clinical trial · NCT04208529
A Long-term Follow-up Study in Participants Who Received CTX001
Vertex Pharmaceuticals Incorporated·phase3·enrolling by invitation·n = 160
Beta-ThalassemiaThalassemiaSickle Cell DiseaseHematologic DiseasesHemoglobinopathiesGenetic Diseases, InbornSickle Cell AnemiaCTX001
brief summary
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease \[SCD\] studies).
started
Jan 20, 2021
primary completion
Sep 30, 2039
completion
Sep 30, 2039
last updated
Mar 25, 2026
official title
A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol