This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649), 161(NCT05477563),171 (NCT05951205) (severe sickle cell disease \[SCD\] studies).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
160
CTX001 infusion.
Lucile Packard Children's Hospital
Palo Alto, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology
Chicago, Illinois, United States
Herbert Irving Pavilion - Hematology
New York, New York, United States
New York Presbyterian Hospital - Morgan Stanley Children's Hospital
New York, New York, United States
Levine Children's Hospital - Hematology
Charlotte, North Carolina, United States
The Children's Hospital of Philadelphia - Hematology
Philadelphia, Pennsylvania, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, United States
Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital
San Antonio, Texas, United States
Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology
Brussels, Belgium
...and 9 more locations
New malignancies
Time frame: Signing of informed consent up to 15 years post CTX001 infusion
New or worsening hematologic disorders
Time frame: Signing of informed consent up to 15 years post CTX001 infusion
All-cause mortality
Time frame: Signing of informed consent up to 15 years post CTX001 infusion
Serious adverse events (SAEs)
Time frame: Signing of informed consent up to 15 years post CTX001 infusion
CTX001-related adverse events (AEs)
Time frame: Signing of informed consent up to 15 years post CTX001 infusion
TDT and SCD: Total Hemoglobin (Hb) concentration over time
Time frame: Up to 15 years post CTX001 infusion
TDT and SCD: Fetal Hemoglobin (HbF) concentration over time
Time frame: Up to 15 years post CTX001 infusion
TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time
Time frame: Up to 15 years post CTX001 infusion
TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time
Time frame: Up to 15 years post CTX001 infusion
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
Time frame: Up to 5 years post CTX001 infusion
TDT and SCD: Change in PROs over time in participants ≥18 years of age assessed using functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) questionnaire for participants from study 111, 121, 161 and 171 only
Time frame: Up to 5 years post CTX001 infusion
TDT and SCD: Change in PROs over time in participants <18 years assessed using EQ-5D-Youth (EQ-5D-Y) from study 111,121,141,151 and 171 only
Time frame: Up to 5 years post CTX001 infusion
TDT and SCD: Change in PROs over time in participants <18 years assessed using pediatric quality of life inventory (PedsQL) Core
Time frame: Up to 5 years post CTX001 infusion
TDT: Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12)
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
TDT: Proportion of participants achieving transfusion independence for at least 6 consecutive months (TI6)
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
TDT: Proportion of participants achieving at least 95%, 90%, 85%, 75%, 50% reduction from baseline in annualized volume of RBC transfusions starting after month 10 after CTX001 infusion for participants who have not achieved TI12
Time frame: From Month 10 up to 15 years post-CTX001 infusion
TDT: Duration of transfusion free in participants who have achieved TI12
Time frame: From 60 days after last RBC transfusion up to 15 years post CTX001 infusion
TDT: Relative reduction from baseline in annualized volume of RBC transfusions starting after Month 10 after CTX001 infusion for participants who have not achieved TI12
Time frame: From Month 10 up to 15 years post-CTX001 infusion
TDT: Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin for beta-Thalassemia participants
Time frame: Up to 8 years post CTX001 infusion for LIC; up to 5 years post CTX001 infusion for CIC and up to 15 years post CTX001 infusion for ferritin
TDT: Proportion of participants receiving iron removal therapy over time
Time frame: Up to 15 years post CTX001 infusion
SCD: Proportion of participants who have not experienced any severe vaso-occlusive crises (VOC) for at least 12 consecutive months (VF12)
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
SCD: Proportion of participants with SCD free from inpatient hospitalization for severe VOCs sustained for at least 12 months (HF12)
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
SCD: Proportion of participants with at least 90 percent (%), 80%, 75% or 50% reduction in annualized rate of severe VOCs
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
SCD: Relative change from baseline in annualized rate of severe VOCs
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
SCD: Duration of severe VOC free in participants who have achieved VF12
Time frame: From 60 days after last RBC transfusion up to 15 years post CTX001 infusion
SCD: Relative change from baseline in rate of inpatient hospitalizations for severe VOCs
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
SCD: Relative change from baseline in annualized duration of hospitalization for severe VOCs
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
SCD: Proportion of participants with sustained HbF ≥20% for at least 3 months
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
SCD: Proportion of participants with sustained HbF ≥20% for at least 6 months
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
SCD: Proportion of participants with sustained HbF ≥20% for at least 12 months
Time frame: From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion
SCD: Change in volume of RBCs transfused for SCD-related indications over time
Time frame: Up to 15 years post CTX001 infusion
SCD: Change from baseline in reticulocytes/erythrocytes over time
Time frame: From baseline up to 15 years post CTX001 infusion
SCD: Change from baseline in lactate dehydrogenase (LDH) over time
Time frame: From baseline up to 15 years post CTX001 infusion
SCD: Change from baseline in haptoglobin over time
Time frame: From baseline up to 15 years post CTX001 infusion
SCD: Change from baseline in total bilirubin over time
Time frame: From baseline up to 15 years post CTX001 infusion
SCD: Change from baseline in indirect bilirubin over time
Time frame: From baseline up to 15 years post CTX001 infusion
SCD: Change in SCD-specific PROs over time in participants ≥18 years of age assessed using adult sickle cell quality of life measurement system (ASCQ-Me) (participants from Study 121,161 and 171 only)
Time frame: Up to 5 years post CTX001 infusion
SCD: Change in SCD-specific PROs over time in participants <18 years of age assessed using PedsQL Generic Core SCD module from studies 111,121,141,151,161 and 171
Time frame: Up to 5 years post CTX001 infusion
SCD: Change in PRO over time assessed using 11-point numerical rating scale (NRS)
Time frame: Up to 5 years post CTX001 infusion
SCD: Change in PROs over time assessed using Wong Baker FACES pain scale
Time frame: Up to 5 years post CTX001 infusion
SCD: Change in PROs over time using face, legs, activity, cry, consolability (FLACC) behavioral pain scale
Time frame: Up to 5 years post CTX001 infusion
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