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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02752048 |
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Date of registration:
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06/04/2016 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
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Scientific title:
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A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy |
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Date of first enrolment:
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May 2016 |
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Target sample size:
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36 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02752048 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 2
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Countries of recruitment
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Japan
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Contacts
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Name:
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Taiho Pharmaceutical Co., Ltd. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Taiho Pharmaceutical Co., Ltd. |
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Key inclusion & exclusion criteria
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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.
Age minimum:
5 Years
Age maximum:
N/A
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Dystrophy
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Intervention(s)
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Drug: TAS-205
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Drug: Placebo
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Primary Outcome(s)
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Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
[Time Frame: baseline, 24 weeks]
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Secondary Outcome(s)
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Mean Change From Baseline in Time to Rise From the Floor
[Time Frame: baseline, and 24 weeks]
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Mean Change From Baseline in Time to up and go (TUG)
[Time Frame: baseline, and 24 weeks]
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Mean Change From Baseline in Time to Walk/Run for 10meters
[Time Frame: baseline, and 24 weeks]
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Secondary ID(s)
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Taiho10053040
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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