A Study of TAS-205 for Duchenne Muscular Dystrophy

Taiho Pharma

Status and phase: Completed; Phase 1

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: Placebo; Drug: TAS-205

Study type: Interventional

Funder types: Industry

Identifiers

NCT02246478

Taiho10053030

Details and patient eligibility

About

The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.

Full description

Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3500 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 loss of upper body function. The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 after single and multiple doses in DMD patients. It is also evaluated if TAS-205 affects the urinary excretion of pharmacodynamic (PD) marker in DMD patients.

Enrollment: 23 patients

Sex: Male

Ages: 5 to 15 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Able to give an informed consent. If applicable, able to give an informed assent.
• Male and >= 5 years and < 16 years of age.
• Bodyweight of >= 15.0 kg and < 75.0 kg.
• Phenotypic evidence of DMD.
• Able to take tablets.
• If taking oral glucocorticosteroids no significant change in total daily dosage or dosing regimen after enrollment.
• Confirmed the urinary PD marker over its criteria.
• Able to follow the study protocol.

Exclusion criteria

• Current diagnosis or history of any drug allergy.
• A forced vital capacity (FVC) < 50% of predicted value.
• A left ventricular ejection fraction (EF) < 50% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
• Ongoing immunosuppressive therapy (other than corticosteroids).
• With severe disease such as hepatic disease, kidney disease and others.
• With any systemic allergic disease or any chronic inflammatory disease.
• Treated with any other investigational agents within 90 days.
• Positive reaction in hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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23 participants in 4 patient groups, including a placebo group

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo

TAS-205 low dose

Active Comparator group

Treatment:

Drug: TAS-205

TAS-205 middle dose

Active Comparator group

Treatment:

Drug: TAS-205

TAS-205 high dose

Active Comparator group

Treatment:

Drug: TAS-205