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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 : November 14, 2018
Sponsor:
Information provided by (Responsible Party):
Mallinckrodt

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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 132 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
Estimated Primary Completion Date : August 2020
Estimated Study Completion Date : August 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 2X week
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 2X week
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 2X week

Placebo Comparator: Treatment D, Placebo Low Dose
Placebo suspension 0.25/0.2 mL for up to 24 weeks
Other: Placebo
Suspension administered subcutaneously 2X week




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



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Ages Eligible for Study:   4 Years to 8 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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 ClinicalTrials.gov identifier (NCT number): NCT03400852


Contacts
Contact: Study Manager 800-556-3314 clinicaltrials@mnk.com

Locations
United States, Florida
NW FL Clinical Research Group, LLC Recruiting
Gulf Breeze, Florida, United States, 32561
United States, Georgia
Rare Disease Research, LLC Recruiting
Atlanta, Georgia, United States, 30318
United States, Illinois
Rush University Medical Center Not yet recruiting
Chicago, Illinois, United States, 60612
United States, Nevada
Las Vegas Clinic Not yet recruiting
Las Vegas, Nevada, United States, 89145
United States, New York
Columbia University Medical Center Not yet recruiting
New York, New York, United States, 10032
United States, Texas
The University of Texas Health Science Center at San Antonio Not yet recruiting
San Antonio, Texas, United States, 78229
United States, Virginia
Children's Specialty Group Not yet recruiting
Norfolk, Virginia, United States, 23507
United States, Wisconsin
Children's Hospital of Wisconsin Not yet recruiting
Wauwatosa, Wisconsin, United States, 53226
Spain
Hospital de La Santa Creu i Sant Pau Not yet recruiting
Barcelona, Spain, 08041
Hospital Sant Joan de Deu Not yet recruiting
Barcelona, Spain, 08950
Sponsors and Collaborators
Mallinckrodt
Investigators
Study Director: Jacqueline Delfgaauw Mallinckrodt

Responsible Party: Mallinckrodt
ClinicalTrials.gov Identifier: NCT03400852     History of Changes
Other Study ID Numbers: MNK14112096
First Posted: January 17, 2018    Key Record Dates
Last Update Posted: November 14, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Mallinckrodt:
Muscular Dystrophy
DMD
Duchenne

Additional relevant 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
Cosyntropin
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs