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Clinical trial

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)

ID
Name
NS-065/NCNP-01-202
Description
This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.
Trial arms
Trial start
2017-07-06
Estimated PCD
2021-10-20
Trial end
2021-11-15
Status
Completed
Phase
Early phase I
Treatment
NS-065/NCNP-01
Received during weekly intravenous infusions
Arms:
NS-065/NCNP-01 40mg/kg, NS-065/NCNP-01 80mg/kg
Size
16
Primary endpoint
Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
Up to 192 weeks of treatment
Eligibility criteria
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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Low Dose cohort of 40 mg/kg and High Dose cohort of 80 mg/kg', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 16, 'type': 'ACTUAL'}}
Updated at
2022-12-28

1 organization

1 product

1 indication

Product
NS-065/NCNP-01
Indication
Duchenne muscular dystrophy
Organization
NS Pharma