Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
NCT ID: NCT03167255
Last Updated: 2022-12-28
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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COMPLETED
PHASE2
16 participants
INTERVENTIONAL
2017-07-06
2021-11-15
Brief Summary
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Detailed Description
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Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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NS-065/NCNP-01 40mg/kg
Patients receiving 40mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.
NS-065/NCNP-01
Received during weekly intravenous infusions
NS-065/NCNP-01 80mg/kg
Patients receiving 80mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.
NS-065/NCNP-01
Received during weekly intravenous infusions
Interventions
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NS-065/NCNP-01
Received during weekly intravenous infusions
Eligibility Criteria
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Inclusion Criteria
2. Willing and able to comply with scheduled visits, investigational product administration plan, and study procedures.
3. Stable dose of glucocorticoid (GC), and is expected to remain on the stable dose for the duration of the study.
Exclusion Criteria
2. Patient had a treatment which was made for the purpose of dystrophin or its related protein induction after completion of Study NS-065/NCNP-01-201.
3. Patient took any other investigational drugs after completion of Study NS-065/NCNP-01-201.
4. Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.
4 Years
10 Years
MALE
No
Sponsors
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Nippon Shinyaku Co., Ltd.
INDUSTRY
Cooperative International Neuromuscular Research Group
NETWORK
Therapeutic Research in Neuromuscular Disorders Solutions
INDUSTRY
NS Pharma, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Paula R. Clemens, MD
Role: STUDY_CHAIR
University of Pittsburgh
Locations
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UC Davis
Sacramento, California, United States
Lurie Children's Hospital
Chicago, Illinois, United States
Washington University
St Louis, Missouri, United States
Duke University Medical Center
Durham, North Carolina, United States
Children's Hospital of Richmond at VCU
Richmond, Virginia, United States
Alberta Children's Hospital
Calgary, Alberta, Canada
Countries
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Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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NS-065/NCNP-01-202
Identifier Type: -
Identifier Source: org_study_id