NCT02858362

Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)

Phaseout DMD: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation With Ezutromid in Ambulatory Paediatric Male Subjects With Duchenne Muscular Dystrophy (SMT C11005)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 43 (actual)
• Sponsor: Summit Therapeutics · Industry
• Sex: Male
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

To Assess the Activity and Safety of SMT C1100 (Ezutromid) in Paediatric Male Participants with Duchenne Muscular Dystrophy (DMD).

Detailed description

This is a Phase 2, open label, study to assess the activity and safety of utrophin modulation with SMT C1100 (ezutromid) administered twice-daily orally in ambulatory paediatric male participants with DMD. This study will be conducted in a multi-centre setting in both the United Kingdom and the United States of America and comprises of a Screening and Baseline Phase of up to 28 days, a 48-week open label Treatment Phase, and either a 30-day Safety Follow up Phase or an optional extension phase where study treatment is provided until discontinuation of the program or regulatory approvals as applicable.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Ezutromid	Administered orally.

Timeline

• Start date: 2016-06-01
• Primary completion: 2018-04-11
• Completion: 2018-09-11
• First posted: 2016-08-08
• Last updated: 2020-01-02
• Results posted: 2020-01-02

Locations

16 sites across 2 countries: United States, United Kingdom