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Clinical trial

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

NCT04105166 is registered in ClinicalTrials.gov with Rocket Pharmaceuticals Inc. as lead sponsor.

NCT NCT04105166
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Registry record

As filed in ClinicalTrials.gov.

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
Lead sponsor
Rocket Pharmaceuticals Inc.
Phase
Phase 1
Overall status
Completed
Start date
July 6, 2020
Completion date
June 9, 2025
Enrollment
4 (actual)
Sites
3

Conditions

As the registry lists them; a condition with its own page is linked.

pyruvate kinase deficiency