Gene Therapy for Pyruvate Kinase Deficiency
Trial Summary
What is the purpose of this trial?
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).
Research Team
José Luis López Lorenzo, MD
Principal Investigator
Hospital Universitario Fundación Jiménez Díaz
Ami Shah, MD
Principal Investigator
Stanford University
Julián Sevilla Navarro, MD, PhD
Principal Investigator
Hospital Universitario Fundación Jiménez Díaz
Eligibility Criteria
This trial is for people with Pyruvate Kinase Deficiency (PKD) who are between 8-50 years old, depending on enrollment phase. They must have a history of multiple blood transfusions and low hemoglobin levels despite treatment like splenectomy. Participants need to be in good health regarding heart, lung, kidney, and liver function.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Gene Therapy Treatment
Autologous hematopoietic stem cells are transduced with a lentiviral vector and infused back into the patient
Follow-up
Participants are monitored for safety, toxicity, and genetic correction over a period of time
Treatment Details
Interventions
- RP-L301
Find a Clinic Near You
Who Is Running the Clinical Trial?
Rocket Pharmaceuticals Inc.
Lead Sponsor