NCT04105166

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Rocket Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 8 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

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).

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 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.

Conditions

• Pyruvate Kinase Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	RP-L301	Autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKD gene

Timeline

• Start date: 2020-07-06
• Primary completion: 2025-06-09
• Completion: 2025-06-09
• First posted: 2019-09-26
• Last updated: 2025-09-05

Locations

3 sites across 2 countries: United States, Spain

Regulatory

• FDA-regulated drug study