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-GR
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-06-11
• Last Update Posted: 26 October 2020

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