A Long-term Follow-up Study in Participants Who Received CTX001
- Conditions
- Beta-ThalassemiaSickle Cell AnemiaGenetic Diseases, InbornHemoglobinopathiesHematologic DiseasesThalassemiaSickle Cell Disease
- Interventions
- Registration Number
- NCT04208529
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 160
- Participants (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
- Participants must have received CTX001 infusion in a parent study
- There are no exclusion criteria
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description CTX001 CTX001 All participants who complete or discontinue one of the multiple parent studies (CTX001-111, CTX001-121, CTX001-141, CTX001-151, CTX001-161 and CTX001-171) after CTX001 infusion will be asked to participate in this long-term follow-up study.
- Primary Outcome Measures
Name Time Method All-cause mortality Signing of informed consent up to 15 years post CTX001 infusion Serious adverse events (SAEs) Signing of informed consent up to 15 years post CTX001 infusion CTX001-related adverse events (AEs) Signing of informed consent up to 15 years post CTX001 infusion New malignancies Signing of informed consent up to 15 years post CTX001 infusion New or worsening hematologic disorders Signing of informed consent up to 15 years post CTX001 infusion
- Secondary Outcome Measures
Name Time Method SCD: Change in PRO over time assessed using 11-point numerical rating scale (NRS) Up to 5 years post CTX001 infusion TDT and SCD: Fetal Hemoglobin (HbF) concentration over time Up to 15 years post CTX001 infusion TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time Up to 15 years post CTX001 infusion SCD: Change in PROs over time using face, legs, activity, cry, consolability (FLACC) behavioral pain scale 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 Up to 5 years post CTX001 infusion TDT: Relative reduction from baseline in annualized volume of RBC transfusions From Day 60 up to 15 years post-CTX001 infusion TDT and SCD: Total Hemoglobin (Hb) concentration over time Up to 15 years post CTX001 infusion TDT: Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12) 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) From 60 days after last RBC transfusion 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 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 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 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 Up to 15 years post CTX001 infusion SCD: Change from baseline in total bilirubin over time 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 PedsQL Generic Core SCD module from studies 111,121,141,151,161 and 171 Up to 5 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 Up to 5 years post CTX001 infusion SCD: Duration of severe VOC free in participants who have achieved VF12 From 60 days after last RBC transfusion up to 15 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 Up to 5 years post CTX001 infusion TDT: Proportion of participants achieving at least 95%, 90%, 85%, 75%, 50% reduction from baseline in annualized transfusions starting 60 days after CTX001 infusion From Day 60 up to 15 years post-CTX001 infusion TDT: Proportion of participants receiving iron chelation therapy over time Up to 15 years post CTX001 infusion SCD: Relative change from baseline in annualized rate of severe VOCs 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 From 60 days after last RBC transfusion 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) 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 Up to 5 years post CTX001 infusion TDT: Duration of transfusion free in participants who have achieved TI12 From 60 days after last RBC transfusion 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 From Up to 5 years post CTX001 infusion (for LIC and CIC) and up to 15 years post CTX001 infusion (for ferritin)] SCD: Proportion of participants who have not experienced any severe vaso-occlusive crises (VOC) for at least 12 consecutive months (VF12) 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 From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion SCD: Change from baseline in reticulocytes/erythrocytes over time From baseline up to 15 years post CTX001 infusion SCD: Change from baseline in indirect bilirubin over time From baseline 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) 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 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 From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion SCD: Change from baseline in lactate dehydrogenase (LDH) over time From baseline up to 15 years post CTX001 infusion SCD: Change from baseline in haptoglobin over time From baseline up to 15 years post CTX001 infusion SCD: Change in PROs over time assessed using Wong Baker FACES pain scale Up to 5 years post CTX001 infusion
Trial Locations
- Locations (19)
University College London Hospitals NHS Foundation Trust
🇬🇧London, United Kingdom
Lucile Packard Children's Hospital
🇺🇸Palo Alto, California, United States
Ann & Robert Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Columbia University Medical Center (21+ years)
🇺🇸New York, New York, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Atrium Health Levine Children's Hospital
🇺🇸Charlotte, North Carolina, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
St. Jude Children's Research Hospital
🇺🇸Memphis, Tennessee, United States
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers
🇺🇸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)
🇧🇪Brussels, Belgium
The Hospital for Sick Children
🇨🇦Toronto, Canada
Toronto General Hospital, University Health Network
🇨🇦Toronto, Canada
St. Paul's Hospital
🇨🇦Vancouver, Canada
University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
🇩🇪Dusseldorf, Germany
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
🇩🇪Regensburg, Germany
University Hospital Tuebingen
🇩🇪Tuebingen, Germany
Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS
🇮🇹Rome, Italy
Imperial College Healthcare NHS Trust, Hammersmith Hospital
🇬🇧London, United Kingdom