Extension Study of NS-089/NCNP-02 in DMD

• ClinicalTrials.gov Identifier: NCT05135663
• Recruitment Status: Active, not recruiting
• First Posted: November 26, 2021
• Last Update Posted: March 3, 2023
• Sponsor: Nippon Shinyaku Co., Ltd.
• Information provided by (Responsible Party): Nippon Shinyaku Co., Ltd.

Tracking Information

• First Submitted Date: October 5, 2021
• First Posted Date: November 26, 2021
• Last Update Posted Date: March 3, 2023
• Actual Study Start Date: June 23, 2021
• Estimated Primary Completion Date: January 31, 2026 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: March 1, 2023): Incidence of adverse events [ Time Frame: Up to Week 247 ]
• Original Primary Outcome Measures (submitted: November 16, 2021): Incidence of adverse events [ Time Frame: Up to Week 102 ]

Current Secondary Outcome Measures (submitted: March 1, 2023)

• Expression of dystrophin protein (Western blot) [ Time Frame: Week 99 ]
• Percentage of exon 44-skipped mRNA of dystrophin [ Time Frame: Week 99 ]
• North Star Ambulatory Assessment (NSAA) [ Time Frame: Up to Week 243 ]
• Time to stand test [ Time Frame: Up to Week 243 ]
• Time to run/walk 10 meters test [ Time Frame: Up to Week 243 ]
• Six-minute walk test/Two-minute walk test [ Time Frame: Up to Week 243 ]
• Timed Up & Go test [ Time Frame: Up to Week 243 ]
• Quantitative muscle strength assessment [ Time Frame: Up to Week 243 ]
• Performance of Upper Limb test [ Time Frame: Up to Week 243 ]
• Change in serum creatine kinase concentration from baseline [ Time Frame: Up to Week 243 ]

Original Secondary Outcome Measures (submitted: November 16, 2021)

• Expression of dystrophin protein (Western blot) [ Time Frame: Week 99 ]
• Percentage of exon 44-skipped mRNA of dystrophin [ Time Frame: Week 99 ]
• North Star Ambulatory Assessment (NSAA) [ Time Frame: Up to Week 99 ]
• Time to stand test [ Time Frame: Up to Week 99 ]
• Time to run/walk 10 meters test [ Time Frame: Up to Week 99 ]
• Six-minute walk test/Two-minute walk test [ Time Frame: Up to Week 99 ]
• Timed Up & Go test [ Time Frame: Up to Week 99 ]
• Quantitative muscle strength assessment [ Time Frame: Up to Week 99 ]
• Performance of Upper Limb test [ Time Frame: Up to Week 99 ]
• Change in serum creatine kinase concentration from baseline [ Time Frame: Up to Week 99 ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Extension Study of NS-089/NCNP-02 in DMD
• Official Title: A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy
• Brief Summary: This is the extension study of NS-089/NCNP-02 (Study NCNP/DMT02), which is designed to assess the safety, tolerability and efficacy of NS-089/NCNP-02 in patients with Duchenne muscular dystrophy (DMD).
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Duchenne Muscular Dystrophy (DMD)

Intervention

• Drug: NS-089/NCNP-02
  The same dose administered in as Part 2 of Study NCNP/DMT02 (40 mg/kg) will be administered once weekly for 216 weeks (The total treatment period is 240 weeks including Part2 of Study NCNP/DMT02).
• Drug: NS-089/NCNP-02
  The same dose administered in as Part 2 of Study NCNP/DMT02 (80 mg/kg) will be administered once weekly for 216 weeks (The total treatment period is 240 weeks including Part2 of Study NCNP/DMT02).

Study Arms

• Experimental: NS-089/NCNP-02 40 mg/kg
  Intervention: Drug: NS-089/NCNP-02
• Experimental: NS-089/NCNP-02 80 mg/kg
  Intervention: Drug: NS-089/NCNP-02

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: November 16, 2021): 6
• Original Actual Enrollment: Same as current
• Estimated Study Completion Date: July 31, 2026
• Estimated Primary Completion Date: January 31, 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

1. Patient completed Study NCNP/DMT02

Exclusion Criteria:

1. Patient had any serious adverse events in Study NCNP/DMT02 that, in the opinion of the Investigator and/or Sponsor, was probably or definitely related to NS-089/NCNP-02 use and precludes safe use of NS-089/NCNP-02 for the patient in this study.
2. Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NCNP/DMT02.
3. Patient took any other investigational drugs after completion of Study NCNP/DMT02.
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.

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Japan

Administrative Information

• NCT Number: NCT05135663
• Other Study ID Numbers: NS089/NCNP02-P2OE; jRCT2031210162 ( Registry Identifier: Japan Registry of Clinical Trials )
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Nippon Shinyaku Co., Ltd.
• Original Responsible Party: Same as current
• Current Study Sponsor: Nippon Shinyaku Co., Ltd.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Nippon Shinyaku Co., Ltd.
• Verification Date: March 2023