A Long-term Follow-up Study of Subjects With ß-thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-002935-88-GB

Enrollment

Registered

2018-11-27

Start date

2020-07-09

Completion date

Unknown

Last updated

2020-09-07

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

Conditions

Transfusion-dependent ß thalassemia (TDT). Severe sickle cell disease (SCD). MedDRA version: 20.0 Level: PT Classification code 10043391 Term: Thalassaemia beta System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0 Level: PT Classification code 10040641 Term: Sickle cell anaemia System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: CTX001 Product Code: CTX001 Pharmaceutical Form: Dispersion for infusion INN or Proposed INN: Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs)

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subjects or legal representative or guardian (if applicable) must sign and date informed consent form (ICF). 2. Subjects must have received CTX001 infusion. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 90 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. There are no exclusion criteria.

Design outcomes

Primary

Measure	Time frame
Main Objective: Evaluate long-term safety up to 15 years after CTX001 infusion in subjects who received CTX001 for treatment of transfusion-dependent thalassemia (TDT) or severe sickle cell disease (SCD);Secondary Objective: To evaluate efficacy of CTX001 up to 15 years after CTX001 infusion, in subjects who received CTX001 for treatment of TDT or SCD;Primary end point(s): • 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 5 years after CTX001 infusion • CTX001-related AEs and SAEs;Timepoint(s) of evaluation of this end point: Up to 15 years after CTX001 infusion (with exception of SAEs as noted above).	—

Measure

Time frame

Main Objective: Evaluate long-term safety up to 15 years after CTX001 infusion in subjects who received CTX001 for treatment of transfusion-dependent thalassemia (TDT) or severe sickle cell disease (SCD);Secondary Objective: To evaluate efficacy of CTX001 up to 15 years after CTX001 infusion, in subjects who received CTX001 for treatment of TDT or SCD;Primary end point(s): • 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 5 years after CTX001 infusion • CTX001-related AEs and SAEs;Timepoint(s) of evaluation of this end point: Up to 15 years after CTX001 infusion (with exception of SAEs as noted above).

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Secondary

Measure	Time frame
Timepoint(s) of evaluation of this end point: Up to 15 years after CTX001 infusion (with exception of LIC and CIC which will be assessed for up to 5 years).;Secondary end point(s): • Total Hemoglobin (Hb) concentration (pre-transfusion) over time • Fetal hemoglobin (HbF) concentration (pre-transfusion) over time • Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time TDT: • TDT-related transfusion • Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin • Proportion of subjects receiving iron chelation therapy over time SCD: • Severe vaso-occlusive crisis (VOC) • SCD-related transfusions	—

Measure

Time frame

Timepoint(s) of evaluation of this end point: Up to 15 years after CTX001 infusion (with exception of LIC and CIC which will be assessed for up to 5 years).;Secondary end point(s): • Total Hemoglobin (Hb) concentration (pre-transfusion) over time • Fetal hemoglobin (HbF) concentration (pre-transfusion) over time • Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time TDT: • TDT-related transfusion • Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin • Proportion of subjects receiving iron chelation therapy over time SCD: • Severe vaso-occlusive crisis (VOC) • SCD-related transfusions

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Countries

Belgium, Canada, France, Germany, Greece, Italy, United Kingdom, United States

Contacts

Public ContactClinical Trials and Medical Info

Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com+1 877 634 8789

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026