Ignite Creation Date:
2024-05-06 @ 3:50 PM
Last Modification Date:
2024-10-26 @ 1:58 PM
Study NCT ID:
NCT04774536
Status:
RECRUITING
Last Update Posted:
2023-07-07
First Post:
2021-02-17
Brief Title:
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells CRISPR_SCD001 in Patients with Severe Sickle Cell Disease
Sponsor:
Mark Walters MD
Organization:
University of California San Francisco
Study Overview
Official Title:
Transplantation of CRISPRCas9 Corrected Hematopoietic Stem Cells CRISPR_SCD001 in Patients with Severe Sickle Cell Disease
Status:
RECRUITING
Status Verified Date:
2024-09
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 an open label non-randomized 2-center phase 12 trial of a single infusion of sickle allele modified cluster of differentiation CD34 hematopoietic stem progenitor cells HSPCs in subjects with in subjects 12 years old to 35 years old severe Sickle Cell Disease SCD The study will evaluate the hematopoietic stem cell transplantation HSCT using CRISPRCas9 edited red blood cells known as CRISPR_SCD001 Drug Product
Detailed Description:
This is an open label non-randomized 2-center phase 12 trial of a single infusion of sickle allele modified CD34 HSPCs in subjects with severe SCD The primary endpoint of the trial will determine the safety of CRISPR_SCD001 through a 33 design with staggered enrollment and a pause in enrollment for safety review after each of the first 3 patients has had drug product infused After safety is assessed in the 3rd patient enrollment of the next 3 patients will not be staggered The first six subjects will be adults If CRISPR_SCD001 is determined to be safe in the first six subjects the trial will continue to enroll 3 adolescents 12 - 18 years of age to evaluate the safety in younger patients The younger age cohort also will follow staggered enrollment
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