Mallinckrodt- MK1441 (BRAVE)

A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy

Hub Summary

This phase 2 study is designed to assess the safety and efficacy of MNK-1441. The double blind, placebo controlled, multiple dose study will take place in DMD boys between the ages of 4 and 8. 

It is thought that MNK-1441 has anti-inflammatory effects which could reduce muscle damage and in-turn delay DMD progression. 

Study Number: NCT03400852

Description by Mallinckrodt

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).

 
Primary Outcome Measures

  • 10 metre walk/run (25 weeks)

 
Secondary Outcome Measures

  • NorthStar Ambulatory Assessment
  • 4 stair climb
  • Rise from supine test
  • Quantitative muscle testing

 
Can I take part?

 
Inclusion Criteria

  • Subjects 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.
  • Subjects taking approved treatments for DMD (by a Health Authority) that target dystrophin gene mutations (eg, 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

  • Subject has had previous systemic treatment with corticosteroids. Transient previous use of corticosteroids will be evaluated on a case-by-case basis by the sponsor or designee. Inhaled corticosteroids will be permitted if given at a stable dose for the 3 months prior to the first dose of study drug and the subject will remain on that dose throughout the study. The use of topical or intra-articular corticosteroids is permitted during the study.
  • Subject has symptomatic cardiomyopathy in the opinion of the investigator.
  • Subject is unable to complete the 10 meter Walk/Run test at the Screening and/or Baseline Visit.
  • Subject has Type 1 or Type 2 diabetes mellitus.
  • Subject has a history of chronic active hepatitis including acute or chronic hepatitis B, or acute or chronic hepatitis C.
  • Subject has a history of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
  • Subject 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.
 
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Trial Status
Not yet recruiting
UK Locations
Trial Sponsor
Mallinckrodt
Age
4 to 8 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
MRI
No
Phase
2
Length Of Participation
48 weeks
Recruitment Target
132
Ambulatory
Ambulant
Therapeutic Category
Steroid alternative