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

• Status: Completed
• Trial end date: 2017-10-17
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Taiho Pharmaceutical Co., Ltd.

Criteria

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.