NCT06422351

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

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: Suspended
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Rocket Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 8 Years – 55 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Pyruvate Kinase Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	RP-L301	Autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKLR (Pyruvate Kinase L/R) gene

Timeline

• Start date: 2026-04-01
• Primary completion: 2029-01-01
• Completion: 2029-01-01
• First posted: 2024-05-21
• Last updated: 2026-04-16

Locations

3 sites across 2 countries: United States, Spain

Regulatory

• FDA-regulated drug study