NCT02752048

A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy

A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (actual)
• Sponsor: Taiho Pharmaceutical Co., Ltd. · Industry
• Sex: Male
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.

Detailed description

Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in 3,500 lives male births. DMD patients suffer from a relentless decline in muscle strength that impairs the ability of walking and breathing, resulting in their lives with wheelchairs and then loss of upper body function. The main objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with DMD in an exploratory manner. The objective of this study is also to evaluate the safety, the dose-response and the urinary excretion of pharmacodynamic (PD) marker after 24-week repeated oral doses of TAS-205 in DMD patients.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	TAS-205	2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
DRUG	Placebo	1 group: Placebo group. Oral administration for 24 weeks, BID after meal

Timeline

• Start date: 2016-05-01
• Primary completion: 2017-05-15
• Completion: 2017-10-17
• First posted: 2016-04-26
• Last updated: 2020-04-20
• Results posted: 2020-04-20

Locations

11 sites across 1 country: Japan