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Extension Study of NS-089/NCNP-02 in 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: NCT05135663
Recruitment Status : Active, not recruiting
First Posted : November 26, 2021
Last Update Posted : March 3, 2023
Sponsor:
Information provided by (Responsible Party):
Nippon Shinyaku Co., Ltd.

Study Description
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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).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy (DMD) Drug: NS-089/NCNP-02 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy
Actual Study Start Date : June 23, 2021
Estimated Primary Completion Date : January 31, 2026
Estimated Study Completion Date : July 31, 2026

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Arms and Interventions
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Arm Intervention/treatment
Experimental: NS-089/NCNP-02 40 mg/kg 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).
Experimental: NS-089/NCNP-02 80 mg/kg 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).


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of adverse events [ Time Frame: Up to Week 247 ]

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

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
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): NCT05135663


Locations
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Japan
Kagoshima University Hospital
Kagoshima, Japan
National Center of Neurology and Psychiatry
Tokyo, Japan
Sponsors and Collaborators
Nippon Shinyaku Co., Ltd.
More Information
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Responsible Party: Nippon Shinyaku Co., Ltd.
ClinicalTrials.gov Identifier: NCT05135663    
Other Study ID Numbers: NS089/NCNP02-P2OE
jRCT2031210162 ( Registry Identifier: Japan Registry of Clinical Trials )
First Posted: November 26, 2021    Key Record Dates
Last Update Posted: March 3, 2023
Last Verified: March 2023

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