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

NCT03167255 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: NS-065/NCNP-01-202
• Study Type: interventional
• Target: 16
• Locations: 2 countries
• Sites: 6

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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (3)

• Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls

  A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND)

  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

  A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) Velocity

  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.

  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.

  Up to 192 weeks of treatment

Secondary Outcomes (11)

• Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls

  Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

• Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls

  Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

• Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls

  Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

• Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls

  Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

• Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls

  Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202

Study Arms (2)

NS-065/NCNP-01 40mg/kg

EXPERIMENTAL

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

NS-065/NCNP-01 80mg/kg

EXPERIMENTAL

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

Interventions

NS-065/NCNP-01 DRUG

Received during weekly intravenous infusions

NS-065/NCNP-01 40mg/kg; NS-065/NCNP-01 80mg/kg

Eligibility Criteria

• Age: 4 Years - 10 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• NS Pharma, Inc.: lead
• Nippon Shinyaku Co., Ltd.: collaborator
• Cooperative International Neuromuscular Research Group: collaborator
• Therapeutic Research in Neuromuscular Disorders Solutions: collaborator

Study Sites (6)

UC Davis

Sacramento, California, 95817, United States

Location

Lurie Children's Hospital

Chicago, Illinois, 60611, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Children's Hospital of Richmond at VCU

Richmond, Virginia, 23298, United States

Location

Alberta Children's Hospital

Calgary, Alberta, Canada

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Medical Affairs
• Organization: NS Pharma, Inc.

trialinfo@nspharma.com

+1(201) 986-3860

Study Officials

• Paula R. Clemens, MD

  University of Pittsburgh

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Low Dose cohort of 40 mg/kg and High Dose cohort of 80 mg/kg

Study Record Dates

• First Submitted: May 22, 2017
• First Posted: May 25, 2017
• Study Start: July 6, 2017
• Primary Completion: October 20, 2021
• Study Completion: November 15, 2021
• Last Updated: December 28, 2022
• Results First Posted: November 18, 2022

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (5)