NS-050/NCNP-03 for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+11 other locations

Age: < 18

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: NS Pharma, Inc.

Must be taking: Glucocorticoids

Must not be taking: Anabolic steroids, Resveratrol

Disqualifiers: Symptomatic cardiomyopathy, Surgery, others

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.

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 the entire study. If you are taking other medications, the protocol does not specify if you need to stop them, but you cannot have taken anabolic steroids or certain other treatments within 3 months before starting the trial.

What safety data exists for NS-050/NCNP-03 in humans?

In a clinical trial for a similar treatment, NS-065/NCNP-01, no severe adverse reactions were observed in patients with Duchenne muscular dystrophy, indicating a favorable safety profile. ^{1 2 3 4 5}

Research Team

Eligibility Criteria

This trial is for boys aged 4 to less than 15 with Duchenne Muscular Dystrophy (DMD) who can walk on their own and have a specific mutation treatable by skipping exon 50. They must be able to stand up quickly without help and have been on a stable dose of glucocorticoids for at least three months.

Inclusion Criteria

I am a boy between 4 and 14 years old.

My DMD is due to a specific mutation that can be treated by skipping exon 50.

I can walk on my own without needing help from devices.

Exclusion Criteria

Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug

I have undergone gene therapy.

I have heart muscle disease with symptoms.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Participants receive NS-050/NCNP-03 or placebo IV infusions once weekly for 2 weeks at each of multiple ascending dose levels

12 weeks

Treatment Part 2

Participants receive NS-050/NCNP-03 IV infusions once weekly for 24 weeks at the dosage selected by the DSMB

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• NS-050/NCNP-03 (Exon Skipping Agent)

Trial Overview The study tests NS-050/NCNP-03, which could potentially skip exon 50 in the dystrophin gene, against a placebo over a period of 12 weeks, followed by an open-label phase where all receive the drug for another 24 weeks.

Participant Groups

Group I: Part 2: NS-050/NCNP-03 Experimental Treatment 1 Intervention

Participants will receive NS-050/NCNP-03 IV infusions once weekly for 24 weeks at the dosage selected by the Data and Safety Monitoring Board (DSMB) at the conclusion of Part 1.

Group II: Part 1: NS-050/NCNP-03 Experimental Treatment 1 Intervention

Participants will be randomized and receive NS-050/NCNP-03 intravenous (IV) infusions once weekly for 2 weeks at each of MAD levels (1.95, 5, 10, 20, 40, and 80 mg/kg).

Group III: Part 1: Placebo Placebo Group 1 Intervention

Participants will be randomized and receive NS-050/NCNP-03 placebo-matching IV infusions once weekly for 2 weeks at each of MAD levels.

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

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Exon-skipping advances for Duchenne muscular dystrophy. [2019]

4. 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]

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

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