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

NCT ID: NCT02752048

Last Updated: 2020-04-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-05-31
• Study Completion Date: 2017-10-17

Brief Summary

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.

Detailed 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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

TAS-205（Low dose group）

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.

Group Type: EXPERIMENTAL

TAS-205

Intervention Type: DRUG

2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal

TAS-205（High dose group）

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.

Group Type: EXPERIMENTAL

TAS-205

Intervention Type: DRUG

2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal

Placebo

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

1 group: Placebo group. Oral administration for 24 weeks, BID after meal

Interventions

TAS-205

2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal

Intervention Type: DRUG

Placebo

1 group: Placebo group. Oral administration for 24 weeks, BID after meal

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 5 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Taiho Pharmaceutical Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Taiho Pharmaceutical Co., Ltd.

Role: STUDY_DIRECTOR

Taiho Pharmaceutical Co., Ltd.

Locations

Nagoya City University Hospital

Aichi, , Japan

National Hospital Organization Nagara Medical Center

Gifu, , Japan

Kobe University Hospital

Hyōgo, , Japan

National Hospital Organization Utano Hospital

Kyoto, , Japan

Shinshu University Hospital

Nagano, , Japan

National Hospital Organization Niigata National Hospital

Niigata, , Japan

National Hospital Organization Toneyama National Hospital

Osaka, , Japan

National Hospital Organization Higashisaitama Hospital

Saitama, , Japan

Tokyo Women's Medical University Hospital

Tokyo, , Japan

National Center of Neurology and Psychiatry

Tokyo, , Japan

Tottori University Hospital

Tottori, , Japan

Countries

Japan

References

Komaki H, Maegaki Y, Matsumura T, Shiraishi K, Awano H, Nakamura A, Kinoshita S, Ogata K, Ishigaki K, Saitoh S, Funato M, Kuru S, Nakayama T, Iwata Y, Yajima H, Takeda S. Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy. Ann Clin Transl Neurol. 2020 Feb;7(2):181-190. doi: 10.1002/acn3.50978. Epub 2020 Jan 20.

Reference Type: DERIVED

PMID: 31957953 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Taiho10053040

Identifier Type: -

Identifier Source: org_study_id