Phase 1
N=21
A Study of TAS-205 for Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT02246478 ↗Enrolled (actual)
21
Serious AEs
0.0%
Results posted
Jun 2021
Primary outcome: Primary: Incidence of Adverse Events — 0; 3; 1; 1 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- TAS-205 (Drug); Placebo (Drug)
- Age
- Pediatric · 5+ yrs
- Sex
- Male
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
- Primary completion
- Jun 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Adverse Events |
0; 3; 1; 1; 0; 1 | — |
| SECONDARY Peak Plasma Concentration (Cmax) of TAS-205 |
839; 1847; 3202; 1004; 1894; 2635 | — |
| SECONDARY Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205 |
2604; 5776; 9118; 2525; 5281; 6391 | — |
| SECONDARY The Urinary Excretion of PD Marker |
1.05; 0.92; 1.13; 1.26; 1.22; 1.23 | — |
Summary
The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
Eligibility Criteria
Inclusion Criteria
- Able to give an informed consent. If applicable, able to give an informed assent.
- Male and >= 5 years and = 15.0 kg and < 75.0 kg.
- Phenotypic evidence of DMD.
- Able to take tablets.
- If taking oral glucocorticosteroids no significant change in total daily dosage or dosing regimen after enrollment.
- Confirmed the urinary PD marker over its criteria.
- Able to follow the study protocol.
Exclusion Criteria
- Current diagnosis or history of any drug allergy.
- A forced vital capacity (FVC) < 50% of predicted value.
- A left ventricular ejection fraction (EF) < 50% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
- Ongoing immunosuppressive therapy (other than corticosteroids).
- With severe disease such as hepatic disease, kidney disease and others.
- With any systemic allergic disease or any chronic inflammatory disease.
- Treated with any other investigational agents within 90 days.
- Positive reaction in hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test.
Data sourced from ClinicalTrials.gov (NCT02246478). 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.