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Trial record 1 of 1 for:    MNK-1411 | Duchenne Muscular Dystrophy
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A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy (BRAVE)

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ClinicalTrials.gov Identifier: NCT03400852
Recruitment Status : Terminated (Slow enrollment)
First Posted : January 17, 2018
Results First Posted : February 21, 2021
Last Update Posted : March 16, 2021
Sponsor:
Information provided by (Responsible Party):
Mallinckrodt ( Mallinckrodt ARD LLC )

Tracking Information
First Submitted Date  ICMJE January 9, 2018
First Posted Date  ICMJE January 17, 2018
Results First Submitted Date  ICMJE February 2, 2021
Results First Posted Date  ICMJE February 21, 2021
Last Update Posted Date March 16, 2021
Actual Study Start Date  ICMJE July 27, 2018
Actual Primary Completion Date February 25, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 2, 2021)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: January 9, 2018)
10 meter walk/run [ Time Frame: 24 weeks ]
Motor performance test
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 2, 2021)
  • North Star Ambulatory Assessment (NSAA) Score [ Time Frame: Baseline, Week 24 ]
    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 ]
    Time to Climb 4 Standardized Stairs is a motor performance test
  • Time to Stand From a Supine Position [ Time Frame: Baseline, Week 24 ]
    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 ]
    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 ]
    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 ]
    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 ]
    Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Original Secondary Outcome Measures  ICMJE
 (submitted: January 9, 2018)
  • NorthStar Ambulatory Assessment [ Time Frame: 24 weeks ]
    Motor performance battery
  • 4 stair climb [ Time Frame: 24 weeks ]
    Motor performance test
  • Rise from supine test [ Time Frame: 24 weeks ]
    Motor performance test
  • Quantitative muscle testing [ Time Frame: 24 weeks ]
    Strength-knee flexion and extension
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
Official Title  ICMJE 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
Brief 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).
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention 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 (Participant, Investigator)
Masking Description:
Double blind (Investigator/Participant) The Care Provider and Outcomes Assessor were also blinded, but it is a double-blind trial, followed by an open-label period.
Primary Purpose: Treatment
Condition  ICMJE Muscular Dystrophy, Duchenne
Intervention  ICMJE
  • Drug: MNK-1411
    MNK-1411 (1 mg/mL suspension) for subcutaneous injection
    Other Names:
    • Cosyntropin acetate
    • Tetracosactide Hexaacetate
  • Other: Placebo
    Placebo suspension for subcutaneous injection
    Other Name: Matching Placebo
Study Arms  ICMJE
  • Experimental: Period 1: MNK-1411
    Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1
    Intervention: Drug: MNK-1411
  • Experimental: Period 1: Placebo
    Participants receive placebo at a volume appropriate to body weight during Period 1
    Intervention: Other: Placebo
  • Experimental: Period 2: MNK-1411
    All participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2
    Intervention: Drug: MNK-1411
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: January 16, 2020)
44
Original Estimated Enrollment  ICMJE
 (submitted: January 9, 2018)
132
Actual Study Completion Date  ICMJE February 25, 2020
Actual Primary Completion Date February 25, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 4 Years to 8 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Bulgaria,   Israel,   Italy,   Mexico,   Serbia,   Spain,   Turkey,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03400852
Other Study ID Numbers  ICMJE MNK14112096
2017-004139-35 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Plan Description: 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.
Responsible Party Mallinckrodt ( Mallinckrodt ARD LLC )
Study Sponsor  ICMJE Mallinckrodt ARD LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Study Lead Mallinckrodt
PRS Account Mallinckrodt
Verification Date February 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP