A Study of TAS-205 for Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Clinical Trial

Official title:

A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02246478
• Other study ID #: Taiho10053030
• Status: Completed
• Phase: Phase 1
• Start date: September 2014
• Est. completion date: September 2015

Study information

• Verified date: May 2021
• 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 safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 23
• Est. completion date: September 2015
• Est. primary completion date: June 2015
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years to 15 Years

Eligibility

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.

Related Conditions & MeSH terms

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

Intervention

Drug:
• TAS-205
Single-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step, single oral administration after meals Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals
• Placebo
Single-dose phase: 3steps (low dose, middle dose or high dose group), 2 patients/step, single oral administration after meals Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 2 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals

Locations

Country	Name	City	State
Japan	National Center of Neurology and Psychiatry	Tokyo

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	Incidence of Adverse Events	Source Vocabulary Name for Table Default: CTCAE (4.03)	From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)
Secondary	Peak Plasma Concentration (Cmax) of TAS-205	Due to inspection missing, some data were not analyzed.	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.
Secondary	Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205	Due to inspection missing, some data were not analyzed.	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)
Secondary	The Urinary Excretion of PD Marker	Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed.	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.