A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Drug: TAS-205 [Ambulatory Cohort] [Non-ambulatory Cohort]Drug: Placebo [Ambulatory Cohort] only
- Registration Number
- NCT04587908
- Lead Sponsor
- Taiho Pharmaceutical Co., Ltd.
- Brief Summary
The purpose of this study is to evaluate the efficacy and safety of TAS-205 in patients with Duchenne muscular dystrophy
- Detailed Description
\[Ambulatory Cohort\] The main purpose of this cohort is to assess the efficacy of TAS-205 in patients with Duchenne muscular dystrophy (DMD) compared with placebo as measured by the mean change from baseline to 52 weeks in the time to rise from the floor． Following completion of the treatment period, patients may elect to continue in open-label extension study.
\[Non-ambulatory Cohort\] The main purpose of this cohort is to assess the safety of TAS-205 in patients with DMD by collecting the incidence of adverse events for 52 weeks.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Male
- Target Recruitment
- 104
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description TAS-205 TAS-205 [Ambulatory Cohort] [Non-ambulatory Cohort] - Placebo Placebo [Ambulatory Cohort] only -
- Primary Outcome Measures
Name Time Method Incidence of Adverse Events and Adverse Reactions Week 52 Non-ambulatory Cohort
Mean change from baseline to Week 52 in the time to rise from the floor Baseline to Week 52 of treatment Ambulatory Cohort
- Secondary Outcome Measures
Name Time Method Change from baseline in North Star Ambulatory Assessment (NSAA) Baseline to 52 weeks of treatment Ambulatory Cohort
Change from baseline in Six-minutes Walk Test (6MWT) Baseline to 52 weeks of treatment Ambulatory Cohort
Echocardiography: Measured EF and FS and change from baseline in measured values week 52 Non-ambulatory Cohort
Change from baseline in the Timed Up and Go Test (TUG) Baseline to 52 weeks of treatment Ambulatory Cohort Timed Up and Go Test (TUG) The time required for the subject to stand up from a sitting position on a table (chair), walk to a cone placed 3 m ahead as quickly as possible, and then return to the table will be evaluated.
The time required for the subject to stand up from a sitting position on a table (chair), walk to a cone placed 3 m ahead as quickly as possible, and then return to the table will be evaluated.Change from baseline in grip strength week 52 Non-ambulatory Cohort
Time measured in the time to rise from the floor test, as well as the change from baseline in each measured value Baseline to 52 weeks of treatment Ambulatory Cohort
Measured values of Muscle volume index (MVI), Percent Muscle volume index (%MVI) and skeletal muscle mass in skeletal muscle computed tomography (CT), as well as the change from baseline in each measured value Baseline to 52 weeks of treatment Ambulatory Cohort
Assessment of upper limb function: The Brooke upper extremity scale, measured values of performance of the upper limb (PUL) and change from baseline in measured values week 52 Non-ambulatory Cohort
Pulmonary function tests: measured effort lung capacity (FVC, %FVC), volume in 1 second (Forced Expiratory Volume :FEV1.0), fraction in 1 second (FEV1.0%), and change from baseline (at enrollment) of measured values. week 52 Non-ambulatory Cohort
Trial Locations
- Locations (1)
A site selected by Taiho Pharmaceutical Co., Ltd.
🇯🇵Tokyo, Japan