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Exon Skipping Agent

NS-050/NCNP-03 for Duchenne Muscular Dystrophy

Phase 1 & 2

Waitlist Available

Research Sponsored by NS Pharma, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male ≥ 4 years and <15 years of age

Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame

Must not have

Having taken any gene therapy

Evidence of symptomatic cardiomyopathy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week13, week25

Summary

"This trial is testing different doses of a drug called NS-050/NCNP-03 in boys with DMD for a total of 36 weeks. The drug will be given once a week to

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Since this is an early-phase trial, exact side effects are not fully known but may include reactions at the injection site, potential kidney or liver issues due to new medication metabolism, muscle pain or weakness beyond usual DMD symptoms.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am a boy between 4 and 14 years old.

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

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

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

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have undergone gene therapy.

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I have heart muscle disease with symptoms.

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week13, week25

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week13, week25

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week13, week25 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Part 1: Amount of Drug Excreted in Urine of NS-050/NCNP-03

Part 1: Area Under the Plasma Concentration Versus Time Curve (AUC) of NS-050/NCNP-03

Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs)

Secondary study objectives

Part 2: North Star Ambulatory Assessment (NSAA) score

Part 2: Performance of Upper Limb (PUL) 2.0. score

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 2: NS-050/NCNP-03Experimental Treatment1 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-03Experimental Treatment1 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: PlaceboPlacebo Group1 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.

0 / 8Eligibility criteria met