Ignite Creation Date:
2024-05-06 @ 1:24 PM
Last Modification Date:
2024-10-26 @ 1:14 PM
Study NCT ID:
NCT04018937
Status:
RECRUITING
Last Update Posted:
2023-11-08
First Post:
2019-07-10
Brief Title:
Early Human Leukocyte Antigen HLA Matched Sibling Hematopoietic Stem Cell Transplantation
Sponsor:
Emory University
Organization:
Emory University
Study Overview
Official Title:
Early HLA Matched Sibling Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease A Sickle Transplant Advocacy and Research Alliance STAR Trial
Status:
RECRUITING
Status Verified Date:
2023-11
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 study aims to enroll 58 pre-adolescent 13 years pediatric participants with sickle cell disease SCD who have a pre-adolescent sibling bone marrow donor All participants will go through a pre-transplant evaluation to find out if there are health problems that will keep them from being able to receive the transplant It usually takes 2 to 3 months to complete the pre-transplant evaluation and make the arrangements for the transplant Once they are found to be eligible for transplant participants will be admitted to the hospital and will start transplant conditioning Conditioning is the chemotherapy and other medicines given to prepare them to receive donor cells It prevents the immune system from rejecting donor cells Conditioning will start 21 days before transplant Once they complete conditioning participants will receive the bone marrow transplant After the transplant participants will stay in the hospital for 4-6 weeks After they leave the hospital participants will be followed closely in the clinic Outpatient treatment and frequent clinic visits usually last 6 to 12 months Routine medical care includes at least a yearly examination for many years after transplant by doctors and nurses familiar with sickle cell disease and transplant The researchers will collect and study information about participants for 2 years after transplant
Detailed Description:
The use of HLA matched sibling donor MSD hematopoietic stem cell transplantation HSCT for sickle cell disease SCD is evolving Because of the low risk for graft versus host disease GVHD associated with younger age HSCT is increasingly being performed prior to adolescence The use of less gonadotoxic reduced intensity regimens RIC are more commonly be utilized to lessen the risk for infertility Finally with the recognition that most children even those who have asymptomatic to relatively mild courses will develop debilitating morbidities as adults and ultimately suffer an early death HSCT is being offered to children across a wider spectrum of SCD severity Long reserved for severely affected children HSCT is being performed for a growing number of less severely affected children as well as children without disease manifestation
This trial is designed to prospectively assess HSCT under these conditions Eligibility will be limited to children less than 13 years of age who have an HLA MSD A RIC regimen - fludarabine alemtuzumab and melphalan FAM - will be employed Finally SCD severity criteria will be broadened to include less severely affected children as well as those who are severely affected
This study has the following two specific aims
Specific Aim 1 To prospectively assess the safety and efficacy of HSCT using FAM conditioning in children with SCD of varying severity who are under 13 years of age
Specific Aim 2 To address current gaps in our understanding of the long-term effects of HSCT in children with SCD by longitudinally assessing sickle cell related cerebrovascular disease sickle cell related nephropathy and health related quality of life
This trial is designed to prospectively assess HSCT under these conditions Eligibility will be limited to children less than 13 years of age who have an HLA MSD A RIC regimen - fludarabine alemtuzumab and melphalan FAM - will be employed Finally SCD severity criteria will be broadened to include less severely affected children as well as those who are severely affected
This study has the following two specific aims
Specific Aim 1 To prospectively assess the safety and efficacy of HSCT using FAM conditioning in children with SCD of varying severity who are under 13 years of age
Specific Aim 2 To address current gaps in our understanding of the long-term effects of HSCT in children with SCD by longitudinally assessing sickle cell related cerebrovascular disease sickle cell related nephropathy and health related quality of life
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?:
None
Is an FDA AA801 Violation?:
None