NCT03400852

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

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 44 (actual)
• Sponsor: Mallinckrodt ARD LLC · Industry
• Sex: Male
• Age: 4 Years – 8 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	MNK-1411	MNK-1411 (1 mg/mL suspension) for subcutaneous injection
OTHER	Placebo	Placebo suspension for subcutaneous injection

Timeline

• Start date: 2018-07-27
• Primary completion: 2020-02-25
• Completion: 2020-02-25
• First posted: 2018-01-17
• Last updated: 2021-03-16
• Results posted: 2021-02-21

Locations

16 sites across 8 countries: United States, Bulgaria, Israel, Italy, Mexico, Serbia, Spain, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study