Duchenne Muscular DystrophyPhase 2NCT03038399
Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
A Phase 2 study testing Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day and Vamorolone 2.0 mg/day/day in people with Duchenne Muscular Dystrophy, run by ReveraGen BioPharma, Inc. at 12 sites in 6 countries.
Trial summary
This long-term extension study is an open-label, multiple-dose study to evaluate the long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who participated in the VBP15-002 Phase IIa and VBP15-003… Read more about this study
- Who is it for?
- Ages 4 to 7 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day and Vamorolone 2.0 mg/day/day
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 12 sitesin 6 countries
- Study dates
- 2 Feb 2017to 30 Apr 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 4 to 7 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Subject's parent or legal guardian has provided written informed consent and HIPAA authorization (if applicable) prior to any VBP15-LTE long-term extension study-specific procedures;
- Subject has previously completed study VBP15-003 up to and including the Week 24 Final assessments, prior to enrolling in the VBP15-LTE study at the conclusion of the VBP15-003 Week 24 Visit [Note: if entering the dose-tapering period, subject is enrolling within 8 weeks after the VBP15-003 final visit following dose-tapering]; and
- Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
Who cannot join
- Subject had a serious or severe adverse event in study VBP15-003 that, in the opinion of the Investigator, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this long-term extension study;
- Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
- Subject has current or history of chronic systemic fungal or viral infections;
- Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication;
- Subject has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
- Subject is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents [Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical glucocorticoids prescribed for an indication other than DMD are permitted but must be administered at stable dose for at least 3 months prior to study drug administration];
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- Subject has used idebenone within 4 weeks prior to the first dose of study medication;
- Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
- Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
- 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
- Subject is currently taking any investigational drug, or has taken any investigational drug other than vamorolone within 3 months prior to the start of study treatment.
- Note: Subjects may be re-evaluated if ineligible due to a transient condition which would prevent the subject from participating.
Trial locations and contacts
12 sites in 6 countries.
12 sites in 6 countries
United States6 sites
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611
University of Texas Southwestern Medical Center
Dallas, Texas, 75207
University of California Davis
Davis, California, 95616
Duke University
Durham, North Carolina, 27710
University of Florida
Gainesville, Florida, 32611
Nemours Children's Hospital
Orlando, Florida, 32827
Australia2 sites
Royal Children's Hospital
Melbourne
Sydney Children's Hospital
Westmead
United Kingdom1 site
Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE7 7DN
About this study
Official title: A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
This long-term extension study is an open-label, multiple-dose study to evaluate the long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who participated in the VBP15-002 Phase IIa and VBP15-003 Phase IIa extension core studies.
Read the detailed description
This study will evaluate if it is safe to use a new medication called vamorolone for more than two weeks in children with DMD, if boys with DMD who take the study medication have improved muscle function compared to boys with DMD in other studies who did not take any type of steroid, and to see if boys with DMD who take the study medication gain less weight compared to boys with DMD in a prior study who took another type of steroid called prednisone. Enrolled participants will take the study medication for 24 months.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day, Vamorolone 6.0 mg/day/day
- Keywords
- Duchenne muscular dystrophy, vamorolone
- 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.
Vamorolone 0.25 mg/day/dayDrug
Also called VBP15
Oral administration of 0.25 mg/kg/day daily for 24 months.
Vamorolone 0.75 mg/day/dayDrug
Also called VBP15
Oral administration of 0.75 mg/kg/day daily for 24 months.
Vamorolone 2.0 mg/day/dayDrug
Also called VBP15
Oral administration of 2.0 mg/kg/day daily for 24 months.
Vamorolone 6.0 mg/day/dayDrug
Also called VBP15
Oral administration of 6.0 mg/kg/day daily for 24 months.
| Study group | Role | What they receive |
|---|---|---|
| Dose Level Group 1 | Experimental | Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Receives: Drug: Vamorolone 0.25 mg/day/day |
| Dose Level Group 2 | Experimental | Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Receives: Drug: Vamorolone 0.75 mg/day/day |
| Dose Level Group 3 | Experimental | Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Receives: Drug: Vamorolone 2.0 mg/day/day |
| Dose Level Group 4 | Experimental | Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Receives: Drug: Vamorolone 6.0 mg/day/day |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03
To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);
Measured: 24 months
Total Number of Adverse Events as Assessed by CTCAE Version 4.03
To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).
Measured: 24 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2017 (estimated)
- Study start2 Feb 2017
- Primary completion30 Apr 2020
- Study completion30 Apr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- ReveraGen BioPharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of Pittsburgh, Cooperative International Neuromuscular Research Group
- Responsible party
- Sponsor
- Investigators
- Paula R Clemens, MD, Study chair, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03038399
- Sponsor's protocol ID
- VBP15-LTE
- Status last verified
- Apr 2021
- First submitted
- 30 Jan 2017
- Last updated
- 20 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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 32956407Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178.
PubMed 35076703
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT03038399
- Acronym / sponsor ID
- VBP15-LTE
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, vamorolone
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- ReveraGen BioPharma, Inc.Industry (pharma, biotech, devices)
- Collaborators
- University of Pittsburgh, Cooperative International Neuromuscular Research Group
- Study start
- 2 Feb 2017 (actual)
- Primary completion
- 30 Apr 2020 (actual)
- Study completion
- 30 Apr 2020 (actual)
- First posted
- 31 Jan 2017 (estimated)
- Results
- Posted 20 May 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This long-term extension study is an open-label, multiple-dose study to evaluate the long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who participated in the VBP15-002 Phase IIa and VBP15-003 Phase IIa extension core studies.
Detailed description
This study will evaluate if it is safe to use a new medication called vamorolone for more than two weeks in children with DMD, if boys with DMD who take the study medication have improved muscle function compared to boys with DMD in other studies who did not take any type of steroid, and to see if boys with DMD who take the study medication gain less weight compared to boys with DMD in a prior study who took another type of steroid called prednisone. Enrolled participants will take the study medication for 24 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Dose Level Group 1 | Experimental | Drug: Vamorolone 0.25 mg/day/dayParticipants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. |
| Dose Level Group 2 | Experimental | Drug: Vamorolone 0.75 mg/day/dayParticipants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. |
| Dose Level Group 3 | Experimental | Drug: Vamorolone 2.0 mg/day/dayParticipants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. |
| Dose Level Group 4 | Experimental | Drug: Vamorolone 6.0 mg/day/dayParticipants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. |
Who may qualify
- Ages
- 4 to 7 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Subject's parent or legal guardian has provided written informed consent and HIPAA authorization (if applicable) prior to any VBP15-LTE long-term extension study-specific procedures;
- Subject has previously completed study VBP15-003 up to and including the Week 24 Final assessments, prior to enrolling in the VBP15-LTE study at the conclusion of the VBP15-003 Week 24 Visit [Note: if entering the dose-tapering period, subject is enrolling within 8 weeks after the VBP15-003 final visit following dose-tapering]; and
- Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
Key exclusion criteria (12 listed)
- Subject had a serious or severe adverse event in study VBP15-003 that, in the opinion of the Investigator, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this long-term extension study;
- Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
- Subject has current or history of chronic systemic fungal or viral infections;
- Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication;
- Subject has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
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 |
|---|---|---|
| Vamorolone 0.25 mg/day/dayAlso: VBP15Oral administration of 0.25 mg/kg/day daily for 24 months. | Drug | Dose Level Group 1 |
| Vamorolone 0.75 mg/day/dayAlso: VBP15Oral administration of 0.75 mg/kg/day daily for 24 months. | Drug | Dose Level Group 2 |
| Vamorolone 2.0 mg/day/dayAlso: VBP15Oral administration of 2.0 mg/kg/day daily for 24 months. | Drug | Dose Level Group 3 |
| Vamorolone 6.0 mg/day/dayAlso: VBP15Oral administration of 6.0 mg/kg/day daily for 24 months. | Drug | Dose Level Group 4 |
Primary outcome
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03
Time frame: 24 months
To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);
Total Number of Adverse Events as Assessed by CTCAE Version 4.03
Time frame: 24 Months
To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 32956407Smith 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.
- Derived from this study: PubMed 35076703Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178.
- Link to publicationLink 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.
Paula R Clemens, MD study chair
University of Pittsburgh
12 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day and Vamorolone 2.0 mg/day/day in people with Duchenne Muscular Dystrophy, run by ReveraGen BioPharma, Inc. at 12 sites in 6 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 12 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: finished, as of the sponsor's last update on 20 May 2021. 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, Australia, Canada, Israel, Sweden and 1 more country. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03 (time frame: 24 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03038399, 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 May 2021.