What side effects are associated with this type of intervention?

The most common side effects of exon-skipping treatments for Duchenne Muscular Dystrophy, such as eteplirsen, viltolarsen, golodirsen, and casimersen, include upper respiratory tract infections, injection site reactions, cough, fever, headache, and gastrointestinal issues like vomiting and nausea. Other reported side effects are balance disorder, contact dermatitis, rash, and musculoskeletal pain. Renal toxicity has also been observed with some antisense oligonucleotides, necessitating regular monitoring of kidney function during treatment.

What prior FDA approvals exist?

The FDA has granted accelerated approval to several exon skipping drugs for the treatment of Duchenne Muscular Dystrophy (DMD). These include eteplirsen, which targets exon 51 and is suitable for approximately 13% of DMD patients, golodirsen, which targets exon 53 and is suitable for about 8% of patients, and viltolarsen, also targeting exon 53. These drugs work by skipping specific exons in the DMD gene to allow the production of a shorter but functional dystrophin protein. While these treatments have shown an increase in dystrophin production, their clinical benefits in terms of improved motor function are still being evaluated in ongoing trials.

What other types of research exist?

Promising alternatives to NS-089/NCNP-02 for DMD patients include: 1) Eteplirsen (Exon 51 Skipping) - Shown to increase dystrophin production and improve walking distance in clinical trials. 2) Viltolarsen (Exon 53 Skipping) - Demonstrated significant increase in dystrophin levels and improvement in timed function tests. 3) Casimersen (Exon 45 Skipping) - FDA-approved based on increased dystrophin production, with ongoing trials to confirm clinical benefits. 4) Ataluren - Targets nonsense mutations by promoting ribosomal read-through, beneficial for 10-15% of DMD cases with such mutations.

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Exon Skipping Agent

NS-089/NCNP-02 for Duchenne Muscular Dystrophy

Phase 2

Recruiting

Research Sponsored by NS Pharma, Inc.

Eligibility Criteria Checklist

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

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week13, week25

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not specified here but could include reactions related to IV infusion, effects from altering genetic expression, or complications due to weakened muscles associated with DMD.

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.

Secondary study objectives

North Star Ambulatory Assessment (NSAA) score

Performance of Upper Limb (PUL) 2.0. score

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: NS-089/NCNP-02Experimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

NS-089/NCNP-02

2019

Completed Phase 2

~10

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Duchenne Muscular Dystrophy (DMD) include genetic therapies such as exon skipping drugs. These treatments work by skipping over specific exons in the DMD gene, allowing the production of a shorter but functional dystrophin protein. For example, drugs like eteplirsen, golodirsen, and viltolarsen target exons 51, 53, and 53 respectively, while NS-089/NCNP-02 targets exon 44. This mechanism is crucial for DMD patients because it helps restore some level of dystrophin production, which is essential for muscle function and stability, potentially slowing disease progression and improving quality of life.