Primary outcomeMedian· Baseline, Week 24
Time to Complete 10 Meter Walk/Run[
| Group | Value (seconds) |
|---|---|
| Period 1: MNK-1411 | 5.9 |
| Period 1: Placebo | 7.8 |
Trial · Interventional
TerminatedPhase 2Results postedRegistry dataA Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
Short name BRAVE
NCTNCT03400852 (opens ClinicalTrials.gov in a new tab)Other idMNK14112096Other id2017-004139-35Other id2017
This trial was stopped early.
Reason given at the registry: Slow enrollment
Results were posted for this trial.
Overview
- Status
- Terminated
- Phase
- Phase 2
- Enrollment
- 44 participants (actual)
- Sponsor
- Mallinckrodt ARD LLC · Industry
- Design
- Randomized · Double masking
- Countries
8 countries
United States, Bulgaria, Israel, Italy, Mexico, Serbia, Spain, Turkey (Türkiye)
- Start
- 27 Jul 2018
- Primary completion
- 25 Feb 2020
- Completion
- 25 Feb 2020
- First posted
- 17 Jan 2018
- Last update posted
- 16 Mar 2021
- Conditions
- Muscular Dystrophy, Duchenne
Timeline
1 trial · 1 stopped early · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
2018201920202021
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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).
Interventions
- MNK-1411Drug
- PlaceboOther
Arms3
Experimental
Period 1: MNK-1411
Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1
Experimental
Period 1: Placebo
Participants receive placebo at a volume appropriate to body weight during Period 1
Experimental
Period 2: MNK-1411
All participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2
Primary outcomes1
Outcomes the sponsor listed as primary.
Time to Complete 10 Meter Walk/Run[
Time frame · Baseline, Week 24
Registry description
10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.
Secondary outcomes(7)
North Star Ambulatory Assessment (NSAA) Score
Time frame · Baseline, Week 24
Registry description
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.
Time to Climb 4 Standardized Stairs
Time frame · Baseline, Week 24
Registry description
Time to Climb 4 Standardized Stairs is a motor performance test
Time to Stand From a Supine Position
Time frame · Baseline, Week 24
Registry description
Time to stand from a supine position is a motor function test to measure the functional capability in subjects with DMD.
Quantitative Muscle Testing Scores at Baseline
Time frame · Baseline
Registry description
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
Quantitative Muscle Testing Scores at Week 24
Time frame · Week 24
Registry description
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
Summary of Adverse Events in the Blinded Treatment Period
Time frame · within 28 weeks
Registry description
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Summary of Adverse Events in the Open Label Period
Time frame · within 28 weeks
Registry description
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(7)
Secondary outcomeMean· Baseline, Week 24
North Star Ambulatory Assessment (NSAA) Score
| Group | Value (score on a scale) | Spread |
|---|---|---|
| Period 1: MNK-1411 | 17.9 | 6.80 |
| Period 1: Placebo | 17.1 | 6.40 |
Secondary outcomeMean· Baseline, Week 24
Time to Climb 4 Standardized Stairs
| Group | Value (seconds) | Spread |
|---|---|---|
| Period 1: MNK-1411 | 8.52 | 8.88 |
| Period 1: Placebo | 8.47 | 4.34 |
Secondary outcomeMean· Baseline, Week 24
Time to Stand From a Supine Position
| Group | Value (seconds) | Spread |
|---|---|---|
| Period 1: MNK-1411 | 11.14 | 9.08 |
| Period 1: Placebo | 15.03 | 12.45 |
Secondary outcomeMean· Baseline
Quantitative Muscle Testing Scores at Baseline
| Group | Value (Newtons) | Spread |
|---|---|---|
| Period 1: MNK-1411 | 26.61 | 14.17 |
| Period 1: Placebo | 29.87 | 14.17 |
Secondary outcomeMean· Week 24
Quantitative Muscle Testing Scores at Week 24
| Group | Value (Newtons) | Spread |
|---|---|---|
| Period 1: MNK-1411 | 33.64 | 1578 |
| Period 1: Placebo | 25.27 | 13.35 |
Secondary outcomeCount of participants· within 28 weeks
Summary of Adverse Events in the Blinded Treatment Period
| Group | Value (Participants) |
|---|---|
| Period 1: MNK-1411 | 29 |
| Period 1: Placebo | 15 |
Secondary outcomeCount of participants· within 28 weeks
Summary of Adverse Events in the Open Label Period
| Group | Value (Participants) |
|---|---|
| Period 2: MNK-1411 | 24 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Period 1: MNK-1411Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1 | 0 / 29 | 0 / 29 | 22 / 29 |
| Period 1: PlaceboParticipants receive placebo at a volume appropriate to body weight during Period 1 | 0 / 15 | 1 / 15 | 15 / 15 |
| Period 2: MNK-1411Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2 | 0 / 24 | 2 / 24 | 11 / 24 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- Male
- Age
- from 4 Years up to 8 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
1. 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.
2. 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.
Exclusion Criteria:
1. 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
2. Participant is unable to complete the 10 meter Walk/Run test at the Screening and/or Baseline Visit.
3. Participant has Type 1 or Type 2 diabetes mellitus.
4. Participant has a history of chronic active hepatitis including acute or chronic hepatitis B, or acute or chronic hepatitis C.
5. 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.
6. 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.
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