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Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04433234
Recruitment Status : Enrolling by invitation
First Posted : June 16, 2020
Last Update Posted : August 3, 2020
Sponsor:
Information provided by (Responsible Party):
Daiichi Sankyo, Inc. ( Daiichi Sankyo Co., Ltd. )

Brief Summary:
This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: DS-5141b Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 8 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Actual Study Start Date : July 8, 2020
Estimated Primary Completion Date : March 31, 2022
Estimated Study Completion Date : March 31, 2022


Arm Intervention/treatment
Experimental: DS-5141b 2.0 mg/kg
Participants who will receive DS-5141b 2.0 mg/kg once weekly.
Drug: DS-5141b
Administered via subcutaneous injection once weekly
Other Name: Renadirsen

Experimental: DS-5141b 6.0 mg/kg
Participants who will receive DS-5141b 6.0 mg/kg once weekly.
Drug: DS-5141b
Administered via subcutaneous injection once weekly
Other Name: Renadirsen




Primary Outcome Measures :
  1. Incidence of Adverse Events (AEs) [ Time Frame: From first injection to after the last injection of DS-5141b in this study (within approximately 2 years) ]
  2. Change in distance walked during 6-minute walk test (6MWT) [ Time Frame: Every 3 months (within approximately 2 years) ]
  3. Change in time to stand (TTSTAND) [ Time Frame: Every 3 months (within approximately 2 years) ]
  4. Change in time in Timed up and go test [ Time Frame: Every 3 months (within approximately 2 years) ]
  5. Change in time in 10-meter Run/Walk test [ Time Frame: Every 3 months (within approximately 2 years) ]
  6. Change in score in the North Star Ambulatory Assessment (NSAA) [ Time Frame: Every 3 months (within approximately 2 years) ]
  7. Change in score in the Performance of Upper Limb (PUL) [ Time Frame: Every 3 months (within approximately 2 years) ]
  8. Change in Left Ventricular Ejection Fraction percentage (LVEF %) [ Time Frame: Every 3 months (within approximately 2 years) ]
  9. Change in in Forced Vital Capacity (FVC) (percent predicted) [ Time Frame: Every 3 months (within approximately 2 years) ]
  10. Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment [ Time Frame: Every 3 months (within approximately 2 years) ]

Secondary Outcome Measures :
  1. Concentration of DS-5141a in plasma [ Time Frame: Every 3 months (within approximately 2 years) ]


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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Has competed a study of DS5141-A-J101

Exclusion Criteria:

  • Significant safety issues in a study of DS5141-A-J101
  • Patient who does not consent to use appropriate contraception
  • Patient not appropriate to participant in the study as determined by the Investigator

Information from the National Library of Medicine

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): NCT04433234


Locations
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Japan
National Center of Neurology and Psychiatry
Tokyo, Kodaira-Shi, Japan, 187-8551
Kobe University Hospital
Hyōgo, Kobe-shi, Japan, 650-0017
Sponsors and Collaborators
Daiichi Sankyo Co., Ltd.
Investigators
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Study Director: Clinical Study Leader Daiichi Sankyo Co., Ltd.
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Responsible Party: Daiichi Sankyo Co., Ltd.
ClinicalTrials.gov Identifier: NCT04433234    
Other Study ID Numbers: DS5141-A-J201
205321 ( Other Identifier: JapicCTI )
First Posted: June 16, 2020    Key Record Dates
Last Update Posted: August 3, 2020
Last Verified: July 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Daiichi Sankyo, Inc. ( Daiichi Sankyo Co., Ltd. ):
Duchenne muscular dystrophy
DMD
Oligonucleotides, antisense
Exon skipping
DS-5141b
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked