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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02752048
• Other study ID #: Taiho10053040
• Status: Completed
• Phase: Phase 2
• Start date: May 2016
• Est. completion date: October 17, 2017

Study information

• Verified date: March 2020
• Source: Taiho Pharmaceutical Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 36
• Est. completion date: October 17, 2017
• Est. primary completion date: May 15, 2017
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years and older

Eligibility

Inclusion Criteria:

• Able to give an informed consent. If applicable, able to give an informed assent.

• Phenotypic evidence of DMD.

• Male and ?5 years of age.

• Bodyweight ?7.5 kg and <60 kg.

• Able to complete the 6MWD test with a distance of at least 75 m.

• Able to take tablets.

• If taking oral glucocorticoids no significant change in the total daily or dosing 6 months before enrollment.

Exclusion Criteria:

• Any serious drug allergy.

• A forced vital capacity (FVC) of <50% of predicted value.

• Wearing a respirator continuously (except for the use during sleep).

• A left ventricular ejection fraction (EF) of <40% or fractional shortening (FS) of <25% on echocardiogram.

• Clinically significant cardiac failure and respiratory failure.

• Ongoing immunosuppressive therapy (other than corticosteroids) .

• Surgical history or plan for surgery that may affect muscular strength or motor function.

• Any injury that may affect muscular strength or motor function.

• With any systemic allergic disease or any chronic inflammatory disease.

• Previous gene therapy (exon skipping, or stop codon read through therapy), cell-based therapy, or any other investigational agents.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

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

Locations

Country	Name	City	State
Japan	Nagoya City University Hospital	Aichi
Japan	National Hospital Organization Nagara Medical Center	Gifu
Japan	Kobe University Hospital	Hyogo
Japan	National Hospital Organization Utano Hospital	Kyoto
Japan	Shinshu University Hospital	Nagano
Japan	National Hospital Organization Niigata National Hospital	Niigata
Japan	National Hospital Organization Toneyama National Hospital	Osaka
Japan	National Hospital Organization Higashisaitama Hospital	Saitama
Japan	National Center of Neurology and Psychiatry	Tokyo
Japan	Tokyo Women's Medical University Hospital	Tokyo
Japan	Tottori University Hospital	Tottori

Sponsors (1)

Lead Sponsor	Collaborator
Taiho Pharmaceutical Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)	The distance the subject can walk as fast as possible in 6 minutes will be evaluated.	baseline, 24 weeks
Secondary	Mean Change From Baseline in Time to Rise From the Floor	The time required for the subject to rise from a supine position on the floor as quickly as possible will be evaluated.	baseline, and 24 weeks
Secondary	Mean Change From Baseline in Time to Walk/Run for 10meters	The time required for the subject to run or walk as quickly as possible a 10 m-wide passage with marks affixed on the floor will be evaluated.	baseline, and 24 weeks
Secondary	Mean Change From Baseline in Time to up and go (TUG)	This test will assess the extent of the subject's composite mobility, including standing up, walking, repositioning the body, and balancing.	baseline, and 24 weeks