Shirley Ryan AbilityLab
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.
Cerebral palsy (CP) has an enduring impact on the development of the muscles after birth. Research showed that a drug that is currently approved by the FDA to treat myelodysplastic syndromes in adults and children, can be potentially adapted ("repurposed") to support muscle growth. In a controlled dose-escalation study, we will study the preliminary safety, biological properties, and efficacy of this drug, called 5-Azacytidine (AZA). Our hope and overarching expectations are that one day AZA will become a new approved pharmacological treatment to support muscle growth and improve body movement…
Inclusion Criteria: 1. Diagnosis of cerebral palsy. 2. Either achilles or hamstring spasticity with contracture necessitation surgical lengthening. 3. Between 2 and 18 years of age 4. Normal renal and liver function as defined by NCI-CTCAE criteria.71 a. Renal Function (Grade 0 - Normal): i. Creatinine: Within the normal range or ≤ 1.0 times the upper limit of normal (ULN). ii. Glomerular filtration rate (GFR): No significant decrease. b. Liver Function (Grade 0 - Normal): i. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT): Within the normal range or ≤ ULN. ii.…
Placebo control group for the 10mg/m\^2, one-time subcutaneous injection without the active treatment.
Placebo control group for the 20mg/m\^2, one-time subcutaneous injection without the active treatment.
Placebo control group for the 35mg/m\^2, one-time subcutaneous injection without the active treatment.
Placebo control group for the 75mg/m\^2, one-time subcutaneous injection without the active treatment.
5-Azacytidine 10mg/m\^2, one-time subcutaneous injection
5-Azacytidine 20mg/m\^2, one-time subcutaneous injection
5-Azacytidine 35mg/m\^2, one-time subcutaneous injection
5-Azacytidine 75mg/m\^2, one-time subcutaneous injection