A phase 3 study of TAS-205 in patients with Duchenne muscular dystrophy

JRCT ID: jRCT2041200055

Registered date:28/10/2020

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Duchenne Muscular Dystrophy
• Date of first enrollment: 22/12/2020
• Target sample size: 80
• Study type: Interventional

Intervention(s)

Drug: TAS-205 or Placebo
Treatment period :oral administration for 52 weeks, BID after meal

Outcome(s)

Primary Outcome

Mean change from baseline to Week 52 in the time to rise from the floor
[Time Frame :baseline to Week 52 treatment]

Secondary Outcome

1) Time measured in the time to rise from the floor test, as well as the change from baseline in each measured value
Time Flame :baseline to 52 weeks treatment
2) Change from baseline in 10 Meter Walk/run test
Time Flame :baseline to 52 weeks treatment
3) Change from baseline in the Timed Up and Go Test (TCG)
Time Flame :baseline to 52 weeks treatment
4) Change from baseline in North Star Ambulatory Assessment (NSAA)
Time Flame :baseline to 52 weeks treatment
5) Change from baseline in Six Minutes Walk Test (6MWT)
Time Flame :baseline to 52 weeks treatment
6) 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 alteration from baseline in each measured value
Time Flame :baseline to 52 weeks treatment

Key inclusion & exclusion criteria

• Age minimum: >= 5age old
• Age maximum: Not applicable
• Gender: Male

Include criteria

(1) 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, Gowers sign)
(2) Patients aged 5 years or more at the time of informed consent
(3) Patients who meet all of the following at the time of screening test
a) walk by themselves
b) time to rise from the floor on own is 3 seconds and over and under 10 seconds
(4) Patients who can expect a Six Minute Walking test of 350 meters or more
(5) If taking oral glucocorticoids no significant change in the total daily or dosing 6 months before enrollment.

Exclude criteria

1)Patients who have serious concomitant drug hypersensitivity or medical history
2)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
3)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
4)Patients who have received gene-/cell-based therapy or stop-codon readthrough therapy with antisense oligonucleotides
5)Patients who have participated in another clinical trial and received a study drug within 90 days before study drug administration in the present study
6)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

Related Information

• Primary Sponsor: Nasermoaddeli Ali