clinical trial · NCT04819841
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Sickle Cell Diseasenula-cel Drug Product
brief summary
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
started
Nov 15, 2021
primary completion
Dec 31, 2026
completion
Dec 31, 2028
last updated
Mar 11, 2026
detailed description
Participants diagnosed with severe SCD will receive nula-cel via IV infusion following myeloablative conditioning in an autologous HSCT setting.
official title
A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol