An open-label, phase 1 gene therapy study consisting of autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a lentiviral vector encoding for the PK enzyme is now enrolling patients.
PKD patients with a confirmed PKLR mutation and severe, transfusion dependent anemia despite splenectomy may be eligible to enroll. The trial will initially enroll adult patients (≥18 and <45 years old) at Hospital Universitario Fundación Jiménez Díaz in Madrid, Spain. Once safety has been established in the adult population, the trial will next enroll older pediatric patients (≥12-17 years), followed by younger pediatric patients (≥8-11 years) at Hospital Infantil Universitario Niño Jesús.
Financial support, including travel and housing accommodations for patients and a family member, both for the treatment and follow-up visits, will be provided.
If you are interested to learn more about the trial please email Dr. Gayatri Rao at PKDclinicaltrial(ELIMINAR)@rocketpharma.com, visit www.clinicaltrial.gov (NCT04105166), or click on https://bit.ly/2Qi6I6d for direct link.