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)

Phase 1

• Conditions: Transfusion-dependent ß thalassemia (TDT). Severe sickle cell disease (SCD).; MedDRA version: 20.0Level: PTClassification code 10043391Term: Thalassaemia betaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 21.0Level: PTClassification code 10040641Term: Sickle cell anaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2018-002935-88-FR
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-03-02
• Last Update Posted: 17 March 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 90

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

1. There are no exclusion criteria.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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<br>• New or worsening hematologic disorders (e.g. immune-mediated cytopenias, aplastic anemia, primary immunodeficiencies)<br>• All-cause mortality<br>• All serious adverse events (SAEs) occurring up to 5 years after CTX001 infusion<br>• 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).

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Total Hemoglobin (Hb) concentration (pre-transfusion) over time<br>• Fetal hemoglobin (HbF) concentration (pre-transfusion) over time<br>• Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time<br><br>TDT:<br>• TDT-related transfusion<br>• Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin<br>• Proportion of subjects receiving iron chelation therapy over time<br><br>SCD:<br>• Severe vaso-occlusive crisis (VOC)<br>• SCD-related transfusions;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).