Repurposing 5-Azacytidine for Muscle Contractures in Children with Cerebral Palsy
This study is looking at a drug called 5-Azacytidine (AZA) to see if it can help children with Cerebral Palsy (CP) who have muscle contractures (when muscles become tight and shortened). AZA is already approved for other conditions, and researchers want to see if it can be “repurposed” to help muscles grow and improve movement in children with CP. This study will test different doses of AZA, given as a one-time shot under the skin, against a placebo (an inactive substance). The main goal is to find the safest dose and see if it can improve how muscle-generating stem cells work. You might be able to join if you are between 2 and 18 years old, have CP with specific muscle tightness requiring surgery, and have normal kidney and liver function. The study plans to enroll 27 participants.
- Study design
- This is a controlled dose-escalation study, meaning different doses of the drug will be tested against a placebo. It plans to enroll 27 participants.
- What's involved
- You would have five study visits, including a screening visit and a visit about 15 days before scheduled surgery for contracture release. Visits involve blood draws, medical assessments, and receiving an injection.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary endpoint, dose-limiting toxicity, is measured through study completion, an average of 2 years.
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Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy
At a glance
Conditions
NCT06377085
Where you'd take part
This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Rady Children's Hospital - San Diego
San Diego, Californiano site contact published
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Andrea Domenighetti, PhD · PRINCIPAL_INVESTIGATOR · Shirley Ryan AbilityLab
- Patrick Curran, MD · PRINCIPAL_INVESTIGATOR · Rady Children's Hospital, San Diego
- Richard L. Lieber, PhD · PRINCIPAL_INVESTIGATOR · Shirley Ryan AbilityLab
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
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Inclusion
What this trial measures
- Dose-limiting toxicity (DLT).Through study completion, an average of 2 years.
The percentage of patients experiencing DLT at the predefined dose level will be calculated. This will determine the Maximum Tolerated Dose (MTD), which will be the highest dose level at which ≤ 33% of patients experience a DLT. DLT is defined as toxic effects, presumably related to AZA, considered unacceptable due to their severity and/or irreversibility, thereby limiting further dose escalation. Thus, the total number of toxicities and the DLT at each step of dose escalation and possible de-escalation will be reported. The scale for the primary endpoint is binary (occurrence of DLT or not). It is measured as a single endpoint, as it is focused on whether or not the predefined dose level causes DLT. The currently recommended clinical dose is 75 mg/m2. For the present study, the following AZA concentrations will be evaluated: 10 mg/m2, 20 mg/m2, 35 mg/m2 and 75 mg/m2. For each dose, 3 experimental and 3 placebo subjects will be recruited.