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).
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
44
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
Time to Complete 10 Meter Walk/Run[
10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.
Time frame: Baseline, Week 24
North Star Ambulatory Assessment (NSAA) Score
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.
Time frame: Baseline, Week 24
Time to Climb 4 Standardized Stairs
Time to Climb 4 Standardized Stairs is a motor performance test
Time frame: Baseline, Week 24
Time to Stand From a Supine Position
Time to stand from a supine position is a motor function test to measure the functional capability in subjects with DMD.
Time frame: Baseline, Week 24
Quantitative Muscle Testing Scores at Baseline
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
Time frame: Baseline
Quantitative Muscle Testing Scores at Week 24
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
Time frame: Week 24
Summary of Adverse Events in the Blinded Treatment Period
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Monroe Carell Jr Childrens Hospital at Vanderbilt
Nashville, Tennessee, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
UT Health Science Center, San Antonio
San Antonio, Texas, United States
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, Bulgaria
Edith Wolfson Medical Center
Holon, Israel
Ospedale San Raffaele S.r.l. - PPDS
Milan, Lombardy, Italy
Hospital Civil Fray Antonio Alcalde
Guadalajara, Jalisco, Mexico
Neurociencias Estudios Clinicos S.C.
Culiacán, Sinaloa, Mexico
...and 6 more locations
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Time frame: within 28 weeks
Summary of Adverse Events in the Open Label Period
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Time frame: within 28 weeks