Ignite Creation Date:
2024-05-06 @ 2:50 PM
Last Modification Date:
2024-10-26 @ 1:38 PM
Study NCT ID:
NCT04443907
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-01-03
First Post:
2020-04-29
Brief Title:
Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease SCD
Sponsor:
Novartis Pharmaceuticals
Organization:
Novartis
Study Overview
Official Title:
A First-in-patient Phase III Clinical Study to Investigate the Safety Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease
Status:
ACTIVE_NOT_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 study is evaluating a genome-edited autologous hematopoietic stem and progenitor cell HSPC product - OTQ923 to reduce the biologic activity of BCL11A increasing fetal hemoglobin HbF and reducing complications of sickle cell disease
Detailed Description:
CADPT03A12101 is a multicenter multi-part first-in-human proof-of-concept open label non-randomized clinical study in Sickle Cell Disease SCD subjects This study includes apheresis of mobilized hematopoietic stem and progenitor cells HSPCs ex vivo CRISPRCas9-mediated genome editing and expansion followed by myeloablative conditioning and autologous hematopoietic stem cell transplant HSCT with follow-up for a minimum of one year and up to two years
The study is divided into the following parts
Part A - Adult subjects dosed with OTQ923
Part B - Assessment of OTQ923 in pediatric patients Part B will not be opened
The study is divided into the following parts
Part A - Adult subjects dosed with OTQ923
Part B - Assessment of OTQ923 in pediatric patients Part B will not be opened
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2019-003489-41 | EUDRACT_NUMBER | None | None |