Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06506461
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-06-14
Brief Title:
Gene Editing For Sickle Cell Disease
Sponsor:
None
Organization:
None
Study Overview
Official Title:
St Jude Autologous Genome Edited Stem Cells For Sickle Cell Disease-1 SAGES1
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease SCD patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin HbF and help reduce the symptoms of SCD
Primary Objective
To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats CRISPR CRISPR associated protein Cas9-edited CD34 hematopoietic stem and progenitor cells HSPCs in patients with severe SCD
Secondary Objective
To assess the efficacy autologous infusion of CRISPRCas9 genome-edited CD34 HSPCs into patients with severe SCD
Primary Objective
To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats CRISPR CRISPR associated protein Cas9-edited CD34 hematopoietic stem and progenitor cells HSPCs in patients with severe SCD
Secondary Objective
To assess the efficacy autologous infusion of CRISPRCas9 genome-edited CD34 HSPCs into patients with severe SCD
Detailed Description:
Participants will receive a daily injection of plerixafor under the skin for 3-5 days to mobilize their hematopoietic stem and progenitor cells HSPCs into peripheral blood About 2-4 hours after each dose of plerixafor is given the collection of HSPCs will start via apheresis The collected HSPCs will be sent to a lab to genetically modify them using CRISPRCas9
In the lab the researchers will take the stem cells and purify them The stem cells will then be mixed with the CRISPR-Cas9 gRNA ribonucleoprotein RNP complex to change edit the genes in the cells and produce the new gene edited cellular product This gene edited drug product will be frozen until ready for infusion
Once the cellular product is ready participants will be given Busulfan a chemotherapy medicine intravenously IV for 4 days The thawed gene product will be given IV about 48 hours after the completion of the last dose of busulfan
Participants will be followed for 3 years on this study After the three years participants will be followed for 12 more years on a long-term follow-up study
In the lab the researchers will take the stem cells and purify them The stem cells will then be mixed with the CRISPR-Cas9 gRNA ribonucleoprotein RNP complex to change edit the genes in the cells and produce the new gene edited cellular product This gene edited drug product will be frozen until ready for infusion
Once the cellular product is ready participants will be given Busulfan a chemotherapy medicine intravenously IV for 4 days The thawed gene product will be given IV about 48 hours after the completion of the last dose of busulfan
Participants will be followed for 3 years on this study After the three years participants will be followed for 12 more years on a long-term follow-up study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None