Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT02667483|
Recruitment Status : Completed
First Posted : January 29, 2016
Last Update Posted : December 16, 2020
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|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: DS-5141b||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||7 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I/II Study of DS-5141b: Open-label Study of DS-5141b in Patients With Duchenne Muscular Dystrophy|
|Actual Study Start Date :||October 2015|
|Actual Primary Completion Date :||October 20, 2020|
|Actual Study Completion Date :||October 20, 2020|
DS-5141b, Subcutaneous injection
Part 1: DS-5141b will be injected subcutaneously once a week for 2 weeks at the following dose levels. Dose escalation will be performed. DS-5141b will be administered at dose levels 1 and 3 in Cohort 1 and at dose levels 2 and 4 in Cohort 2.
Part 2: Two doses of DS-5141b will be selected based on the results obtained in Part 1. Each selected dose will be administered subcutaneously once a week for 12 weeks.
Part 2-Extension: Two doses, 2.0 mg/kg or 6.0 mg/kg, of DS-5141b will be administered subcutaneously once a week for 48 weeks.
DS-5141b, Subcutaneous injection
- Number of participants with treatment-emergent adverse events (TEAEs) by the end of the trial [ Time Frame: 48 Weeks of Part 2-Extension ]TEAEs are adverse events (including clinically significant laboratory values) temporally associated with use of DS-5141b, whether or not attributable to the product.
- Maximum concentration (Cmax) of DS-5141b [ Time Frame: Week 48 of Part 2-Extension ]
- Area under the curve (AUC) for DS-5141b [ Time Frame: Week 48 of Part 2-Extension ]
- Time to maximum concentration (Tmax) of DS-5141b [ Time Frame: Week 48 of Part 2-Extension ]
- Half-life (T1/2) of DS-5141b [ Time Frame: Week 48 of Part 2-Extension ]
- Dystrophin protein expression in muscle tissue [ Time Frame: Week 48 of Part 2-Extension ]
- Production of exon 45-skipped dystrophin mRNA in muscle tissue [ Time Frame: Week 48 of Part 2-Extension ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||5 Years to 10 Years (Child)|
|Sexes Eligible for Study:||Male|
|Accepts Healthy Volunteers:||No|
- Confirmation of out-of-frame deletion(s) that could be corrected by dystrophin gene exon 45 skipping.
- Intact muscles of adequate quality for biopsy to allow evaluation of the efficacy of the study drug.
- Boys aged from 5 years to <11 years.
- Patients able to walk at least 325 meters in the 6-minutes walk test.
- Glucocorticoid-naive patients, or patients who have used glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.
- A genetic mutation that can not be expected the expression of dystrophin protein by dystrophin gene exon 45 skipping.
- A concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function.
- Current or history of severe disorder.
- Left ventricular ejection fraction (LEVF) <55%.
- Corrected QT interval (QTc) >0.45 sec.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02667483
|Kobe University Hospital|
|Hyogo, Kobe-shi, Japan, 650-0017|
|National Center of Neurology and Psychiatry|
|Tokyo, Kodaira-shi, Japan, 187-8551|
|Study Director:||Global Clinical Leader||Daiichi Sankyo, Inc.|
|Responsible Party:||Daiichi Sankyo Co., Ltd.|
|Other Study ID Numbers:||
153072 ( Registry Identifier: JAPIC CTI )
|First Posted:||January 29, 2016 Key Record Dates|
|Last Update Posted:||December 16, 2020|
|Last Verified:||December 2020|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Duchenne muscular dystrophy
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked