Ignite Creation Date:
2024-05-06 @ 3:56 PM
Last Modification Date:
2024-10-26 @ 2:00 PM
Study NCT ID:
NCT04819841
Status:
RECRUITING
Last Update Posted:
2024-02-02
First Post:
2021-03-24
Brief Title:
Gene Correction in Autologous CD34 Hematopoietic Stem Cells HbS to HbA to Treat Severe Sickle Cell Disease
Sponsor:
Kamau Therapeutics
Organization:
Kamau Therapeutics
Study Overview
Official Title:
A Phase III Study of Nula-cel in Autologous CD34 Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
Status:
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:
Restore
Brief Summary:
This study is a first-in-human single-arm open-label Phase III study of nula-cel in approximately 15 participants diagnosed with severe Sickle Cell Disease The primary objective is to evaluate safety of the treatment in this patient population as well as preliminary efficacy and pharmacodynamic data
Detailed Description:
Participants diagnosed with severe SCD will receive nula-cel via IV infusion following myeloablative conditioning in an autologous HSCT setting
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