A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
- Registration Number
- NCT03400852
- Lead Sponsor
- 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).
- 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 44
- 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.
- 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.
- 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
- Participant is unable to complete the 10 meter Walk/Run test at the Screening and/or Baseline Visit.
- Participant has Type 1 or Type 2 diabetes mellitus.
- Participant has a history of chronic active hepatitis including acute or chronic hepatitis B, or acute or chronic hepatitis C.
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Period 1: Placebo Placebo Participants receive placebo at a volume appropriate to body weight during Period 1 Period 1: MNK-1411 MNK-1411 Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1 Period 2: MNK-1411 MNK-1411 All participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2
- Primary Outcome Measures
Name Time Method Time to Complete 10 Meter Walk/Run[ Baseline, Week 24 10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.
- Secondary Outcome Measures
Name Time Method Time to Climb 4 Standardized Stairs Baseline, Week 24 Time to Climb 4 Standardized Stairs is a motor performance test
Quantitative Muscle Testing Scores at Baseline Baseline Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
North Star Ambulatory Assessment (NSAA) Score 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.
Time to Stand From a Supine Position Baseline, Week 24 Time to stand from a supine position is a motor function test to measure the functional capability in subjects with DMD.
Quantitative Muscle Testing Scores at Week 24 Week 24 Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
Summary of Adverse Events in the Blinded Treatment Period within 28 weeks Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Summary of Adverse Events in the Open Label Period within 28 weeks Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Trial Locations
- Locations (16)
Neurociencias Estudios Clinicos S.C.
🇲🇽Culiacán, Sinaloa, Mexico
Hospital de La Santa Creu i Sant Pau
🇪🇸Barcelona, Spain
Hospital Universitari i Politecnic La Fe Valencia
🇪🇸Valencia, Spain
Instituto de Investigaciones Aplicadas a la Neurociencia A.C.
🇲🇽Durango, Mexico
NW FL Clinical Research Group, LLC
🇺🇸Gulf Breeze, Florida, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
UT Health Science Center, San Antonio
🇺🇸San Antonio, Texas, United States
Ospedale San Raffaele S.r.l. - PPDS
🇮🇹Milano, Lombardia, Italy
Hospital Sant Joan de Deu - PIN
🇪🇸Esplugues De Llobregat, Spain
Edith Wolfson Medical Center
🇮🇱H̱olon, Israel
Mersin Universitesi Tip Fakultesi Hastanesi
🇹🇷Mersin, Turkey
Hospital Civil Fray Antonio Alcalde
🇲🇽Guadalajara, Jalisco, Mexico
Clinic of Neurology and Psychiatry for Children and Youth
🇷🇸Belgrade, Serbia
Monroe Carell Jr Childrens Hospital at Vanderbilt
🇺🇸Nashville, Tennessee, United States
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
🇧🇬Sofia, Bulgaria