NS-089/NCNP-02 for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+10 other locations

Age: < 18

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: NS Pharma, Inc.

Must be taking: Glucocorticoids

Must not be taking: Anabolic steroids, Resveratrol

Disqualifiers: Symptomatic cardiomyopathy, Gene therapy, others

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial tests an IV medication called NS-089/NCNP-02 in boys aged 4 to 14 with a specific type of Duchenne Muscular Dystrophy. The treatment aims to help their bodies make better muscle proteins by skipping over a broken part of their gene. NS-089/NCNP-02 is a new drug utilizing exon-skipping therapy, similar to NS-065/NCNP-01, which targets specific deletions in the dystrophin gene.

Will I have to stop taking my current medications?

The trial requires that participants stay on a stable dose of glucocorticoids (a type of steroid medication) for at least 3 months before and during the study. If you are taking anabolic steroids, resveratrol, adenosine triphosphate, or any investigational drugs, you must stop these at least 3 months before starting the trial.

What safety data exists for NS-089/NCNP-02 in humans?

NS-089/NCNP-02 has been tested in a phase 1/2 clinical trial for Duchenne muscular dystrophy, focusing on safety and how the body processes the drug. While specific safety results for NS-089/NCNP-02 are not detailed, a similar drug, NS-065/NCNP-01, showed no severe adverse reactions in a trial, suggesting a potentially favorable safety profile. ^{1 2 3 4 5}

Research Team

Eligibility Criteria

This trial is for boys aged 4 to under 15 with Duchenne Muscular Dystrophy (DMD) who can stand up quickly without help and walk on their own. They must have a specific mutation in the dystrophin gene and be on a stable dose of glucocorticoids for at least three months.

Inclusion Criteria

My DMD is due to a specific mutation that can be treated by skipping exon 44.

I can stand up from sitting without help in less than 7 seconds.

I have been on a stable dose of glucocorticoid for at least 3 months.

Exclusion Criteria

Previously treated in an interventional study of NS-089/NCNP-02

I haven't had surgery in the last 3 months and don't plan to during the study.

I haven't taken steroids, resveratrol, or ATP products in the last 3 months.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Participants receive NS-089/NCNP-02 via weekly IV infusion

13 weeks

13 visits (in-person)

Treatment Part 2

Continuation of NS-089/NCNP-02 treatment with additional participants

12 weeks

12 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• NS-089/NCNP-02 (Exon Skipping Agent)

Trial Overview The study tests NS-089/NCNP-02, given weekly through an IV, aiming to skip exon 44 in the dystrophin gene. It's divided into two parts: an initial phase with six participants followed by a second part including another fourteen boys.

Participant Groups

Group I: NS-089/NCNP-02 Experimental Treatment 1 Intervention

Experimental: NS-089/NCNP-02 NS-089/NCNP-02 solution for infusion (Cohort 1) NS-089/NCNP-02 solution for infusion (Cohort 2)

NS-089/NCNP-02 is already approved in Japan for the following indications:

Approved in Japan as NS-089/NCNP-02 for:

• Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping

Who Is Running the Clinical Trial?

NS Pharma, Inc.

Lead Sponsor

Trials

14

Recruited

460+

Nippon Shinyaku Co., Ltd.

Industry Sponsor

Trials

14

Recruited

500+

References

1. United States pubmed.ncbi.nlm.nih.gov

Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Systemic administration of the antisense oligonucleotide NS-089/NCNP-02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/II clinical trial. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Safety pharmacology and genotoxicity evaluation of AVI-4658. [2016]