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Exon Skipping Agent

Part 1: NS-050/NCNP-03 for Duchenne Muscular Dystrophy

Q: Are there any available positions for participants in this research study?

According to details on clinicaltrials.gov, the current investigation is not actively seeking subjects. The trial was initially posted on July 1st, 2024, with the most recent update occurring on February 13th, 2024. Although this specific study is not enrolling participants currently, there are a total of 98 other trials presently recruiting eligible candidates.

Q: Is it possible for me to be enrolled as a participant in this medical study?

The trial aims to recruit 20 male participants aged between 4 and 14 with <a href="https://www.withpower.com/clinical-trials/duchenne-muscular-dystrophy">Duchenne muscular dystrophy</a>. Crucially, candidates must satisfy the subsequent conditions: Being a male individual aged ≥ 4 years and &#x3C;15 years;, Having confirmed DMD mutations in the dystrophin gene that are responsive to exon 50 skipping for restoring normal dystrophin mRNA reading frame;, Capable of independent ambulation without any walking <a href="https://www.withpower.com/clinical-trials/aids">aids</a>;, Achieving completion of TTSTAND within &#x3C;7 seconds unaided;, Maintaining a consistent glucocorticoid dose for at least three months with an anticipated stable dosage throughout the study period., Additional eligibility

Q: Are individuals above the age of 50 eligible to participate in this research project?

This investigation is seeking participants aged between 4 and 14 years old.

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week13, week25

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Select...

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

Select...

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

Select...

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

Select...

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

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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)

+1 more

Secondary outcome measures

Part 2: Change from baseline in percentage of exon 50-skipped mRNA of skeletal muscle dystrophin

Part 2: Change from baseline in skeletal muscle dystrophin protein by mass spectrometry

Part 2: Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining

+8 more

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 / 5Eligibility criteria met

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Who is running the clinical trial?

NS Pharma, Inc.Lead Sponsor

13 Previous Clinical Trials

554 Total Patients Enrolled

Nippon Shinyaku Co., Ltd.Industry Sponsor

12 Previous Clinical Trials

581 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any available positions for participants in this research study?

"According to details on clinicaltrials.gov, the current investigation is not actively seeking subjects. The trial was initially posted on July 1st, 2024, with the most recent update occurring on February 13th, 2024. Although this specific study is not enrolling participants currently, there are a total of 98 other trials presently recruiting eligible candidates."

Answered by AI

Is it possible for me to be enrolled as a participant in this medical study?

"The trial aims to recruit 20 male participants aged between 4 and 14 with Duchenne muscular dystrophy. Crucially, candidates must satisfy the subsequent conditions: Being a male individual aged ≥ 4 years and <15 years;, Having confirmed DMD mutations in the dystrophin gene that are responsive to exon 50 skipping for restoring normal dystrophin mRNA reading frame;, Capable of independent ambulation without any walking aids;, Achieving completion of TTSTAND within <7 seconds unaided;, Maintaining a consistent glucocorticoid dose for at least three months with an anticipated stable dosage throughout the study period., Additional eligibility"

Answered by AI