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Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Pyruvate Kinase Deficiency Clinical Trial

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

Clinical Trial 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).

Clinical Trial 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 Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06422351
Study type Interventional
Source Rocket Pharmaceuticals Inc.
Contact Rocket Clinical Trials
Phone 646-627-0033
Email PKDclinicaltrial@rocketpharma.com
Status Not yet recruiting
Phase Phase 2
Start date August 2024
Completion date January 2029
View Details
See also
  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
Active, not recruiting NCT04105166 - Gene Therapy for Pyruvate Kinase Deficiency (PKD) Phase 1
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