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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)

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.
 
ClinicalTrials.gov Identifier: NCT03400852
Recruitment Status : Terminated (Slow enrollment)
First Posted : January 17, 2018
Results First Posted : February 21, 2021
Last Update Posted : February 21, 2021
Sponsor:
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: MNK-1411 Other: Placebo Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 44 participants
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
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 27, 2018
Actual Primary Completion Date : February 25, 2020
Actual Study Completion Date : February 25, 2020


Arm Intervention/treatment
Experimental: Period 1: MNK-1411
Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1
Drug: MNK-1411
MNK-1411 (1 mg/mL suspension) for subcutaneous injection
Other Names:
  • Cosyntropin acetate
  • Tetracosactide Hexaacetate

Experimental: Period 1: Placebo
Participants receive placebo at a volume appropriate to body weight during Period 1
Other: Placebo
Placebo suspension for subcutaneous injection
Other Name: Matching Placebo

Experimental: Period 2: MNK-1411
All participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2
Drug: MNK-1411
MNK-1411 (1 mg/mL suspension) for subcutaneous injection
Other Names:
  • Cosyntropin acetate
  • Tetracosactide Hexaacetate




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. Time to Climb 4 Standardized Stairs [ Time Frame: Baseline, Week 24 ]
    Time to Climb 4 Standardized Stairs is a motor performance test

  3. 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.

  4. Quantitative Muscle Testing Scores at Baseline [ Time Frame: Baseline ]
    Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer

  5. 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

  6. 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)

  7. 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)



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.


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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


Locations
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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
Bulgaria
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, Bulgaria, 1431
Israel
Edith Wolfson Medical Center
H̱olon, Israel, 5810001
Italy
Ospedale San Raffaele S.r.l. - PPDS
Milano, Lombardia, Italy, 20132
Mexico
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
Serbia
Clinic of Neurology and Psychiatry for Children and Youth
Belgrade, Serbia, 11000
Spain
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
Turkey
Mersin Universitesi Tip Fakultesi Hastanesi
Mersin, Turkey, 33343
Sponsors and Collaborators
Mallinckrodt ARD LLC
Investigators
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Study Director: Clinical Study Lead Mallinckrodt
  Study Documents (Full-Text)

Documents provided by Mallinckrodt ( Mallinckrodt ARD LLC ):
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Responsible Party: Mallinckrodt ARD LLC
ClinicalTrials.gov Identifier: NCT03400852    
Other Study ID Numbers: MNK14112096
2017-004139-35 ( EudraCT Number )
First Posted: January 17, 2018    Key Record Dates
Results First Posted: February 21, 2021
Last Update Posted: February 21, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
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.

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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
DMD
Duchenne
Additional relevant MeSH terms:
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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