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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 : Completed
First Posted : May 25, 2017
Results First Posted : November 18, 2022
Last Update Posted : December 28, 2022
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 192 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 192 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 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.

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

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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
Actual Primary Completion Date : October 20, 2021
Actual Study Completion Date : November 15, 2021


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 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, 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 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.
Drug: NS-065/NCNP-01
Received during weekly intravenous infusions




Primary Outcome Measures :
  1. Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]
    A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND)

  2. Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]
    A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) Velocity

  3. Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. [ Time Frame: Up to 192 weeks of treatment ]

    For adverse events (AEs) starting in study 201 (NCT02740972) which are not resolved at the time of enrollment into this study 202, any change in outcome or relatedness were reported in study 201.

    For AEs starting in study 201 which increase in severity or becomes serious after enrollment in this study 202, a new AE was reported in this study.

    Treatment-emergent AEs (TEAEs) were summarized by dose level. Coding was done by system organ class and preferred term (using the Medical Dictionary for Regulatory Activities (MedDRA)). Level of severity was assessed using the CTCAE grading system.



Secondary Outcome Measures :
  1. Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]
    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Run/Walk 10 meters test (TTRW)

  2. Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]

    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Run/Walk 10 meters test (TTRW) Velocity.

    The results were converted into velocity (meter/time).


  3. Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]
    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB)

  4. Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]
    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB) Velocity. The results were converted into velocity (meter/time).

  5. Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202 ]

    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): North Star Ambulatory Assessment (NSAA) score

    The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD).

    It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.


  6. Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202 ]
    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Six-Minute Walk Test (6MWT)

  7. Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]

    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Handgrip For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side.

    QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.


  8. Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]

    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Elbow Flexors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side.

    QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.


  9. Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]

    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Elbow Extensors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side.

    QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.


  10. Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]

    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Knee Flexors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side.

    QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.


  11. Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls [ Time Frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 ]

    A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Knee Extensors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side.

    QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.




Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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
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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
  Study Documents (Full-Text)

Documents provided by NS Pharma, Inc.:
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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
Results First Posted: November 18, 2022
Last Update Posted: December 28, 2022
Last Verified: December 2022
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