Clinical trial NCT02752048: A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
Official title: A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: Taiho Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2016-04-26
Enrollment: 36
Sex: MALE
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
timeFrame: baseline, 24 weeks
Secondary outcomes:
measure: Mean Change From Baseline in Time to Rise From the Floor
timeFrame: baseline, and 24 weeks
measure: Mean Change From Baseline in Time to Walk/Run for 10meters
timeFrame: baseline, and 24 weeks
measure: Mean Change From Baseline in Time to up and go (TUG)
timeFrame: baseline, and 24 weeks
Other outcomes:
Locations:
city: Aichi
country: Japan
facility: Nagoya City University Hospital
city: Gifu
country: Japan
facility: National Hospital Organization Nagara Medical Center
city: Hyogo
country: Japan
facility: Kobe University Hospital
city: Kyoto
country: Japan
facility: National Hospital Organization Utano Hospital
city: Nagano
country: Japan
facility: Shinshu University Hospital
city: Niigata
country: Japan
facility: National Hospital Organization Niigata National Hospital
city: Osaka
country: Japan
facility: National Hospital Organization Toneyama National Hospital
city: Saitama
country: Japan
facility: National Hospital Organization Higashisaitama Hospital
city: Tokyo
country: Japan
facility: Tokyo Women's Medical University Hospital
city: Tokyo
country: Japan
facility: National Center of Neurology and Psychiatry
city: Tottori
country: Japan
facility: Tottori University Hospital
Study officials:
affiliation: Taiho Pharmaceutical Co., Ltd.
name: Taiho Pharmaceutical Co., Ltd.
role: STUDY_DIRECTOR
References:
citation: 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.