NCT02246478

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

Completed Phase 1 Results posted Last updated 11 May 2021

What this trial tests

Phase 1 trial testing TAS-205 in Duchenne Muscular Dystrophy in 23 participants. Completed in 1 September 2015.

Timeline

1 September 2014

Primary endpoint
1 June 2015

1 September 2015

Quick facts

Lead sponsor	Taiho Pharmaceutical Co., Ltd.
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	23
Start date	1 September 2014
Primary completion	1 June 2015
Estimated completion	1 September 2015
Sites	1 location across Japan

Drugs / interventions tested

• TAS-205 — full drug profile →
• Placebo

Conditions studied

• Duchenne Muscular Dystrophy — all drugs for Duchenne Muscular Dystrophy →

Sponsor

Taiho Pharmaceutical Co., Ltd. — full company profile →

Who can join

Adults 5 to 15, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Incidence of Adverse Events
  Time frame: From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)
  Source Vocabulary Name for Table Default: CTCAE (4.03)

Sponsor's own description

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

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials.
   Shimizu-Motohashi Y, Miyatake S, Komaki H, Takeda S, et al · · 2016 · cited 68× · PMID 27398133
2. Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future.
   Łoboda A, Dulak J. · · 2020 · cited 57× · PMID 32691346 · DOI 10.1007/s43440-020-00134-x
3. Progress and prospects of gene therapy clinical trials for the muscular dystrophies.
   Bengtsson NE, Seto JT, Hall JK, Chamberlain JS, et al · · 2016 · cited 48× · PMID 26450518 · DOI 10.1093/hmg/ddv420
4. "The Social Network" and Muscular Dystrophies: The Lesson Learnt about the Niche Environment as a Target for Therapeutic Strategies.
   Cappellari O, Mantuano P, De Luca A. · · 2020 · cited 32× · PMID 32660168 · DOI 10.3390/cells9071659
5. A phase I study of TAS-205 in patients with Duchenne muscular dystrophy.
   Takeshita E, Komaki H, Shimizu-Motohashi Y, Ishiyama A, et al · · 2018 · cited 20× · PMID 30480028 · DOI 10.1002/acn3.651
6. Targeted genetic therapies for inherited disorders that affect both cardiac and skeletal muscle.
   Psaras Y, Toepfer CN. · · 2024 · cited 3× · PMID 38095849 · DOI 10.1113/ep090436
7. Urinary prostaglandin D<sub>2</sub> and E<sub>2</sub> metabolites are elevated with disease severity in patients with Fukuyama congenital muscular dystrophy.
   Ishigaki K, Takeuchi A, Taniguchi-Ikeda M, Sato T, et al · · 2025 · cited 1× · PMID 40011677 · DOI 10.1038/s41598-025-91539-2