A Study of TAS-205 for Duchenne Muscular Dystrophy

NCT02246478 | Completed Phase 1 Results

• 1 other identifier: Taiho10053030
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Peak Plasma Concentration (Cmax) of TAS-205

  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.

• Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205

  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)

• The Urinary Excretion of PD Marker

  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.

Study Arms (4)

Placebo

PLACEBO COMPARATOR

Drug: Placebo

TAS-205 low dose

ACTIVE COMPARATOR

Drug: TAS-205

TAS-205 middle dose

ACTIVE COMPARATOR

Drug: TAS-205

TAS-205 high dose

ACTIVE COMPARATOR

Drug: TAS-205

Interventions

TAS-205 DRUG

* 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

TAS-205 high dose; TAS-205 low dose; TAS-205 middle dose

Placebo DRUG

* 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

Placebo

Eligibility Criteria

• Age: 5 Years - 15 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Taiho Pharmaceutical Co., Ltd.: lead

Study Sites (1)

National Center of Neurology and Psychiatry

Tokyo, 187-8551, Japan

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

TAS-205

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Taiho Pharmaceutical Co., Ltd.
• Organization: Clinical Trial Registration Contact

toiawase@taiho.co.jp

Study Officials

• Taiho Pharmaceutical Co.,Ltd.

  Taiho Pharmaceutical Co., Ltd.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 9, 2014
• First Posted: September 22, 2014
• Study Start: September 1, 2014
• Primary Completion: June 1, 2015
• Study Completion: September 1, 2015
• Last Updated: June 4, 2021
• Results First Posted: June 4, 2021

Record last verified: 2021-05

Locations

JP (1)