Phase 2 N=36 Randomized Quadruple-blind Treatment

A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Bottom Line

View on ClinicalTrials.gov: NCT02752048 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Apr 2020

Primary outcome: Primary: Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD) — -3.5; -7.5; -17.0 meter

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: TAS-205 (Drug); Placebo (Drug)
Age: Pediatric, Adult, Older Adult · 5+ yrs
Sex: Male
Sponsor: Taiho Pharmaceutical Co., Ltd.
Primary completion: May 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)	-3.5; -7.5; -17.0	—
SECONDARY Mean Change From Baseline in Time to Rise From the Floor	4.011; 1.283; 2.633	0.596
SECONDARY Mean Change From Baseline in Time to Walk/Run for 10meters	1.113; 1.025; 0.629	0.382
SECONDARY Mean Change From Baseline in Time to up and go (TUG)	0.594; 0.286; 0.061	0.549

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.

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.

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Data sourced from ClinicalTrials.gov (NCT02752048). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.