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Tests treatment safety and results for Duchenne Muscular Dystrophy (DMD)
Official title Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
ClinicalTrials.gov ID: NCT07608432
What this study is testing
What is Zeleciment Rostudirsen (DYNE-251)?
Zeleciment Rostudirsen (DYNE-251) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy (dmd).
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
- The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug.
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 18, men only
You may be able to join if
- Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51...
- Rise From Floor (RFF) time must be \< 10 seconds for both screening assessments .
- Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a...
You likely can't join if
- Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization
- Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to...
- Any change in prophylaxis/treatment for congestive heart failure (CHF) within 12 weeks prior to randomization
- Receipt of eteplirsen within 1 week prior to randomization
- Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization
- Receipt of givinostat within 12 weeks prior to randomization
See the full eligibility criteria
- Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping .
- Rise From Floor (RFF) time must be \< 10 seconds for both screening assessments .
- Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a stable dose during the compared with an inactive treatment Period of the...
- Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization
- Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to randomization
- Any change in prophylaxis/treatment for congestive heart failure (CHF) within 12 weeks prior to randomization
- Receipt of eteplirsen within 1 week prior to randomization
- Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization
- Receipt of givinostat within 12 weeks prior to randomization
- Receipt of gene therapy at any time Note: Other inclusion or exclusion criteria may apply
The study team makes the final eligibility decision.
Where it's taking place
- Hillsborough, North Carolina, United States
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 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Hillsborough, North Carolina, United States. 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.