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

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Muscular Dystrophy, Duchenne

In brief

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).

Key facts

Study ID: NCT03400852
Run by: Mallinckrodt ARD LLC
People needed: 44
Starts: 2018-07-27
Expected to finish: 2020-02-25
Last updated by the study team: 2021-03-16

Who can join

Age: 4 and older, up to 8. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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.

Where it is running

NW FL Clinical Research Group, LLC — Gulf Breeze, Florida, United States
Rare Disease Research, LLC — Atlanta, Georgia, United States
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
Instituto de Investigaciones Aplicadas a la Neurociencia A.C. — Durango, Mexico
Clinic of Neurology and Psychiatry for Children and Youth — Belgrade, Serbia
Hospital de La Santa Creu i Sant Pau — Barcelona, Spain
Hospital Sant Joan de Deu - PIN — Esplugues de Llobregat, Spain
Hospital Universitari i Politecnic La Fe Valencia — Valencia, Spain
Mersin Universitesi Tip Fakultesi Hastanesi — Mersin, Turkey (Türkiye)

Full record on ClinicalTrials.gov

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