Ignite Creation Date:
2025-12-24 @ 11:45 PM
Ignite Modification Date:
2026-01-05 @ 7:52 PM
Study NCT ID:
NCT06422351
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-05-22
First Post:
2024-05-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
Sponsor:
Rocket Pharmaceuticals Inc.
Organization:
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 L/R) 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:
True
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?: