Duchenne Muscular DystrophyNCT03863119
Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
A study testing Vamorolone in people with Duchenne Muscular Dystrophy, run by Santhera Pharmaceuticals at 12 sites in 3 countries.
Trial summary
The intent of this protocol is to provide continued access to vamorolone for subjects in the United States and Canada who have completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone… Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Vamorolone
- How many people
- Not given
- Where
- 12 sitesin 3 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- Child, Adult, Older adult
Who may be able to join
- Subject's parent or legal guardian has provided written informed consent/HIPAA authorization
- Subject has previously completed at a participating US or Canada study site VBP15-LTE up to and including the Month 24 assessments, OR VBP15-004 up to and including the Week 48 assessments, VBP15-006 up to and including the Week 12 assessment
- Subject and parent/guardian are willing and able to comply with recommended study drug administration plan, and standard of care follow-up and monitoring as recommended by their Treating Physician
Who cannot join
- Subject had a serious or severe adverse event in study VBP15-LTE or VBP15-004 or VBP15-006 that, in the opinion of the Treating Physician and Sponsor, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this expanded access program
- Subject and/or parent/guardian are unable and/or unwilling to comply with regular medical care and follow-up as recommended by their Treating Physician throughout participation in the VBP15-EAP
Trial locations and contacts
12 sites in 3 countries.
12 sites in 3 countries
United States6 sites
- Available
urie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nancy L. Kuntz, MD
- Available
University of Texas Southwestern Medical Center
Dallas, Texas, 75207
Kaitlin Batley, MD
- Available
University of California Davis
Davis, California, 95616
Craig McDonald, MD
- Available
Duke University
Durham, North Carolina, 27710
Mai K. ElMallah, M.D.
- Available
Nemours Children's Hospital
Orlando, Florida, 32827
Omer Abdul Hamid, MD
- Available
Seattle Children's
Seattle, Washington, 98105
Seth J. Perlman, MD
Canada5 sites
- Available
Alberta's Children Hospital
Calgary, Alberta, T3B 6A8
Julie Dao, 4059552296
Jean Mah, MD
- Available
Montreal Childrens Hospital
Montreal, Quebec, H4A 3J1
Maryam Oskoui, maryam.oskoui@mcgill.ca
Maryam Oskoui, MD, M.Sc.
- Available
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
Laura Thompson, (613) 737-7600 ext. 6051, LaThompson@cheo.on.ca
Hanns Lochmuller, MD
- Available
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Ana Stosic, 416- 813-5668 ext. 224579, ana.stosic@sickkids.ca
Hernan Gonorazky, MD
- Available
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3N1
Kathryn Selby, 604-875-2345 ext. 2161, selby@cw.bc.ca
Nela Martic, NMartic3@cw.bc.ca
Israel1 site
- Available
Schneider Chidlren's Medical Center
Petah Tikva, 4920435
Yoram Nevo
About this study
Official title: An Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 or VBP15-006 Studies
The intent of this protocol is to provide continued access to vamorolone for subjects in the United States and Canada who have completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.
Read the detailed description
Vamorolone will be shipped to the subject's family by the study site. The patient will receive standard of care treatment and procedures for management of DMD. Treating Physicians participating in the expanded access program are required to collect and document any physician, patient, or caregiver reported safety events and report to the Sponsor. The subject's dose of vamorolone may be increased or decreased within a range of 2.0 to 6.0 mg/kg/day (only doses of 2 mg/kg, 4 mg/kg and 6 mg/kg are allowed), given once daily. Administration of vamorolone (taken with an 8 ounce (240 ml) glass of full fat milk, or equivalent high-fat food portion) will be unchanged from the VBP15-LTE, VBP15-004, or VBP15-006 studies. In the absence of safety concerns, and while this Expanded Access protocol is active, vamorolone may be provided indefinitely or until approval, provided that the Treating Physician and family agrees that continued administration of vamorolone is in the best interest of the child.
VBP15-EAP (under IND 118942) has been closed as vamaorolone is approved for marketing in the US VBP15-EAP CANADA has been approved by Health Canada on 11 July 2019 and is currently on-going,but not recruiting
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Vamorolone
- Keywords
- Duchenne Muscular Dystrophy, Vamorolone, VBP-15, DMD
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VamoroloneDrug
2.0 mg/kg/day, 4.0 mg/kg/day, or 6.0 mg/kg/day at physician discretion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03863119
- Sponsor's protocol ID
- VBP15-EAP
- Status last verified
- Jan 2026
- First submitted
- 21 Feb 2019
- Last updated
- 20 Jan 2026
- Results posted
- No
Publications
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 42139656Lochmuller H, Gonorazky H, Nigro E, Mah JK, Aleman A, Yaworski A, Oskoui M, Sbrocchi AM, Selby K, de Vera A, McAdam L, Gresko E, Linden A, Dutreix C, Hoffman EP. Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy. J Neurol. 2026 Mar 3;273(3):177. doi: 10.1007/s00415-026-13711-6.
PubMed 41774261
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 or VBP15-006 Studies
- ClinicalTrials.gov ID
- NCT03863119
- Acronym / sponsor ID
- VBP15-EAP
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Vamorolone, VBP-15, DMD
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 5 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The intent of this protocol is to provide continued access to vamorolone for subjects in the United States and Canada who have completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.
Detailed description
Vamorolone will be shipped to the subject's family by the study site. The patient will receive standard of care treatment and procedures for management of DMD. Treating Physicians participating in the expanded access program are required to collect and document any physician, patient, or caregiver reported safety events and report to the Sponsor. The subject's dose of vamorolone may be increased or decreased within a range of 2.0 to 6.0 mg/kg/day (only doses of 2 mg/kg, 4 mg/kg and 6 mg/kg are allowed), given once daily. Administration of vamorolone (taken with an 8 ounce (240 ml) glass of full fat milk, or equivalent high-fat food portion) will be unchanged from the VBP15-LTE, VBP15-004, or VBP15-006 studies. In the absence of safety concerns, and while this Expanded Access protocol is active, vamorolone may be provided indefinitely or until approval, provided that the Treating Physician and family agrees that continued administration of vamorolone is in the best interest of the child.
VBP15-EAP (under IND 118942) has been closed as vamaorolone is approved for marketing in the US VBP15-EAP CANADA has been approved by Health Canada on 11 July 2019 and is currently on-going,but not recruiting
Study design
Who may qualify
- Sex
- Male only
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Subject's parent or legal guardian has provided written informed consent/HIPAA authorization
- Subject has previously completed at a participating US or Canada study site VBP15-LTE up to and including the Month 24 assessments, OR VBP15-004 up to and including the Week 48 assessments, VBP15-006 up to and including the Week 12 assessment
- Subject and parent/guardian are willing and able to comply with recommended study drug administration plan, and standard of care follow-up and monitoring as recommended by their Treating Physician
Key exclusion criteria (2 listed)
- Subject had a serious or severe adverse event in study VBP15-LTE or VBP15-004 or VBP15-006 that, in the opinion of the Treating Physician and Sponsor, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this expanded access program
- Subject and/or parent/guardian are unable and/or unwilling to comply with regular medical care and follow-up as recommended by their Treating Physician throughout participation in the VBP15-EAP
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vamorolone2.0 mg/kg/day, 4.0 mg/kg/day, or 6.0 mg/kg/day at physician discretion | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2026.
- Derived from this study: PubMed 42139656Mah 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.
- Derived from this study: PubMed 41774261Lochmuller H, Gonorazky H, Nigro E, Mah JK, Aleman A, Yaworski A, Oskoui M, Sbrocchi AM, Selby K, de Vera A, McAdam L, Gresko E, Linden A, Dutreix C, Hoffman EP. Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy. J Neurol. 2026 Mar 3;273(3):177. doi: 10.1007/s00415-026-13711-6.
- Vamorolone Phase IIa trial in Duchenne Muscular DystrophyLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
12 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Vamorolone in people with Duchenne Muscular Dystrophy, run by Santhera Pharmaceuticals at 12 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Canada, Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Medical director (+41 61 906 89 51, ekaterina.gresko@santhera.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03863119, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2026.