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

Recruiting

• Conditions: Sickle Cell Disease, Transfusion dependent thalassemia; MedDRA version: 21.0Level: PTClassification code: 10040641Term: Sickle cell anaemia Class: 100000004850; MedDRA version: 22.0Level: LLTClassification code: 10081904Term: Transfusion dependent thalassemia Class: 10010331; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2024-512654-19-00
• Lead Sponsor: Vertex Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-05-09
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

Subjects or legal representative or guardian (if applicable) must sign and date informed consent form (ICF)., Subjects must have received CTX001 infusion.

Exclusion Criteria

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 of CTX001 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 5 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 15 years after CTX001 infusion, CTX001-related AE's

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):TDT and SCD: Total Hemoglobin (Hb) concentration over time;Secondary end point(s):TDT and SCD: Fetal Hemoglobin (HbF) concentration over time;Secondary end point(s):TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time;Secondary end point(s):TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time;Secondary end point(s):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