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New treatment option for Duchenne Muscular Dystrophy
Official title NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
ClinicalTrials.gov ID: NCT05996003
What this study is testing
What is NS-089/NCNP-02?
NS-089/NCNP-02 is an investigational medicine, given as an once-weekly infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy.
Also referred to as Brogidirsen.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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 ≥4 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 14, men only
You may be able to join if
- Male ≥ 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.
You likely can't join if
- 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...
- Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the...
- Surgery within the 3 months prior to the first dose of study drug or planned during the study duration
- Previously treated in an treatment study of NS-089/NCNP-02
See the full eligibility criteria
- Male ≥ 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 may apply.
- 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 the half-life of a medication, whichever is longer
- Surgery within the 3 months prior to the first dose of study drug or planned during the study duration
- Previously treated in an treatment study of NS-089/NCNP-02
- Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP
- Other exclusion criteria may apply.
The study team makes the final eligibility decision.
Where it's taking place
- Aurora, Colorado, United States
- Kissimmee, Florida, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- Kansas City, Kansas, United States
- Cincinnati, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Pittsburgh, Pennsylvania, United States
- Dallas, Texas, United States
- Richmond, Virginia, United States
- Nedlands, Western Australia, Australia
- Calgary, Alberta, Canada
- Vancouver, British Columbia, Canada
- London, Ontario, Canada
- Fukui-shi, Fukui, Japan
- Nagara, Gifu-shi, Gifu, Japan
- Toyonaka, Osaka, Japan
- Moriyama-shi, Shiga, Japan
- Kodaira, Tokyo, Japan
- Auckland, New Zealand
+ 5 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 4 years to 14 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Aurora, Colorado, United States; Kissimmee, Florida, United States; Atlanta, Georgia, United States; Chicago, Illinois, United States; Kansas City, Kansas, United States; Cincinnati, Ohio, United States and 19 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.