (90%)
Treatment Group 30/6 (0%)0/6 (0%)6/6 (100%)
Treatment Group 40/6 (0%)1/6 (16.7%)4/6 (66.7%)
Treatment Group 50/6 (0%)0/6 (0%)3/6 (50%)
Treatment Group 60/16 (0%)1/16 (6.3%)11/16 (68.8%)
Most frequent serious events
Most frequent serious events
EventTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
VomitingGastrointestinal disorders0/100/100/61/60/60/16
RhabdomyolysisMusculoskeletal and connective tissue disorders0/100/100/60/60/61/16
Most frequent other events
Showing 10 of 54
Most frequent other events
EventTreatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6
Adrenal suppressionEndocrine disorders0/105/100/61/60/61/16
NasopharyngitisInfections and infestations4/103/102/60/61/62/16
AstheniaGeneral disorders0/100/100/60/62/60/16
Cortisol decreasedInvestigations0/101/102/60/60/62/16
Increased appetiteMetabolism and nutrition disorders0/100/100/62/60/60/16
Pain in extremityMusculoskeletal and connective tissue disorders0/102/100/60/62/60/16
DiarrhoeaGastrointestinal disorders0/103/100/60/60/60/16
HeadacheNervous system disorders0/101/101/60/60/64/16
Abdominal pain upperGastrointestinal disorders0/102/100/60/60/61/16
ToothacheGastrointestinal disorders0/102/100/60/60/60/16

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6Total
<=18 years10106661654
Between 18 and 65 years0000000
>=65 years0000000
Sex: Female, Male
Sex: Female, Male(Participants)Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6Total
Female0000000
Male10106661654
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6Total
Hispanic or Latino0001124
Not Hispanic or Latino10106341447
Unknown or Not Reported0002103
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6Total
American Indian or Alaska Native0000000
Asian51122314
Native Hawaiian or Other Pacific Islander0000000
Black or African American0100001
White585331337
More than one race0001001
Unknown or Not Reported0000101
Region of Enrollment
Region of Enrollment(participants)Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6Total
Canada10106661654
Age at first symptom
Age at first symptom(months)Treatment Group 1Treatment Group 2Treatment Group 3Treatment Group 4Treatment Group 5Treatment Group 6Total
Mean20.8 ± 5.6714.2 ± 4.4730.0 ± 15.1851.0 ± 23.9232.2 ± 14.2642.8 ± 27.5931.7 ± 21.88
08

Study locations

5 sites
  • Alberta's Children Hospital
    Calgary, Alberta AB T3B 6A8, Canada
  • British Columbia Children's Hospital
    Vancouver, British Columbia V6H 3N1, Canada
  • Children's Hospital of Eastern Ontario
    Ottawa, Ontario K1H 8L1, Canada
  • The Hospital for Sick Children
    Toronto, Ontario M5G 1X8, Canada
  • Montreal Childrens Hospital
    Montreal, H4A 3J1, Canada
09

References and documents

Publications

  • Hoffman EP, Schwartz BD, Mengle-Gaw LJ, Smith EC, Castro D, Mah JK, McDonald CM, Kuntz NL, Finkel RS, Guglieri M, Bushby K, Tulinius M, Nevo Y, Ryan MM, Webster R, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Siener C, Jaros M, Shale P, McCall JM, Nagaraju K, van den Anker J, Conklin LS, Cnaan A, Gordish-Dressman H, Damsker JM, Clemens PR; Cooperative International Neuromuscular Research Group. Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology. 2019 Sep 24;93(13):e1312-e1323. doi: 10.1212/WNL.0000000000008168. Epub 2019 Aug 26. PubMed 31451516 ↗
  • Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM; CINRG VBP15 and DNHS Investigators. Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. eCollection 2020 Sep. PubMed 32956407 ↗
  • Heier CR, Yu Q, Fiorillo AA, Tully CB, Tucker A, Mazala DA, Uaesoontrachoon K, Srinivassane S, Damsker JM, Hoffman EP, Nagaraju K, Spurney CF. Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Sci Alliance. 2019 Feb 11;2(1):e201800186. doi: 10.26508/lsa.201800186. Print 2019 Feb. PubMed 30745312 ↗
  • Mavroudis PD, van den Anker J, Conklin LS, Damsker JM, Hoffman EP, Nagaraju K, Clemens PR, Jusko WJ. Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy. J Clin Pharmacol. 2019 Jul;59(7):979-988. doi: 10.1002/jcph.1388. Epub 2019 Feb 11. PubMed 30742306 ↗
  • Damsker JM, Cornish MR, Kanneboyina P, Kanneboyina I, Yu Q, Lipson R, Phadke A, Knoblach SM, Panchapakesan K, Morales M, Fiorillo AA, Partridge T, Nagaraju K. Vamorolone, a dissociative steroidal compound, reduces collagen antibody-induced joint damage and inflammation when administered after disease onset. Inflamm Res. 2019 Nov;68(11):969-980. doi: 10.1007/s00011-019-01279-z. Epub 2019 Aug 24. PubMed 31446438 ↗
  • Akkad H, Cacciani N, Llano-Diez M, Corpeno Kalamgi R, Tchkonia T, Kirkland JL, Larsson L. Vamorolone treatment improves skeletal muscle outcome in a critical illness myopathy rat model. Acta Physiol (Oxf). 2019 Feb;225(2):e13172. doi: 10.1111/apha.13172. Epub 2018 Sep 6. PubMed 30120816 ↗
  • Li X, Conklin LS, van den Anker J, Hoffman EP, Clemens PR, Jusko WJ. Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy. J Clin Pharmacol. 2020 Oct;60(10):1385-1396. doi: 10.1002/jcph.1632. Epub 2020 May 20. PubMed 32434278 ↗
  • Hoffman EP, Riddle V, Siegler MA, Dickerson D, Backonja M, Kramer WG, Nagaraju K, Gordish-Dressman H, Damsker JM, McCall JM. Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes. Steroids. 2018 Jun;134:43-52. doi: 10.1016/j.steroids.2018.02.010. Epub 2018 Mar 8. PubMed 29524454 ↗
  • Almeida LEF, Damsker JM, Albani S, Afsar N, Kamimura S, Pratt D, Kleiner DE, Quezado M, Gordish-Dressman H, Quezado ZMN. The corticosteroid compounds prednisolone and vamorolone do not alter the nociception phenotype and exacerbate liver injury in sickle cell mice. Sci Rep. 2018 Apr 17;8(1):6081. doi: 10.1038/s41598-018-24274-6. PubMed 29666400 ↗
  • Wells E, Kambhampati M, Damsker JM, Gordish-Dressman H, Yadavilli S, Becher OJ, Gittens J, Stampar M, Packer RJ, Nazarian J. Vamorolone, a dissociative steroidal compound, reduces pro-inflammatory cytokine expression in glioma cells and increases activity and survival in a murine model of cortical tumor. Oncotarget. 2017 Feb 7;8(6):9366-9374. doi: 10.18632/oncotarget.14070. PubMed 28030841 ↗
  • Conklin LS, Damsker JM, Hoffman EP, Jusko WJ, Mavroudis PD, Schwartz BD, Mengle-Gaw LJ, Smith EC, Mah JK, Guglieri M, Nevo Y, Kuntz N, McDonald CM, Tulinius M, Ryan MM, Webster R, Castro D, Finkel RS, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, McCall JM, Hathout Y, Nagaraju K, van den Anker J, Ward LM, Ahmet A, Cornish MR, Clemens PR. Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug. Pharmacol Res. 2018 Oct;136:140-150. doi: 10.1016/j.phrs.2018.09.007. Epub 2018 Sep 13. PubMed 30219580 ↗
  • Fiorillo AA, Tully CB, Damsker JM, Nagaraju K, Hoffman EP, Heier CR. Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone. Physiol Genomics. 2018 Sep 1;50(9):735-745. doi: 10.1152/physiolgenomics.00134.2017. Epub 2018 Jun 8. PubMed 29883261 ↗
  • Sreetama SC, Chandra G, Van der Meulen JH, Ahmad MM, Suzuki P, Bhuvanendran S, Nagaraju K, Hoffman EP, Jaiswal JK. Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to Dysferlin Deficit. Mol Ther. 2018 Sep 5;26(9):2231-2242. doi: 10.1016/j.ymthe.2018.07.021. Epub 2018 Aug 27. PubMed 30166241 ↗
  • Dang UJ, Ziemba M, Clemens PR, Hathout Y, Conklin LS; CINRG Vamorolone 002/003 Investigators; Hoffman EP. Serum biomarkers associated with baseline clinical severity in young steroid-naive Duchenne muscular dystrophy boys. Hum Mol Genet. 2020 Aug 29;29(15):2481-2495. doi: 10.1093/hmg/ddaa132. PubMed 32592467 ↗
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  • Garvin LM, Chen Y, Damsker JM, Rose MC. A novel dissociative steroid VBP15 reduces MUC5AC gene expression in airway epithelial cells but lacks the GRE mediated transcriptional properties of dexamethasone. Pulm Pharmacol Ther. 2016 Jun;38:17-26. doi: 10.1016/j.pupt.2016.04.004. Epub 2016 Apr 29. PubMed 27133900 ↗
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  • Mah JK, Gonorazky HD, Nigro E, Lochmuller H, Aleman A, Yaworski A, Oskoui M, Sbrocchi AM, Selby KA, de Vera A, Mathur R, Gresko E, Linden A, Dutreix C, Hoffman EP. Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys. Neurology. 2026 Jun 9;106(11):e218066. doi: 10.1212/WNL.0000000000218066. Epub 2026 May 15. PubMed 42139656 ↗
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Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Mar 2022 to primary completion in Jul 2024, 27 months.

Results were first posted in Oct 2025, 1 year 3 months after primary completion.

Of 40 industry-sponsored phase 2 interventional studies under Muscular Dystrophy, Duchenne, half reached primary completion within 31 months of starting (the median), counting those still running; a quarter within 20 and three quarters within 42. This study's 27 months is below that.

Counting every sponsor, half of 47 studies reached it within 31 months; a quarter within 20 and three quarters within 42.

How these figures are worked out, and which studies are left out of them.

Registered
Nov 9, 2021
First posted
Jan 11, 2022
StartActual
Mar 21, 2022
Primary completionActual
Jul 16, 2024
CompletionActual
Jul 16, 2024
Results first posted
Oct 24, 2025
Last update
Oct 24, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Oct 24, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT05185622
Lead sponsor
Santhera Pharmaceuticals
Responsible party
Sponsor
First posted
Jan 11, 2022
Start date
Mar 21, 2022
Primary completion
Jul 16, 2024
Completion
Jul 16, 2024
Results posted
Oct 24, 2025
Last update
Oct 24, 2025

Study contacts

Jean K Mah, M.D.
principal investigator · Alberta Children's Hospital Research Institute, University of Calgary

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.

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Discussion

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Studies of the drug tested here: Vamorolone

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