A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
Condition: Muscular Dystrophy, Duchenne
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: MNK-1411; Placebo
Intervention description: MNK-1411 (1 mg/mL suspension) for subcutaneous injection; Placebo suspension for subcutaneous injection
Intervention MeSH terms: Cosyntropin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: In the double-blind period, participants are randomized to receive study drug or placebo. It is followed by an open label period wherein all participants receive study drug.
Masking: Double (Participant, Investigator)
Masking description: Double blind (Investigator/Participant) The Care Provider and Outcomes Assessor were also blinded, but it is a double-blind trial, followed by an open-label period.
Enrollment: 44.0
Sex: Male
Minimum age: 4 Years
Maximum age: 8 Years
Healthy volunteers: No
Keywords: Muscular Dystrophy; DMD; Duchenne

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).
    

Eligibility criteria:

        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.