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Clinical Trials/EUCTR2018-002935-88-BE
EUCTR2018-002935-88-BEActive, not recruitingPhase 1

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)

Vertex Pharmaceuticals Incorporated0 sites160 target enrollmentStarted: January 7, 2019Last updated:
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Conditions

Trial Snapshot

Phase
Phase 1
Status
Active, not recruiting
Enrollment
160
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional

Eligibility Criteria

Sex
All

Inclusion Criteria

  • •1. Subjects (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form.
  • •2. Subjects must have received CTX001 infusion in a parent study.
  • •Are the trial subjects under 18? yes
  • •Number of subjects for this age range: 34
  • •F.1.2 Adults (18-64 years) yes
  • •F.1.2.1 Number of subjects for this age range 80
  • •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.

Investigators

Similar Trials

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Phase 1
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)Transfusion-dependent ß thalassemia (TDT). Severe sickle cell disease (SCD).MedDRA version: 26.1Level: PTClassification code 10043391Term: Thalassaemia betaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 21.0Level: PTClassification code 10040641Term: Sickle cell anaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
EUCTR2018-002935-88-DEVertex Pharmaceuticals Incorporated160
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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)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 disordersMedDRA version: 21.0Level: PTClassification code 10040641Term: Sickle cell anaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
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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)MedDRA version: 20.0Level: PTClassification code 10043391Term: Thalassaemia betaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 21.0Level: PTClassification code 10040641Term: Sickle cell anaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTransfusion-dependent ß thalassemia (TDT). Severe sickle cell disease (SCD).
EUCTR2018-002935-88-GRVertex Pharmaceuticals Incorporated90
Active, not recruiting
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A Long-term Follow-up Study of Subjects With ß-thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified HematopoieticStem Cells (CTX001)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 disordersMedDRA version: 21.0Level: PTClassification code 10040641Term: Sickle cell anaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
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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)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 disordersMedDRA version: 21.0Level: PTClassification code 10040641Term: Sickle cell anaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
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