Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
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| ClinicalTrials.gov Identifier: NCT03167255 |
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Recruitment Status :
Enrolling by invitation
First Posted : May 25, 2017
Last Update Posted : December 22, 2017
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Sponsor:
NS Pharma, Inc.
Collaborators:
Nippon Shinyaku Co., Ltd.
Cooperative International Neuromuscular Research Group (CINRG)
Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
Information provided by (Responsible Party):
NS Pharma, Inc.
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Brief Summary:
This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 72 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Duchenne Muscular Dystrophy | Drug: NS-065/NCNP-01 | Phase 2 |
This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 72 weeks to boys with DMD who complete Study NS-065/NCNP-01-201. This study will evaluate the safety, tolerability, and clinical efficacy of NS-065/NCNP-01 at dose levels of up to 80 mg/kg/week administered by weekly IV infusion over an additional treatment period of 72 weeks.
Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 16 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Parallel Assignment |
| Intervention Model Description: | Low Dose cohort of 40 mg/kg and High Dose cohort of 80 mg/kg |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | 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) |
| Actual Study Start Date : | July 6, 2017 |
| Estimated Primary Completion Date : | July 2019 |
| Estimated Study Completion Date : | July 2019 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Duchenne and Becker muscular dystrophy
MedlinePlus related topics:
Muscular Dystrophy
| Arm | Intervention/treatment |
|---|---|
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Experimental: 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 72 weeks
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Drug: NS-065/NCNP-01
Received during weekly intravenous infusions |
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Experimental: 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 72 weeks
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Drug: NS-065/NCNP-01
Received during weekly intravenous infusions |
Primary Outcome Measures :
- Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0. [ Time Frame: 72 weeks of treatment ]
- Change in Time to Stand (TTSTAND) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
Secondary Outcome Measures :
- Change in Time to Run/Walk 10 meters (TTRW) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
- Change in Time to Climb 4 stairs (TTCLIMB) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
- North Star Ambulatory Assessment (NSAA) results versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
- Change in distance traveled in the Six-Minute Walk Test (6MWT) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
- Muscle strength as measured by Quantitative Muscle Testing (QMT) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
Other Outcome Measures:
- Change from baseline on pharmacodynamic (PD) biomarkers. [ Time Frame: 72 weeks of treatment ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | 4 Years to 10 Years (Child) |
| Sexes Eligible for Study: | Male |
| Gender Based Eligibility: | Yes |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Completed Study NS-065/NCNP-01-201 through Week 25.
- Willing and able to comply with scheduled visits, investigational product administration plan, and study procedures.
- Stable dose of glucocorticoid (GC), and is expected to remain on the stable dose for the duration of the study.
Exclusion Criteria:
- Serious or severe adverse event in Study NS-065/NCNP-01-201 that precludes safe use of NS-065/NCNP-01.
- 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.
- Patient took any other investigational drugs after completion of Study NS-065/NCNP-01-201.
- Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.
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): NCT03167255
Locations
| United States, California | |
| UC Davis | |
| Sacramento, California, United States, 95817 | |
| United States, Illinois | |
| Lurie Children's Hospital | |
| Chicago, Illinois, United States, 60611 | |
| United States, Missouri | |
| Washington University | |
| Saint Louis, Missouri, United States, 63110 | |
| United States, North Carolina | |
| Duke University Medical Center | |
| Durham, North Carolina, United States, 27710 | |
| United States, Virginia | |
| Children's Hospital of Richmond at VCU | |
| Richmond, Virginia, United States, 23298 | |
| Canada, Alberta | |
| Alberta Children's Hospital | |
| Calgary, Alberta, Canada | |
Sponsors and Collaborators
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.
Cooperative International Neuromuscular Research Group (CINRG)
Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
Investigators
| Study Chair: | Paula R. Clemens, MD | University of Pittsburgh |
| Responsible Party: | NS Pharma, Inc. |
| ClinicalTrials.gov Identifier: | NCT03167255 History of Changes |
| Other Study ID Numbers: |
NS-065/NCNP-01-202 |
| First Posted: | May 25, 2017 Key Record Dates |
| Last Update Posted: | December 22, 2017 |
| Last Verified: | December 2017 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes | |
| Studies a U.S. FDA-regulated Device Product: | No | |
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 |