Access cutting-edge sickle cell disease treatment through this clinical trial at a research site in Philadelphia. Study-provided care at no cost to qualified participants.
Access sickle cell disease specialists in Philadelphia at no cost
This study follows strict safety protocols and ethical guidelines
All study-related sickle cell disease treatment provided free
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
Sponsor: Vertex Pharmaceuticals Incorporated
Check if you qualify for this sickle cell disease clinical trial in Philadelphia, PA
If you're searching for sickle cell disease treatment options in Philadelphia, PA, this clinical trial may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.
Our Philadelphia research site is actively enrolling participants for this clinical trial. You'll receive care from experienced sickle cell disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.