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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 day1, week4 for each dose for part 1, day1 and week24 for part 2] urinary excretion of ns-089/ncnp-02
Awards & highlights
Study Summary
This trial studies an IV drug to treat Duchenne Muscular Dystrophy in boys aged 4-15 and follows their progress over two parts.
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.Check my eligibility
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.See study design
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 ~ day1, week4 for each dose for part 1, day1 and week24 for part 2] urinary excretion of ns-089/ncnp-02
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day1, week4 for each dose for part 1, day1 and week24 for part 2] urinary excretion of ns-089/ncnp-02
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Adverse Event and Adverse Drug Reaction
Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot).
Plasma pharmacokinetic (PK) parameters
+1 moreSecondary outcome measures
Change from baseline in percentage of exon 44-skipped mRNA of skeletal muscle dystrophin
Change from baseline in skeletal muscle dystrophin protein by mass spectrometry.
Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining.
+8 moreTrial 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
Find a Location
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
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My DMD is due to a specific mutation that can be treated by skipping exon 44.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.I weigh less than 20 kg.I have heart muscle disease with symptoms.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.I have received gene therapy or exon-skipping treatment.I am a boy between 4 and 14 years old.I can walk on my own without needing help from devices.
Research Study Groups:
This trial has the following groups:- Group 1: NS-089/NCNP-02
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Could I potentially join this research endeavor?
"Eligibility criteria for this research requires that participants have duchenne muscular dystrophy and be between 4 and 14 years of age. The total recruitment goal is 20 individuals."
Answered by AI
Has the FDA authorized NS-089/NCNP-02 for public consumption?
"Although evidence of efficacy is still lacking, prior clinical data suggests that NS-089/NCNP-02 has a safety score of 2."
Answered by AI
Have recruitment efforts for this trial been ongoing?
"Clinicaltrials.gov does not list this trial as presently recruiting, with the initial post and most recent edit both occuring on August 10th 2023. However, there are 91 alternative studies that are actively enrolling patients at the moment."
Answered by AI
Does this trial accept individuals aged 55 and above?
"This medical investigation will only accept children between 4 and 14 years old. There are 63 studies for participants younger than 18, and 52 trials catering to those over the age of 65."
Answered by AI
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