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

Taiho Pharma

Status and phase: Completed; Phase 2

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: TAS-205; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02752048

Taiho10053040

Details and patient eligibility

About

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.

Full 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.

Enrollment: 36 patients

Sex: Male

Ages: 5+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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36 participants in 3 patient groups, including a placebo group

TAS-205（Low dose group）

Experimental group

Description:

Low dose group：Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (6.67-13.33 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner.

Treatment:

Drug: TAS-205

TAS-205（High dose group）

Experimental group

Description:

High dose group: Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (13.33-26.67 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner.

Treatment:

Drug: TAS-205

Placebo

Placebo Comparator group

Description:

Placebo group: Oral administration of tablets for 24 weeks, BID after meal

Treatment:

Drug: Placebo