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Tests treatment safety and results for Duchenne Muscular Dystrophy
Official title A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
ClinicalTrials.gov ID: NCT04626674
What this study is testing
What is delandistrogene moxeparvovec?
delandistrogene moxeparvovec is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy.
Also referred to as SRP-9001, delandistrogene moxeparvovec-rokl.
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
- Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older, men only
You may be able to join if
- For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the...
- Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to...
- Cohort 1: Is ambulatory, and ≥4 to \<8 years of age at the time of Screening.
- Cohort 2: Is ambulatory, and ≥8 to \<18 years of age at the time of Screening.
You likely can't join if
- Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its...
- Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator...
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time...
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests. Note: Other inclusion/exclusion criteria apply.
See the full eligibility criteria
- For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of...
- Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in...
- Cohort 1: Is ambulatory, and ≥4 to \<8 years of age at the time of Screening.
- Cohort 2: Is ambulatory, and ≥8 to \<18 years of age at the time of Screening.
- Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.
- Cohort 4: Is ambulatory and ≥3 to \<4 years of age at the time of Screening.
- Cohort 5a: Is ambulatory and ≥4 to \<9 years of age with time to rise from the floor ≤7 seconds at the screening visit.
- Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
- Cohort 6: Is ambulatory, and ≥2 to \<3 years of age at the time of Screening.
- Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.
- Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.
- Genetic mutation vary by cohort. All Cohorts:
- Ability to cooperate with motor assessment testing.
- rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
- Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.
- Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests. Note: Other inclusion/exclusion criteria apply.
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- Palo Alto, California, United States
- Sacramento, California, United States
- St Louis, Missouri, United States
- Columbus, Ohio, United States
- Flower Mound, Texas, United States
- Norfolk, Virginia, 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, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; Palo Alto, California, United States; Sacramento, California, United States; St Louis, Missouri, United States; Columbus, Ohio, United States; Flower Mound, Texas, United States and 1 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.