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

NCT ID: NCT03400852

Last Updated: 2021-03-16

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 44 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-07-27
• Study Completion Date: 2020-02-25

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).

Detailed Description

The main purpose of this study is to determine the effect of MNK-1411 on motor function in participants with Duchenne Muscular Dystrophy (DMD). Information is collected only from caretakers who are fluent in English, using the Pediatric Outcomes Data Collection Instrument (PODCI).

The PODCI is a validated 86-question instrument completed by the parent or legal guardian of children 2 to 10 years of age to assess a variety of health outcome measures (Uzark et al, 2012). This study will only collect information for the PODCI domains of sports and physical functioning and transfer/basic mobility.

Conditions

Muscular Dystrophy, Duchenne

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Period 1: MNK-1411

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

Group Type: EXPERIMENTAL

MNK-1411

Intervention Type: DRUG

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

Period 1: Placebo

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

Group Type: EXPERIMENTAL

Placebo

Intervention Type: OTHER

Placebo suspension for subcutaneous injection

Period 2: MNK-1411

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

Group Type: EXPERIMENTAL

MNK-1411

Intervention Type: DRUG

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

Interventions

MNK-1411

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

Intervention Type: DRUG

Placebo

Placebo suspension for subcutaneous injection

Intervention Type: OTHER

Other Intervention Names

Cosyntropin acetate; Tetracosactide Hexaacetate; Matching Placebo

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.

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 8 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Mallinckrodt ARD LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Study Lead

Role: STUDY_DIRECTOR

Mallinckrodt

Locations

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)

Countries

United States; Bulgaria; Israel; Italy; Mexico; Serbia; Spain; Turkey (Türkiye)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

2017-004139-35

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MNK14112096

Identifier Type: -

Identifier Source: org_study_id