Transfusion-dependent ß thalassemia (TDT). Severe sickle cell disease (SCD). MedDRA version: 26.1 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
Conditions
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
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
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 severe 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) • CTX001-related AEs ;Timepoint(s) of evaluation of this end point: Up to 15 years after CTX001 infusion | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): TDT and SCD: • Total Hemoglobin (Hb) concentration over time • Fetal hemoglobin (HbF) concentration over time • Proportion of alleles with intended genetic modification present in peripheral blood over time • Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time • Change in patient-reported outcomes (PROs) over time in subject = 18 years of age using: o EuroQol Questionnaire – 5 dimensions – 5 levels of severity (EQ-5D-5L; subjects from Study 111 and Study 121, and Study 171 only) o Functional assessment of cancer therapy bone marrow transplant (FACT-BMT) (subjects from Study 111, Study 121, Study 161, and 171 only) • Change in PROs over time in subjects <18 years of age using: o EuroQol Questionnaire – 5 dimensions – youth (EQ-5D-Y; selfcomplete and proxy versions based on age; subjects from Study 111, Study 121, Study 141, Study 151^, and Study 171 only) o Pediatric Quality of Life Inventory (PedsQL) Genetic Core (self-complete and proxy versions based on age; all studies) TDT only: • Proportion of subjects achieving TI12, defined as maintaining weighted average Hb =9 g/dL without RBC transfusions for at least 12 consecutive months any time after CTX001 infusion. The evaluation of TI12 starts 60 days after last RBC transfusion for post-transplant support or TDT disease management. • Proportion of subjects achieving TI6, defined as maintaining weighted average Hb =9 g/dL without RBC transfusions for at least 6 consecutive months any time after CTX001 infusion. The evaluation of TI6 starts 60 days after last RBC transfusion for post-transplant support or TDT disease management. • Proportion of subjects achieving at least 95%, 90%, 85%, 75%, 50% reduction from baseline in annualized transfusions starting 60 days after CTX001 infusion. • Duration transfusion free in subjects who have achieved TI12 • Relative reduction from baseline in annualized volume of RBC transfusion • Iron overload as | — |
Countries
Belgium, Canada, France, Germany, Greece, Italy, United Kingdom, United States
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed