A Phase 1 interventional study of Placebo for the AZA 10mg/m^2 and Placebo for the AZA 20mg/m^2 in Cerebral Palsy and Contracture, sponsored by Shirley Ryan AbilityLab. Enrolling by invitation at 1 site in United States. Open to participants aged 2 Years to 18 Years. Per ClinicalTrials.gov, last updated 2025-07-03.
Sponsored by Shirley Ryan AbilityLab · Phase 1, Interventional, and Treatment
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:
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 and quality of life in children with CP.
Research participants will have five study visits.
The amount of blood drawn at each time point will be approximately 3 mL, equating to 12 mL of blood total (less than a tablespoon). The purpose of the blood draws is to evaluate safety and biological efficacy of the study drug. The medical assessment will consist of range of motion assessment and wound check performed by the clinicians. These are the same assessments that the clinicians would typically do as part of usual care prior to and after surgery. The purpose of the medical assessment is to evaluate the efficacy of the study drug and ensure the surgical site is healing.
1,936 studies on the registry are indexed under Cerebral Palsy; 458 are open to participants now.
This study's planned enrollment of 27 is below the median of 34 across 1,431 interventional studies indexed under Cerebral Palsy.
Browse Cerebral Palsy studies →Shirley Ryan AbilityLab is the lead sponsor of 180 studies on the registry; 44 are open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 4 (40%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Normal renal and liver function as defined by criteria.71
a. Renal Function (Grade 0 - Normal): i. Creatinine: Within the normal range or ≤ 1.0 times the (ULN).
ii. (GFR): No significant decrease. b. Liver Function (Grade 0 - Normal): i. (AST) or alanine aminotransferase (ALT): Within the normal range or ≤ ULN.
ii. Total : Within the normal range or ≤ 1.0 times ULN
Females of childbearing potential must agree to use consistently from screening to 6 months after their injection. Highly effective methods of contraception are required for females of childbearing potential:
Males of childbearing potential** must agree to use contraception consistently from screening until 3 months after the injection. Acceptable methods of contraception for males of childbearing potential are:
Condom with spermicide (cream, spray, foam, gel, suppository, or polymer film).
Female of childbearing potential is defined as a female capable of becoming pregnant, which includes patients who have had their first menstrual cycle (menarche).
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
5-Azacytidine, subcutaneous injection, 10mg/m\^2, one subcutaneous injection for duration of study (estimated at 46 - 76 days)
Drug: 5-Azacytidine 10mg/m^2
5-Azacytidine, subcutaneous injection, 20mg/m\^2, one subcutaneous injection for duration of study (estimated at 46 - 76 days)
Drug: 5-Azacytidine 20mg/m^2
5-Azacytidine, subcutaneous injection, 35mg/m\^2, one subcutaneous injection for duration of study (estimated at 46 - 76 days)
Drug: 5-Azacytidine 35mg/m^2
5-Azacytidine, subcutaneous injection, 75mg/m\^2, one subcutaneous injection for duration of study (estimated at 46 - 76 days)
Drug: 5-Azacytidine 75mg/m^2
Placebo, subcutaneous injection, 10mg/m\^2, one subcutaneous injection without the active treatment for duration of study (estimated at 46 - 76 days)
Drug: Placebo for the AZA 10mg/m^2
Placebo, subcutaneous injection, 20mg/m\^2, one subcutaneous injection without the active treatment for duration of study (estimated at 46 - 76 days)
Drug: Placebo for the AZA 20mg/m^2
Placebo, subcutaneous injection, 35mg/m\^2, one subcutaneous injection without the active treatment for duration of study (estimated at 46 - 76 days)
Drug: Placebo for the AZA 35mg/m^2
Placebo, subcutaneous injection, 75mg/m\^2, one subcutaneous injection without the active treatment for duration of study (estimated at 46 - 76 days)
Drug: Placebo for the AZA 75mg/m^2
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.
Drug page: Azacitidine
5-Azacytidine 10mg/m\^2, one-time subcutaneous injection
Drug page: Azacitidine
5-Azacytidine 20mg/m\^2, one-time subcutaneous injection
Drug page: Azacitidine
5-Azacytidine 35mg/m\^2, one-time subcutaneous injection
Drug page: Azacitidine
5-Azacytidine 75mg/m\^2, one-time subcutaneous injection
Dose-limiting toxicity (DLT).
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.
Time frame: Through study completion, an average of 2 years.
Satellite Cell Fusion Index.
Resident muscle-forming stem cells, called Satellite Cells, will be isolated from the muscle biopsy obtained from subjects and cultured in vitro. Satellite Cell Fusion Index quantifies the proportion of these stem cells that will fuse to form new muscle fibers in vitro (multinucleated structures called myotubes). It provides a measure of the efficiency of subjects' satellite cells to contribute to muscle growth, repair and regeneration.
Time frame: Through study completion, an average of 2 years.
DNA methylation quantification in Satellite Cells and Blood Mononucleated Cells.
Global DNA methylation analysis will be quantified (% tot DNA in ng) using DNA from Satellite Cell cultures and Blood Mononucleated Cells (isolated form blood) using an ELISA-based global DNA methylation kit.
Time frame: Through study completion, an average of 2 years.
DNA methylation profiling in Satellite Cells.
Following Satellite Cell isolation, expansion in culture and DNA extractions, qualitative methylation analysis procedures will be conducted using an Infinium Human MethylationEPIC Beadchip array (Illumina Inc., CA), which targets over 850,000 DNA methylation sites at the dinucleotide (CpG) level throughout the entire genome.
Time frame: Through study completion, an average of 2 years.
Chromatin immunoprecipitation sequencing (ChIP-seq), and assay for transposase-accessible chromatin sequencing (ATAC-seq)
ChIP-seq will facilitate the genome-wide profiling of changes in chromatin structure and transcription factor binding events in response to AZA treatment and muscle contracture development. This technique is particularly as it can identify alterations in DNA methylation patterns and histone modifications associated with Satellite Cell dysfunction and impaired muscle regeneration. Furthermore, ATAC-seq will offer a complementary approach to ChIP-seq by providing information on chromatin accessibility, which reflects regulatory elements such as enhancers and promoters involved in gene expression control.
Time frame: Through study completion, an average of 2 years.
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Jul 2025; primary completion for Dec 2026, 17 months later.
Of 22 phase 1 interventional studies under Cerebral Palsy, half reached primary completion within 30 months of starting (the median), counting those still running; a quarter within 13 and three quarters within 45. This study's planned 17 months is below that.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Azacitidine