Ignite Creation Date:
2024-05-06 @ 8:25 PM
Last Modification Date:
2024-10-26 @ 3:27 PM
Study NCT ID:
NCT06377085
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-04-22
First Post:
2024-03-05
Brief Title:
Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy
Sponsor:
Shirley Ryan AbilityLab
Organization:
Shirley Ryan AbilityLab
Study Overview
Official Title:
Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 tests 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
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 tests 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
Detailed Description:
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
1 The first study visit will be a screening within 30 days of the baseline visit and will determine eligibility for the study The participant will have their blood drawn to check for normal kidney and liver functioning do a pregnancy test if applicable and then the researcher will complete a medical assessment
2 If the participant is eligible they will be invited to a second study visit approximately 15 days prior to their scheduled surgery for contracture release At this visit the participants will get their blood drawn do a pregnancy test if applicable have a medical assessment performed and receive their injection The injection will be a single sub-cutaneous shot in their leg near the muscle group that will undergo surgical repair
3 The third visit will be at the participants scheduled surgery A small sample of the muscle ie about the size of a pencil eraser will be surgically removed by the researcher from a muscle group that is already exposed during the procedure The biopsy will add approximately two to five minutes to the overall procedure time The participant will also have their blood drawn
4 The participants will be seen approximately one week after their surgery to complete a medical assessment
5 The last visit will be about four weeks after the surgery at their post-operative appointment At this visit the participant will have their blood drawn and complete a medical assessment to test their range of motion following the surgery
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
Research participants will have five study visits
1 The first study visit will be a screening within 30 days of the baseline visit and will determine eligibility for the study The participant will have their blood drawn to check for normal kidney and liver functioning do a pregnancy test if applicable and then the researcher will complete a medical assessment
2 If the participant is eligible they will be invited to a second study visit approximately 15 days prior to their scheduled surgery for contracture release At this visit the participants will get their blood drawn do a pregnancy test if applicable have a medical assessment performed and receive their injection The injection will be a single sub-cutaneous shot in their leg near the muscle group that will undergo surgical repair
3 The third visit will be at the participants scheduled surgery A small sample of the muscle ie about the size of a pencil eraser will be surgically removed by the researcher from a muscle group that is already exposed during the procedure The biopsy will add approximately two to five minutes to the overall procedure time The participant will also have their blood drawn
4 The participants will be seen approximately one week after their surgery to complete a medical assessment
5 The last visit will be about four weeks after the surgery at their post-operative appointment At this visit the participant will have their blood drawn and complete a medical assessment to test their range of motion following the surgery
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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None