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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 : April 15, 2019
Sponsor:
Information provided by (Responsible Party):
Mallinckrodt

Tracking Information
First Submitted Date  ICMJE January 9, 2018
First Posted Date  ICMJE January 17, 2018
Last Update Posted Date April 15, 2019
Actual Study Start Date  ICMJE July 17, 2018
Estimated Primary Completion Date July 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 9, 2018)
10 meter walk/run [ Time Frame: 24 weeks ]
Motor performance test
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03400852 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 17, 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 measured in Newtons, using a dynamometer
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 Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Muscular Dystrophy, Duchenne
Intervention  ICMJE
  • Drug: Cosyntropin
    Suspension administered subcutaneously 2X week
    Other Name: MNK1411
  • Other: Placebo
    Suspension administered subcutaneously 2X week
Study Arms  ICMJE
  • 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
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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 9, 2018)
132
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2020
Estimated Primary Completion Date July 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: Study Manager 800-556-3314 clinicaltrials@mnk.com
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
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 Not Provided
Responsible Party Mallinckrodt
Study Sponsor  ICMJE Mallinckrodt
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Study Director Mallinckrodt
PRS Account Mallinckrodt
Verification Date January 2019

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