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NCT05996003RECRUITINGIRB Ready

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Sponsored by NS Pharma, Inc.

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
๐Ÿ“Š
1 of 20 participants interested
5% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged โ‰ฅ4 to \<15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.

Who Can Participate

Inclusion Criteria

Male โ‰ฅ 4 years and \<15 years of age
Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame
Able to walk independently without assistive devices
Ability to complete the TTSTAND without assistance in \<20 seconds
Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.
Other inclusion criteria may apply.

Exclusion Criteria

Has a body weight of \<20 kg at the time of informed consent (applies to participants screening for Part 1 only)
Evidence of symptomatic cardiomyopathy
Current or previous treatment with anabolic steroids (e.g., oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug
Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times t

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

20 participants

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Age Range

4 Years - 14 Years

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Gender

MALE

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Sponsor

NS Pharma, Inc.

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Aurora

Colorado

Location available
View Aurora location page
RECRUITING

Kissimmee

Florida

Location available
View Kissimmee location page
RECRUITING

Atlanta

Georgia

Location available
View Atlanta location page
RECRUITING

Chicago

Illinois

Location available
View Chicago location page
RECRUITING

Kansas City

Kansas

Location available
View Kansas City location page
RECRUITING

Cincinnati

Ohio

Location available
View Cincinnati location page
RECRUITING

Philadelphia

Pennsylvania

Location available
View Philadelphia location page
RECRUITING

Pittsburgh

Pennsylvania

Location available
View Pittsburgh location page
RECRUITING

Dallas

Texas

Location available
View Dallas location page

๐Ÿ“ And 1 more location available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Looking for Duchenne Muscular Dystrophy Treatment in Aurora?

Join others in Colorado exploring innovative treatment options through clinical research

Duchenne Muscular Dystrophy Treatment Options in Aurora, Colorado

If you're searching for Duchenne Muscular Dystrophy treatment in Aurora, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Aurora, Kissimmee, Atlanta and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Duchenne Muscular Dystrophy. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Colorado
Now Enrolling
Up to 20 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Duchenne Muscular Dystrophy?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Duchenne Muscular Dystrophy

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Duchenne Muscular Dystrophy Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT05996003. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.