Ignite Creation Date:
2024-06-16 @ 11:48 AM
Last Modification Date:
2024-10-26 @ 3:30 PM
Study NCT ID:
NCT06422351
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-05-22
First Post:
2024-05-15
Brief Title:
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
Sponsor:
Rocket Pharmaceuticals Inc
Organization:
Rocket Pharmaceuticals Inc
Study Overview
Official Title:
A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34 Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase coRPK Gene in Subjects With Pyruvate Kinase Deficiency
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-05
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 Phase II trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency PKD
Detailed Description:
Autologous hematopoietic stem cells from mobilized peripheral blood will be transduced ex vivo outside the body with a lentiviral vector carrying a correct copy of the deficient PKLR Pyruvate Kinase LR gene The corrected stem cells will be infused intravenously back into the patient to correct the hematological manifestations of the disease
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