Muscular Dystrophy, DuchennePhase 2BRAVENCT03400852
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
A Phase 2 study testing MNK-1411 in people with Muscular Dystrophy, Duchenne, run by Mallinckrodt ARD LLC at 16 sites in 8 countries.
Trial summary
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD). Read more about this study
- Who is it for?
- Ages 4 to 8 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- What is being tested?
- MNK-1411
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 16 sitesin 8 countries
- Study dates
- 27 Jul 2018to 25 Feb 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 8 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Participants must have a documented diagnosis of Duchenne Muscular Dystrophy (DMD) confirmed by complete dystrophin deficiency (by immunofluorescence and/or immunoblot), or identifiable mutation in the DMD gene where reading frame can be predicated as "out of frame," or complete dystrophin gene sequencing consistent with DMD; AND in the opinion of the Investigator, a typical clinical profile consistent with DMD.
- Participants taking approved treatments for DMD (by a Health Authority) that target dystrophin gene mutations (e.g., eteplirsen or ataluren) may be enrolled in the study if they have been on a stable dose for 30 days prior to the first dose of study drug, and plan to remain on that dose throughout the study.
Who cannot join
- Participant has had previous systemic treatment with corticosteroids within 2 months prior to the Screening Visit. Exception: In subjects who were down-titrated to a physiological dose of corticosteroids (ie, 3mg/m2 of prednisone or deflazacort) a maximum of 1 month of no greater than a physiological dose followed by 1 month completely off corticosteroids prior to the Screening Visit will be acceptable for study entry. Transient previous use of corticosteroids will be evaluated on a case-by-case basis by the sponsor or designee. The use of topical or intra-articular corticosteroids is permitted during the study
- Participant is unable to complete the 10 meter Walk/Run test at the Screening and/or Baseline Visit.
- Participant has Type 1 or Type 2 diabetes mellitus.
- Participant has a history of chronic active hepatitis including acute or chronic hepatitis B, or acute or chronic hepatitis C.
- Participant has a history of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
- Participant has known immune compromised status (not related to disease/condition under study), including but not limited to, individuals who have undergone organ transplantation or who are known to be positive for the human immunodeficiency virus.
Trial locations and contacts
16 sites in 8 countries.
16 sites in 8 countries
United States5 sites
Rare Disease Research, LLC
Atlanta, Georgia, 30318
University of Texas Southwestern Medical Center
Dallas, Texas, 75207
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, 32561
Monroe Carell Jr Childrens Hospital at Vanderbilt
Nashville, Tennessee, 37232
UT Health Science Center, San Antonio
San Antonio, Texas, 78229
Mexico3 sites
Neurociencias Estudios Clinicos S.C.
Culiacán, Sinaloa, 80020
Instituto de Investigaciones Aplicadas a la Neurociencia A.C.
Durango, 34000
Hospital Civil Fray Antonio Alcalde
Guadalajara, Jalisco, 44280
Spain3 sites
Hospital de La Santa Creu i Sant Pau
Barcelona, 08025
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat, 08950
Hospital Universitari i Politecnic La Fe Valencia
Valencia, 46026
Bulgaria1 site
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, 1431
Serbia1 site
Clinic of Neurology and Psychiatry for Children and Youth
Belgrade, 11000
Turkey (Türkiye)1 site
Mersin Universitesi Tip Fakultesi Hastanesi
Mersin, 33343
About this study
Official title: A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Participants 4 to 8 Years of Age With Duchenne Muscular Dystrophy
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
Read the detailed description
The main purpose of this study is to determine the effect of MNK-1411 on motor function in participants with Duchenne Muscular Dystrophy (DMD). Information is collected only from caretakers who are fluent in English, using the Pediatric Outcomes Data Collection Instrument (PODCI).
The PODCI is a validated 86-question instrument completed by the parent or legal guardian of children 2 to 10 years of age to assess a variety of health outcome measures (Uzark et al, 2012). This study will only collect information for the PODCI domains of sports and physical functioning and transfer/basic mobility.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- MNK-1411, Placebo
- Keywords
- Muscular Dystrophy, DMD, Duchenne
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MNK-1411Drug
Also called Cosyntropin acetate, Tetracosactide Hexaacetate
MNK-1411 (1 mg/mL suspension) for subcutaneous injection
PlaceboOther
Also called Matching Placebo
Placebo suspension for subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Period 1: MNK-1411 | Experimental | Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1 Receives: Drug: MNK-1411 |
| Period 1: Placebo | Experimental | Participants receive placebo at a volume appropriate to body weight during Period 1 Receives: Other: Placebo |
| Period 2: MNK-1411 | Experimental | All participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2 Receives: Drug: MNK-1411 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Complete 10 Meter Walk/Run[
10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.
Measured: Baseline, Week 24
Secondary outcomes (7)
North Star Ambulatory Assessment (NSAA) Score
The NSAA is comprised of 17 items, each of which is graded using the standard scorecard. Each assessment is rated as 0 - unable to achieve independently, 1 - modified method but achieves goal independent of physical assistance from another, or 2 - normal with no obvious modification of activity. The subscale scores are summed for a total score ranging from 0 to 34. The higher the total score, the better the outcome.
Measured: Baseline, Week 24
Time to Climb 4 Standardized Stairs
Time to Climb 4 Standardized Stairs is a motor performance test
Measured: Baseline, Week 24
Time to Stand From a Supine Position
Time to stand from a supine position is a motor function test to measure the functional capability in subjects with DMD.
Measured: Baseline, Week 24
Quantitative Muscle Testing Scores at Baseline
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
Measured: Baseline
Quantitative Muscle Testing Scores at Week 24
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
Measured: Week 24
Summary of Adverse Events in the Blinded Treatment Period
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Measured: within 28 weeks
Summary of Adverse Events in the Open Label Period
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Measured: within 28 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2018
- Study start27 Jul 2018
- Primary completion25 Feb 2020
- Study completion25 Feb 2020
Why it stopped: Slow enrollment
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.
- Other trials for Muscular Dystrophy, DuchenneStudies 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- In the double-blind period, participants are randomized to receive study drug or placebo. It is followed by an open label period wherein all participants receive study drug.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mallinckrodt ARD LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Study Lead, Study director, Mallinckrodt
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03400852
- Sponsor's protocol ID
- MNK14112096
- Other IDs
- 2017-004139-35, 2017
- Acronym
- BRAVE
- Status last verified
- Feb 2021
- First submitted
- 9 Jan 2018
- Last updated
- 16 Mar 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Discussion of statistical endpoints and analysis are included in manuscripts. Summary aggregate (basic) results (including adverse events information) and the study protocol are made available on clinicaltrials.gov (NCT03400852) when required by regulation. Individual de-identified patient data will not be disclosed. Requests for additional information should be directed to the company at medinfo@mnk.com.
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Participants 4 to 8 Years of Age With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03400852
- Acronym / sponsor ID
- BRAVE / MNK14112096
- Conditions
- Muscular Dystrophy, Duchenne
- Keywords
- Muscular Dystrophy, DMD, Duchenne
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow enrollment
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Mallinckrodt ARD LLCIndustry (pharma, biotech, devices)
- Study start
- 27 Jul 2018 (actual)
- Primary completion
- 25 Feb 2020 (actual)
- Study completion
- 25 Feb 2020 (actual)
- First posted
- 17 Jan 2018 (actual)
- Results
- Posted 21 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
Detailed description
The main purpose of this study is to determine the effect of MNK-1411 on motor function in participants with Duchenne Muscular Dystrophy (DMD). Information is collected only from caretakers who are fluent in English, using the Pediatric Outcomes Data Collection Instrument (PODCI).
The PODCI is a validated 86-question instrument completed by the parent or legal guardian of children 2 to 10 years of age to assess a variety of health outcome measures (Uzark et al, 2012). This study will only collect information for the PODCI domains of sports and physical functioning and transfer/basic mobility.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
- Model description
- In the double-blind period, participants are randomized to receive study drug or placebo. It is followed by an open label period wherein all participants receive study drug.
| Arm | Type | Receives |
|---|---|---|
| Period 1: MNK-1411 | Experimental | Drug: MNK-1411Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1 |
| Period 1: Placebo | Experimental | Other: PlaceboParticipants receive placebo at a volume appropriate to body weight during Period 1 |
| Period 2: MNK-1411 | Experimental | Drug: MNK-1411All participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2 |
Who may qualify
- Ages
- 4 to 8 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Participants must have a documented diagnosis of Duchenne Muscular Dystrophy (DMD) confirmed by complete dystrophin deficiency (by immunofluorescence and/or immunoblot), or identifiable mutation in the DMD gene where reading frame can be predicated as "out of frame," or complete dystrophin gene sequencing consistent with DMD; AND in the opinion of the Investigator, a typical clinical profile consistent with DMD.
- Participants taking approved treatments for DMD (by a Health Authority) that target dystrophin gene mutations (e.g., eteplirsen or ataluren) may be enrolled in the study if they have been on a stable dose for 30 days prior to the first dose of study drug, and plan to remain on that dose throughout the study.
Key exclusion criteria (6 listed)
- Participant has had previous systemic treatment with corticosteroids within 2 months prior to the Screening Visit. Exception: In subjects who were down-titrated to a physiological dose of corticosteroids (ie, 3mg/m2 of prednisone or deflazacort) a maximum of 1 month of no greater than a physiological dose followed by 1 month completely off corticosteroids prior to the Screening Visit will be acceptable for study entry. Transient previous use of corticosteroids will be evaluated on a case-by-case basis by the sponsor or designee. The use of topical or intra-articular corticosteroids is permitted during the study
- Participant is unable to complete the 10 meter Walk/Run test at the Screening and/or Baseline Visit.
- Participant has Type 1 or Type 2 diabetes mellitus.
- Participant has a history of chronic active hepatitis including acute or chronic hepatitis B, or acute or chronic hepatitis C.
- Participant has a history of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
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 |
|---|---|---|
| MNK-1411Also: Cosyntropin acetate, Tetracosactide HexaacetateMNK-1411 (1 mg/mL suspension) for subcutaneous injection | Drug | Period 1: MNK-1411, Period 2: MNK-1411 |
| PlaceboAlso: Matching PlaceboPlacebo suspension for subcutaneous injection | Other | Period 1: Placebo |
Primary outcome
Time to Complete 10 Meter Walk/Run[
Time frame: Baseline, Week 24
10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.
7 secondary outcomes
North Star Ambulatory Assessment (NSAA) Score
Time frame: Baseline, Week 24
Time to Climb 4 Standardized Stairs
Time frame: Baseline, Week 24
Time to Stand From a Supine Position
Time frame: Baseline, Week 24
Quantitative Muscle Testing Scores at Baseline
Time frame: Baseline
Quantitative Muscle Testing Scores at Week 24
Time frame: Week 24
Summary of Adverse Events in the Blinded Treatment Period
Time frame: within 28 weeks
Summary of Adverse Events in the Open Label Period
Time frame: within 28 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No. Discussion of statistical endpoints and analysis are included in manuscripts. Summary aggregate (basic) results (including adverse events information) and the study protocol are made available on clinicaltrials.gov (NCT03400852) when required by regulation. Individual de-identified patient data will not be disclosed. Requests for additional information should be directed to the company at medinfo@mnk.com.
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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.
Clinical Study Lead study director
Mallinckrodt
16 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MNK-1411 in people with Muscular Dystrophy, Duchenne, run by Mallinckrodt ARD LLC at 16 sites in 8 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 2 inclusion and 6 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: stopped early, as of the sponsor's last update on 16 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Mexico, Spain, Bulgaria, Israel and 3 more countries. 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 21 Feb 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?
Time to Complete 10 Meter Walk/Run[ (time frame: Baseline, Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 16 Mar 2021.