Pyruvate Kinase Deficiency Clinical Trial
Official title:
Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects With PKD
This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
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 PKD gene. The corrected stem cells will be infused intravenously back to the patient with the goal of correcting the hematological manifestations of the disease. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Active, not recruiting |
NCT03853798 -
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
|
Phase 3 | |
| Completed |
NCT03559699 -
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
|
Phase 3 | |
| Active, not recruiting |
NCT02476916 -
A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
|
Phase 2 | |
| Enrolling by invitation |
NCT05777993 -
A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
|
Phase 4 | |
| Terminated |
NCT04964323 -
Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
|
||
| Completed |
NCT04995315 -
Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008
|
||
| Completed |
NCT03866590 -
Pyruvate Kinase Deficiency Epidemiological Study (PIECE)
|
||
| Recruiting |
NCT04902833 -
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
|
||
| Completed |
NCT02053480 -
Pyruvate Kinase Deficiency Natural History Study
|
||
| Completed |
NCT03548220 -
A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
|
Phase 3 | |
| Recruiting |
NCT03481738 -
Pyruvate Kinase Deficiency Global Longitudinal Registry
|