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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03167255
Recruitment Status : Active, not recruiting
First Posted : May 25, 2017
Last Update Posted : April 22, 2020
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.

Study Description
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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 168 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 168 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 168 weeks or until NS-065/NCNP-01 is commercially available, whichever is earlier.

Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual 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 : May 2021
Estimated Study Completion Date : August 2021

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Arms and Interventions
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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 168 weeks or until NS-065/NCNP-01 is commercially available, whichever is earlier.
 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 168 weeks or until NS-065/NCNP-01 is commercially available, whichever is earlier.
 Drug: NS-065/NCNP-01
Received during weekly intravenous infusions


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0. [ Time Frame: 168 weeks of treatment ]
  2. Change in Time to Stand (TTSTAND) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]

Secondary Outcome Measures :
  1. Change in Time to Run/Walk 10 meters (TTRW) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
  2. Change in Time to Climb 4 stairs (TTCLIMB) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
  3. North Star Ambulatory Assessment (NSAA) results versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
  4. Change in distance traveled in the Six-Minute Walk Test (6MWT) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]
  5. Muscle strength as measured by Quantitative Muscle Testing (QMT) versus matched historical controls. [ Time Frame: 168 weeks of treatment ]

Other Outcome Measures:
  1. Change from baseline on pharmacodynamic (PD) biomarkers. [ Time Frame: 168 weeks of treatment ]

Eligibility Criteria
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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.
Contacts and Locations
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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
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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
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Study Chair: Paula R. Clemens, MD University of Pittsburgh
More Information
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Responsible Party: NS Pharma, Inc.
ClinicalTrials.gov Identifier: NCT03167255    
Other Study ID Numbers: NS-065/NCNP-01-202
First Posted: May 25, 2017    Key Record Dates
Last Update Posted: April 22, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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