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Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov Identifier: NCT03167255
Recruitment Status : Enrolling by invitation
First Posted : May 25, 2017
Last Update Posted : December 22, 2017
Sponsor:
Collaborators:
Nippon Shinyaku Co., Ltd.
Cooperative International Neuromuscular Research Group
Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
Information provided by (Responsible Party):
NS Pharma, Inc.

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

Detailed Description:

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


Arm Intervention/treatment
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
Drug: NS-065/NCNP-01
Received during weekly intravenous infusions

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
Drug: NS-065/NCNP-01
Received during weekly intravenous infusions




Primary Outcome Measures :
  1. Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0. [ Time Frame: 72 weeks of treatment ]
  2. Change in Time to Stand (TTSTAND) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]

Secondary Outcome Measures :
  1. Change in Time to Run/Walk 10 meters (TTRW) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
  2. Change in Time to Climb 4 stairs (TTCLIMB) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
  3. North Star Ambulatory Assessment (NSAA) results versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
  4. Change in distance traveled in the Six-Minute Walk Test (6MWT) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]
  5. Muscle strength as measured by Quantitative Muscle Testing (QMT) versus matched historical controls. [ Time Frame: 72 weeks of treatment ]

Other Outcome Measures:
  1. Change from baseline on pharmacodynamic (PD) biomarkers. [ Time Frame: 72 weeks of treatment ]


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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:

  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.

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
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:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked