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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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6 December 2021 |
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Main ID: |
NCT03167255 |
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Date of registration:
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22/05/2017 |
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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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Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
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Scientific title:
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A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) |
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Date of first enrolment:
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July 6, 2017 |
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Target sample size:
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16 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT03167255 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Canada
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United States
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Contacts
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Name:
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Paula R. Clemens, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Pittsburgh |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Completed Study NS-065/NCNP-01-201 through Week 25.
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:
1. Serious or severe adverse event in Study NS-065/NCNP-01-201 that precludes safe use of
NS-065/NCNP-01.
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.
Age minimum:
4 Years
Age maximum:
10 Years
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: NS-065/NCNP-01
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Primary Outcome(s)
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Change in Time to Stand (TTSTAND) versus matched historical controls.
[Time Frame: 192 weeks of treatment]
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Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0.
[Time Frame: 192 weeks of treatment]
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Secondary Outcome(s)
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Muscle strength as measured by Quantitative Muscle Testing (QMT) versus matched historical controls.
[Time Frame: 192 weeks of treatment]
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North Star Ambulatory Assessment (NSAA) results versus matched historical controls.
[Time Frame: 192 weeks of treatment]
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Change in Time to Run/Walk 10 meters (TTRW) versus matched historical controls.
[Time Frame: 192 weeks of treatment]
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Change in distance traveled in the Six-Minute Walk Test (6MWT) versus matched historical controls.
[Time Frame: 192 weeks of treatment]
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Change in Time to Climb 4 stairs (TTCLIMB) versus matched historical controls.
[Time Frame: 192 weeks of treatment]
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Secondary ID(s)
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NS-065/NCNP-01-202
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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