A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy(REACH-DMD)

Overview

The purpose of this study is to evaluate the efficacy and safety of TAS-205 in patients with Duchenne muscular dystrophy

Full Title of Study: “A Phase 3, Randomized, Placebo-controlled, Double-blind and Open-label, Extension Study of TAS-205 in Patients With Duchenne Muscular Dystrophy”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: May 2027

Detailed Description

The main purpose of this study 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.

Interventions

  • Drug: TAS-205
    • ・Treatment period:oral administration for 52 weeks, BID after meal
  • Drug: Placebo
    • Observation period:oral administration for 2 weeks, BID after meal Treatment period:oral administration for 52 weeks, BID after meal

Arms, Groups and Cohorts

  • Experimental: TAS-205
  • Placebo Comparator: Placebo

Clinical Trial Outcome Measures

Primary Measures

  • Mean change from baseline to Week 52 in the time to rise from the floor
    • Time Frame: Baseline to Week 52 of treatment

Secondary Measures

  • Time measured in the time to rise from the floor test, as well as the change from baseline in each measured value
    • Time Frame: Baseline to 52 weeks of treatment
  • Change from baseline in the Timed Up and Go Test (TUG)
    • Time Frame: Baseline to 52 weeks of treatment
    • 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.
  • Change from baseline in North Star Ambulatory Assessment (NSAA)
    • Time Frame: Baseline to 52 weeks of treatment
  • Change from baseline in Six-minutes Walk Test (6MWT)
    • Time Frame: Baseline to 52 weeks of treatment
  • 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
    • Time Frame: Baseline to 52 weeks of treatment

Participating in This Clinical Trial

Key Inclusion Criteria:

  • Patients with a diagnosis of dystrophinopathy as determined by a dystrophin genetic test at the time of informed consent, symptoms or signs characteristic to DMD (e.g., proximal muscular weakness, waddling gait, Gower's sign)
  • Patients aged 5 years or more at the time of informed consent
  • Patients who meet all of the following at the time of screening test
  • walk by themselves
  • time to rise from the floor on own is ≥ 3 seconds and <10 seconds
  • Patients who can expect a 6-minute walking test of 350 meters or more
  • If taking oral glucocorticoids no significant change in the total daily or dosing 6 months before enrollment.

Key Exclusion Criteria

  • Patients who have serious concomitant drug hypersensitivity or medical history
  • Patients who have used cyclooxygenase-1 (COX-1) or COX-2 inhibitors, or nonsteroidal anti-inflammatory drugs (NSAIDs) during 7 days before the measurement of time to rise from the floor in the screening period
  • Patients who have incurred an injury (trauma/damage) that may affect muscle strength or motor function within 3 months before enrollment or who have an uncured injury (trauma/damage) that may affect muscle strength or motor function at the enrollment
  • Patients who have received gene-/cell-based therapy or stop-codon readthrough therapy with antisense oligonucleotides
  • Patients who have participated in another clinical trial and received a study drug within 90 days before study drug administration in the present study
  • Patients with a left ventricular ejection fraction (EF) of <40% or left ventricular fractional shortening (FS) of <25% on the cardiac ultrasonography (echocardiography) at observation period

Gender Eligibility: Male

Minimum Age: 5 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Taiho Pharmaceutical Co., Ltd.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Taiho Pharmaceutical Co., Ltd., Study Director, Taiho Pharmaceutical Co., Ltd.
  • Overall Contact(s)
    • Drug Information Center, +81-3-3294-4527, toiawase@taiho.co.jp

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