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

NCT03400852 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: MNK141120962017-004139-35
• Study Type: interventional
• Target: 44
• Locations: 8 countries
• Sites: 16

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).

Conditions

Muscular Dystrophy, Duchenne

Keywords

Muscular Dystrophy; DMD; Duchenne

Outcome Measures

Primary Outcomes (1)

• Time to Complete 10 Meter Walk/Run[

  10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.

  Baseline, Week 24

Secondary Outcomes (7)

• North Star Ambulatory Assessment (NSAA) Score

  Baseline, Week 24

• Time to Climb 4 Standardized Stairs

  Baseline, Week 24

• Time to Stand From a Supine Position

  Baseline, Week 24

• Quantitative Muscle Testing Scores at Baseline

  Baseline

• Quantitative Muscle Testing Scores at Week 24

  Week 24

Study Arms (3)

Period 1: MNK-1411

EXPERIMENTAL

Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1

Drug: MNK-1411

Period 1: Placebo

EXPERIMENTAL

Participants receive placebo at a volume appropriate to body weight during Period 1

Other: Placebo

Period 2: MNK-1411

EXPERIMENTAL

All participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2

Drug: MNK-1411

Interventions

MNK-1411 DRUG

MNK-1411 (1 mg/mL suspension) for subcutaneous injection

Also known as: Cosyntropin acetate, Tetracosactide Hexaacetate

Period 1: MNK-1411; Period 2: MNK-1411

Placebo OTHER

Placebo suspension for subcutaneous injection

Also known as: Matching Placebo

Period 1: Placebo

Eligibility Criteria

• Age: 4 Years - 8 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

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.

Sponsors & Collaborators

• Mallinckrodt ARD LLC: lead

Study Sites (16)

NW FL Clinical Research Group, LLC

Gulf Breeze, Florida, 32561, United States

Location

Rare Disease Research, LLC

Atlanta, Georgia, 30318, United States

Location

Monroe Carell Jr Childrens Hospital at Vanderbilt

Nashville, Tennessee, 37232, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75207, United States

Location

UT Health Science Center, San Antonio

San Antonio, Texas, 78229, United States

Location

University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

Sofia, 1431, Bulgaria

Location

Edith Wolfson Medical Center

Holon, 5810001, Israel

Location

Ospedale San Raffaele S.r.l. - PPDS

Milan, Lombardy, 20132, Italy

Location

Hospital Civil Fray Antonio Alcalde

Guadalajara, Jalisco, 44280, Mexico

Location

Neurociencias Estudios Clinicos S.C.

Culiacán, Sinaloa, 80020, Mexico

Location

Instituto de Investigaciones Aplicadas a la Neurociencia A.C.

Durango, 34000, Mexico

Location

Clinic of Neurology and Psychiatry for Children and Youth

Belgrade, 11000, Serbia

Location

Hospital de La Santa Creu i Sant Pau

Barcelona, 08025, Spain

Location

Hospital Sant Joan de Deu - PIN

Esplugues de Llobregat, 08950, Spain

Location

Hospital Universitari i Politecnic La Fe Valencia

Valencia, 46026, Spain

Location

Mersin Universitesi Tip Fakultesi Hastanesi

Mersin, 33343, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Interventions

Cosyntropin

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Adrenocorticotropic Hormone; Melanocortins; Pro-Opiomelanocortin; Hypothalamic Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Pituitary Hormones, Anterior; Pituitary Hormones; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Nerve Tissue Proteins; Proteins

Results Point of Contact

• Title: Medical Information Call Center
• Organization: Mallinckrodt

MedInfo@mnk.com

1-800-844-2830

Study Officials

• Clinical Study Lead

  Mallinckrodt

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: January 9, 2018
• First Posted: January 17, 2018
• Study Start: July 27, 2018
• Primary Completion: February 25, 2020
• Study Completion: February 25, 2020
• Last Updated: March 16, 2021
• Results First Posted: February 21, 2021

Record last verified: 2021-02

Data Sharing

• IPD Sharing: Will not share

Locations

ME (3); ES (3); TU (1); US (5); IL (1); BU (1); SE (1); IT (1)