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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 : Recruiting
First Posted : January 17, 2018
Last Update Posted : December 5, 2018
Sponsor:
Information provided by (Responsible Party):
Mallinckrodt

January 9, 2018
January 17, 2018
December 5, 2018
July 17, 2018
August 2020   (Final data collection date for primary outcome measure)
10 meter walk/run [ Time Frame: 24 weeks ]
Motor performance test
Same as current
Complete list of historical versions of study NCT03400852 on ClinicalTrials.gov Archive Site
  • 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 measured in Newtons, using a dynamometer
  • 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
Not Provided
Not Provided
 
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
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).
Not Provided
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Muscular Dystrophy, Duchenne
  • Drug: Cosyntropin
    Suspension administered subcutaneously 2X week
    Other Name: MNK1411
  • Other: Placebo
    Suspension administered subcutaneously 2X week
  • Experimental: Treatment A, MNK1411 High Dose
    Cosyntropin suspension 0.5/0.4 mL for up to 48 weeks
    Intervention: Drug: Cosyntropin
  • Experimental: Treatment B, MNK1411 Low Dose
    Cosyntropin suspension 0.25/0.2 mL for up to 48 weeks
    Intervention: Drug: Cosyntropin
  • Placebo Comparator: Treatment C, Placebo High Dose
    Placebo suspension 0.4/0.5 mL for up to 24 weeks
    Intervention: Other: Placebo
  • Placebo Comparator: Treatment D, Placebo Low Dose
    Placebo suspension 0.25/0.2 mL for up to 24 weeks
    Intervention: Other: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
132
Same as current
August 2020
August 2020   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: Male
4 Years to 8 Years   (Child)
No
Contact: Study Manager 800-556-3314 clinicaltrials@mnk.com
Spain,   United States
 
 
NCT03400852
MNK14112096
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Mallinckrodt
Mallinckrodt
Not Provided
Study Director: Jacqueline Delfgaauw Mallinckrodt
Mallinckrodt
December 2018

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