Ignite Creation Date:
2024-07-17 @ 11:09 AM
Last Modification Date:
2024-10-26 @ 3:32 PM
Study NCT ID:
NCT06464588
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-06-18
First Post:
2024-06-13
Brief Title:
A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells nMSCs in Young Adult 1A and Pediatric 1B Patients With Dilated Cardiomyopathy DCM
Sponsor:
Emory University
Organization:
Emory University
Study Overview
Official Title:
A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells nMSCs in Young Adult 1A and Pediatric 1B Patients With Dilated Cardiomyopathy DCM
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
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:
This is a Phase 1 study to determine the safety and efficacy of allogeneic neonatal mesenchymal stromal cells nMSCs for the treatment of Dilated Cardiomyopathy The purpose of the study is to help doctors and scientists learn if allogeneic neonatal mesenchymal stromal cells nMSCs infusions are a safe and effective way to improve cardiac function and left ventricular ejection fraction
Detailed Description:
This study is a single site open label study with 2 phases The 2 phases will be broken into an adult group Phase 1A with group pediatric group Phase 1B This study will enroll patients between the ages of 4 years and 30 years old The investigators will be looking at the safety feasibility and maximum tolerated dose of allogeneic neonatal mesenchymal stromal cells nMSCs as defined by freedom from CTCAE or Grade 3 AE that is probably or definitely related to the IP throughout the duration of the study
A minimum of 9 and a maximum of 18 patients will be enrolled into both Phase 1A adult groups and 1B pediatric groups Phase 1A subjects will receive study products with doses defined by the study group and Phase 1B will begin after all adult subjects have completed Phase 1A infusions FDA and a DSMC review All pediatric subjects will receive study product dosed per body weight in the defined study group
Allogeneic neonatal mesenchymal stromal cells nMSCs will be infused via IV every 15 days for a total of 3 infusions A check-in will be scheduled the morning after each infusion for pediatric patients There will be a baseline visit before allogeneic neonatal mesenchymal stromal cells nMSCs therapy is initiated followed by a phone call 30 days after the last infusion There will be in person visits at 3-month 6 months and 1 year mark The total duration for each patient will be 14 months
Labs will be collected at baseline during nMSC infusions and at in person follow up visits to assess cardiac function Any leftover blood samples may be stored for future research by the sponsor of the study Echocardiograms will be completed at baseline and 3 month- 6 month- and 1-year visits to look at left ventricular ejection fraction and electrocardiograms will be completed to provide measures of cardiac rhythm or rhythm Other assessments include physical exam 6-minute walk test Cardiac MRI vital signs PedsQL questionnaire for pediatrics and The Kansas City Cardiomyopathy Questionnaire for adults
It is expected to recruit 18-36 participants through face-to-face encounters between participants and study staff during clinical encounters at Childrens Healthcare of Atlanta and Emory Health care system Clinicaltrialsgov registration and Institutional Review Board approved advertisements to surrounding hospitals with cardiac programs Patients will also be recruited through MyChart If identified as eligible to participate the study team will seek approval by the subjects primary cardiologist and consent andor assent with the permission of the parent or legally authorized representative will occur in person during a baseline visit
After allogeneic neonatal mesenchymal stromal cells nMSCs infusions pediatric patients will be provided an overnight stay at Ronald McDonald House or a hospital affiliated hotel in case of any unanticipated effects and follow up visits the next morning This is so pediatric patients are near the hospital for such events Adults will not be required to stay overnight for a follow-up visit There will be financial compensation for each study visit and patients will be reimbursed for parking
A minimum of 9 and a maximum of 18 patients will be enrolled into both Phase 1A adult groups and 1B pediatric groups Phase 1A subjects will receive study products with doses defined by the study group and Phase 1B will begin after all adult subjects have completed Phase 1A infusions FDA and a DSMC review All pediatric subjects will receive study product dosed per body weight in the defined study group
Allogeneic neonatal mesenchymal stromal cells nMSCs will be infused via IV every 15 days for a total of 3 infusions A check-in will be scheduled the morning after each infusion for pediatric patients There will be a baseline visit before allogeneic neonatal mesenchymal stromal cells nMSCs therapy is initiated followed by a phone call 30 days after the last infusion There will be in person visits at 3-month 6 months and 1 year mark The total duration for each patient will be 14 months
Labs will be collected at baseline during nMSC infusions and at in person follow up visits to assess cardiac function Any leftover blood samples may be stored for future research by the sponsor of the study Echocardiograms will be completed at baseline and 3 month- 6 month- and 1-year visits to look at left ventricular ejection fraction and electrocardiograms will be completed to provide measures of cardiac rhythm or rhythm Other assessments include physical exam 6-minute walk test Cardiac MRI vital signs PedsQL questionnaire for pediatrics and The Kansas City Cardiomyopathy Questionnaire for adults
It is expected to recruit 18-36 participants through face-to-face encounters between participants and study staff during clinical encounters at Childrens Healthcare of Atlanta and Emory Health care system Clinicaltrialsgov registration and Institutional Review Board approved advertisements to surrounding hospitals with cardiac programs Patients will also be recruited through MyChart If identified as eligible to participate the study team will seek approval by the subjects primary cardiologist and consent andor assent with the permission of the parent or legally authorized representative will occur in person during a baseline visit
After allogeneic neonatal mesenchymal stromal cells nMSCs infusions pediatric patients will be provided an overnight stay at Ronald McDonald House or a hospital affiliated hotel in case of any unanticipated effects and follow up visits the next morning This is so pediatric patients are near the hospital for such events Adults will not be required to stay overnight for a follow-up visit There will be financial compensation for each study visit and patients will be reimbursed for parking
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?:
False
Is an FDA AA801 Violation?:
None