COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu
Trial record 1 of 1 for:    MNK-1411 | Muscular Dystrophies
Previous Study | Return to List | Next Study

A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy (BRAVE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03400852
Recruitment Status : Terminated (Slow enrollment)
First Posted : January 17, 2018
Last Update Posted : August 14, 2020
Information provided by (Responsible Party):
Mallinckrodt ( Mallinckrodt ARD LLC )

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).

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Drug: Cosyntropin Other: Placebo Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 44 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
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.
Primary Purpose: Treatment
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
Actual Study Start Date : July 17, 2018
Actual Primary Completion Date : February 25, 2020
Actual Study Completion Date : February 25, 2020

Arm Intervention/treatment
Experimental: Treatment A, MNK1411 High Dose
Cosyntropin suspension 0.5/0.4 mL for up to 48 weeks
Drug: Cosyntropin
Suspension administered subcutaneously twice weekly
Other Name: MNK1411

Experimental: Treatment B, MNK1411 Low Dose
Cosyntropin suspension 0.25/0.2 mL for up to 48 weeks
Drug: Cosyntropin
Suspension administered subcutaneously twice weekly
Other Name: MNK1411

Placebo Comparator: Treatment C, Placebo High Dose
Placebo suspension 0.4/0.5 mL for up to 24 weeks
Other: Placebo
Suspension administered subcutaneously twice weekly
Other Name: Matching Placebo

Placebo Comparator: Treatment D, Placebo Low Dose
Placebo suspension 0.25/0.2 mL for up to 24 weeks
Other: Placebo
Suspension administered subcutaneously twice weekly
Other Name: Matching Placebo

Primary Outcome Measures :
  1. 10 meter walk/run [ Time Frame: 24 weeks ]
    Motor performance test

Secondary Outcome Measures :
  1. NorthStar Ambulatory Assessment [ Time Frame: 24 weeks ]
    Motor performance battery

  2. 4 stair climb [ Time Frame: 24 weeks ]
    Motor performance test

  3. Rise from supine test [ Time Frame: 24 weeks ]
    Motor performance test

  4. Quantitative muscle testing [ Time Frame: 24 weeks ]
    Strength-knee flexion and extension measured in Newtons, using a dynamometer

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   4 Years to 8 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03400852

Layout table for location information
United States, Florida
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States, 32561
United States, Georgia
Rare Disease Research, LLC
Atlanta, Georgia, United States, 30318
United States, Tennessee
Monroe Carell Jr Childrens Hospital at Vanderbilt
Nashville, Tennessee, United States, 37232
United States, Texas
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75207
UT Health Science Center, San Antonio
San Antonio, Texas, United States, 78229
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, Bulgaria, 1431
Edith Wolfson Medical Center
H̱olon, Israel, 5810001
Ospedale San Raffaele S.r.l. - PPDS
Milano, Lombardia, Italy, 20132
Hospital Civil Fray Antonio Alcalde
Guadalajara, Jalisco, Mexico, 44280
Neurociencias Estudios Clinicos S.C.
Culiacán, Sinaloa, Mexico, 80020
Instituto de Investigaciones Aplicadas a la Neurociencia A.C.
Durango, Mexico, 34000
Clinic of Neurology and Psychiatry for Children and Youth
Belgrade, Serbia, 11000
Hospital de La Santa Creu i Sant Pau
Barcelona, Spain, 08025
Hospital Sant Joan de Deu - PIN
Esplugues De Llobregat, Spain, 08950
Hospital Universitari i Politecnic La Fe Valencia
Valencia, Spain, 46026
Mersin Universitesi Tip Fakultesi Hastanesi
Mersin, Turkey, 33343
Sponsors and Collaborators
Mallinckrodt ARD LLC
Layout table for investigator information
Study Director: Clinical Study Lead Mallinckrodt
Layout table for additonal information
Responsible Party: Mallinckrodt ARD LLC Identifier: NCT03400852    
Other Study ID Numbers: MNK14112096
2017-004139-35 ( EudraCT Number )
First Posted: January 17, 2018    Key Record Dates
Last Update Posted: August 14, 2020
Last Verified: August 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Mallinckrodt ( Mallinckrodt ARD LLC ):
Muscular Dystrophy
Additional relevant MeSH terms:
Layout table for MeSH terms
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs