Back to search results
NCT06377085

Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy

Enrolling by Invitation
PHASE1Ages 2–18InterventionalTreatment
Shirley Ryan AbilityLab
~27 participants
Updated 2025-07-03 on ClinicalTrials.gov
What's tested:Placebo for the AZA 10mg/m^2Placebo for the AZA 20mg/m^2Placebo for the AZA 35mg/m^2Placebo for the AZA 75mg/m^25-Azacytidine 10mg/m^25-Azacytidine 20mg/m^2

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Dose-limiting toxicity (DLT).
Measured over Through study completion, an average of 2 years.

Conditions

Cerebral Palsy
Contracture

Explore related trials

Cerebral PalsyMuscle Contracture

Where it's being run

1 sites across 1 states
California1

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

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

Female of childbearing potential is defined as a female capable of becoming pregnant, which includes patients who have had their first menstrual cycle (menarche).
Male of childbearing potential is defined as a subject who has reached spermarche.

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.