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)

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-512654-19-00

Acronym

VX18-CTX001-131

Enrollment

Registered

2024-06-05

Start date

2020-03-06

Completion date

Unknown

Last updated

2025-11-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Sickle Cell Disease, Transfusion dependent thalassemia

Brief summary

New Malignancies, New or worsening hematologic disorders (e.g. immune-mediated cytopenias, aplastic anemia, primary immunodeficiencies), All-cause mortality, All serious adverse events (SAEs) occurring up to 15 years after CTX001 infusion, CTX001-related AE's

Detailed description

TDT and SCD: Total Hemoglobin (Hb) concentration over time, TDT and SCD: Fetal Hemoglobin (HbF) concentration over time, TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time, TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time, TDT and SCD: Change in patient-reported outcome (PRO) over time in participants ≥18 years of age assessed using EuroQol quality of life scale (EQ-5D-5L) for participants from study 111,121 and 171 only

Interventions

DRUGCasgevy 4 - 13 x 10^6 cells/mL dispersion for infusion

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
New Malignancies, New or worsening hematologic disorders (e.g. immune-mediated cytopenias, aplastic anemia, primary immunodeficiencies), All-cause mortality, All serious adverse events (SAEs) occurring up to 15 years after CTX001 infusion, CTX001-related AE's	—

Secondary

Measure	Time frame
TDT and SCD: Total Hemoglobin (Hb) concentration over time, TDT and SCD: Fetal Hemoglobin (HbF) concentration over time, TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time, TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time, TDT and SCD: Change in patient-reported outcome (PRO) over time in participants ≥18 years of age assessed using EuroQol quality of life scale (EQ-5D-5L) for participants from study 111,121 and 171 only	—

Measure

Time frame

—

Countries

Belgium, Germany, Italy

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026