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

study id #: NCT03400852

condition: Muscular Dystrophy, Duchenne

status: recruiting

purpose:

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). Research: Anti-inflammatory

intervention: Cosyntropin (MNK-1411), Placebo

mechanism of action: Melanocortin receptor agonist to reduce inflammation and muscle damage

results: https://clinicaltrials.gov/ct2/show/results/NCT03400852

last updated: April 27, 2019

start date: July 17, 2018

estimated completion: July 2020

phase of development: Phase 2

size / enrollment: 132

primary outcomes:

• 10 meter walk/run [ Time Frame: 24 weeks ]
  Motor performance test

secondary outcomes:

• NorthStar Ambulatory Assessment [ Time Frame: 24 weeks ]
  Motor performance battery
  
• 4 stair climb [ Time Frame: 24 weeks ]
  Motor performance test
  
• Rise from supine test [ Time Frame: 24 weeks ]
  Motor performance test
  
• Quantitative muscle testing [ Time Frame: 24 weeks ]
  Strength-knee flexion and extension measured in Newtons, using a dynamometer

inclusion criteria:

• 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.

exclusion criteria:
• 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 has symptomatic cardiomyopathy in the opinion of the investigator.
• 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.

sponsor: Mallinckrodt

contacts: Study Manager, 800-556-3314, Clinicaltrials@mnk.com

investigators: Study Director, Mallinckrodt

trial center locations: United States, Bulgaria, Israel, Italy, Mexico, Serbia, Spain, Turkey