Clinical trial NCT02246478: A Study of TAS-205 for Duchenne Muscular Dystrophy
Official title: A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: Taiho Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2014-09-22
Enrollment: 23
Sex: MALE
Minimum age (years): 5
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of Adverse Events
timeFrame: From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)
Secondary outcomes:
measure: Peak Plasma Concentration (Cmax) of TAS-205
timeFrame: Single-dose phase: immediately before dosing, 0, 0.5, 1, 2, 4, 8, 24, 48 hours post-dose, Multiple-dose phase: Days 1 and 7, immediately before morning dose, 0.5, 1, 2, 4, and 8 hours post-dose and Day 4, immediately before morning dose.
measure: Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205
timeFrame: Administration period (ie. single-dose phase: from single administration day to 48 hours after the administration, multiple-dose phase: from the first administration day to 8 hours after the last administration)
measure: The Urinary Excretion of PD Marker
timeFrame: Single-dose: Day -1 before administration, 0-24 hr post-dose, and 24-48 hr post-dose, Multiple-doses: Day -1 before administration, 0 hr after administration on Day 1 and 4 to the following day (Day 2 and 5), and 0-24 hr after administration on Day 7.
Other outcomes:
Locations:
city: Tokyo
country: Japan
facility: National Center of Neurology and Psychiatry
Study officials:
affiliation: Taiho Pharmaceutical Co., Ltd.
name: Taiho Pharmaceutical Co.,Ltd.
role: STUDY_DIRECTOR
References: