Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy (NCT06377085) | Clinical Trial Compass
By InvitationPhase 1
Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy
United States27 participantsStarted 2025-07
Plain-language summary
In this controlled dose-escalation study, we will study the initial safety, biological properties, and potential efficacy of 5-azacytidine (AZA). Our overarching aspiration is for AZA to evolve into an approved pharmacological treatment, fostering muscle growth and enhancing body movement, ultimately contributing to an improved quality of life in children with CP.
The main questions this study aims to answer are:
1. What is the optimal dose of AZA injection that can be used safely in children with CP?
2. Can the optimal safe dose of AZA improve the function of muscle-generating stem cells in children with CP?
Each participant will have up to five research visits over the course of the study duration, in which they will participate in: blood draws, pregnancy test(s) (if applicable), medical assessments, and a muscle biopsy during a surgery for muscle contractures.
Researchers will compare participants with four different dosages of AZA injections to those with four different dosages of placebo injections. A placebo is a look-alike substance that contains no active drug. They will see if a single injection of AZA at a standard concentration currently approved by the FDA to treat myelodysplastic syndromes, can also safely improve muscle growth and function in children with CP.
Who can participate
Age range2 Years – 18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Diagnosis of cerebral palsy.
✓. Either achilles or hamstring spasticity with contracture necessitation surgical lengthening.
✓. Between 2 and 18 years of age
✓. Normal renal and liver function as defined by NCI-CTCAE criteria.71
✓. A negative pregnancy test for females of childbearing potential\*.
✓. Females of childbearing potential must agree to use contraception consistently from screening to 6 months after their injection. Highly effective methods of contraception are required for females of childbearing potential:
✓. Total abstinence from sexual intercourse.
✓. Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).
Exclusion criteria
✕. Active infection.
✕. Cardiac disease.
What they're measuring
1
Dose-limiting toxicity (DLT).
Timeframe: Through study completion, an average of 2 years.