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



Status:Recruiting
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:4 - 8
Updated:3/8/2019
Start Date:July 17, 2018
End Date:August 2020
Contact:Study Manager
Email:clinicaltrials@mnk.com
Phone:800-556-3314

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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).


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.
We found this trial at
10
sites
Wauwatosa, Wisconsin 53226
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Los Angeles, CA
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4502 Medical Drive
San Antonio, Texas 78284
(210) 567-7000
University of Texas Health Science Center at San Antonio The University of Texas Health Science...
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San Antonio, TX
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Atlanta, Georgia 30318
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Atlanta, GA
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1653 W. Congress Parkway
Chicago, Illinois 60612
(312) 942-5000
Rush University Medical Center Rush University Medical Center encompasses a 664-bed hospital serving adults and...
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Chicago, IL
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Gulf Breeze, Florida 32561
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Gulf Breeze, FL
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H?olon,
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Las Vegas, Nevada 89145
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Las Vegas, NV
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630 W 168th St
New York, New York
212-305-2862
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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New York, NY
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Norfolk, Virginia 23510
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Norfolk, VA
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