A Long-term Follow-up Study in Subjects Who Received CTX001
Study Details
Study Description
Brief Summary
This is a multi-site, observational study to evaluate the long-term safety and efficacy of CTX001 in subjects who received CTX001 in Study CTX001-111 (NCT03655678) or VX21-CTX001-141 (transfusion-dependent β-thalassemia [TDT] studies) or Study CTX001-121 (NCT03745287) or VX21-CTX001-151 (severe sickle cell disease [SCD] studies; NCT05329649).
Condition or Disease | Intervention/Treatment | Phase |
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Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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CTX001 All subjects who complete or discontinue the parent study (CTX001-111 or CTX001-121 or VX21-CTX001-141 or VX21-CTX001-151) after CTX001 infusion will be asked to participate in this long-term follow-up study. |
Biological: CTX001
CTX001 infusion.
Other Names:
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Outcome Measures
Primary Outcome Measures
- 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]
- All-cause mortality [Signing of informed consent up to 15 years post CTX001 infusion]
- Serious adverse events (SAEs) occurring up to 5 years after CTX001 infusion [Signing of informed consent up to 5 years post CTX001 infusion]
- CTX001-related AEs [Signing of informed consent up to 15 years post CTX001 infusion]
Secondary Outcome Measures
- TDT and SCD: Hemoglobin (Hb) concentration over time [Up to 15 years post CTX001 infusion]
- TDT and SCD: 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]
- TDT and SCD: Change in patient-reported outcome (PRO) over time in subjects ≥18 years of age assessed using EuroQol quality of life scale (EQ-5D-5L) for subjects from study CTX001-111 and study CTX001-121 only [Up to 5 years post CTX001 infusion]
- TDT and SCD: Change in PROs over time in subjects ≥18 years of age assessed using functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) questionnaire for subjects from study CTX001-111 and study CTX001-121 only [Up to 5 years post CTX001 infusion]
- TDT and SCD: Change in PROs over time in subjects <18 years assessed using EQ-5D-Youth (EQ-5D-Y) [Up to 5 years post CTX001 infusion]
- TDT and SCD: Change in PROs over time in subjects <18 years assessed using pediatric quality of life inventory (PedsQL) Core [Up to 5 years post CTX001 infusion]
- TDT: Proportion of subjects 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 subjects 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: Proportion of subjects 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: Duration of transfusion free in subjects who have achieved TI12 [From 60 days after last RBC transfusion up to 15 years post CTX001 infusion]
- TDT: Relative change from baseline in transfusions starting 60 days after CTX001 infusion [From Day 60 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 subjects [From Up to 5 years post CTX001 infusion (for LIC and CIC) and up to 15 years post CTX001 infusion (for ferritin)]]
- TDT: Proportion of subjects receiving iron chelation therapy over time [Up to 15 years post CTX001 infusion]
- SCD: Proportion of subjects 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 subjects 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: Proportion of subjects 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 annualized rate of severe VOCs [From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion]
- SCD: Duration of severe VOC free in subjects who have achieved VF12 [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: 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 subjects with sustained HbF ≥20% for at least 3 months [From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion]
- SCD: Proportion of subjects with sustained HbF ≥20% for at least 6 months [From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion]
- SCD: Proportion of subjects 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 volume of RBCs transfused for SCD-related indications over time [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 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 from baseline in total bilirubin 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: Change in SCD-specific PROs over time in subjects ≥18 years of age assessed using adult sickle cell quality of life measurement system (ASCQ-Me) (subjects from Study 121 only) [Up to 5 years post CTX001 infusion]
- SCD: Change in SCD-specific PROs over time in subjects <18 years of age assessed using PedsQL SCD module [Up to 5 years post CTX001 infusion]
- SCD: Change in PRO over time assessed using 11-point numerical rating scale (NRS) [Up to 5 years post CTX001 infusion]
- SCD: Change in PROs over time assessed using Wong Baker FACES pain scale [Up to 5 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]
Eligibility Criteria
Criteria
Inclusion Criteria:
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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
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Subjects must have received CTX001 infusion in a parent study
Exclusion Criteria:
- There are no exclusion criteria
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
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1 | Columbia University Medical Center (21+ years) | New York | New York | United States | 10032 |
2 | Columbia University Medical Center | New York | New York | United States | 10032 |
3 | St. Jude Children's Research Hospital | Memphis | Tennessee | United States | 38105 |
4 | The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers | Nashville | Tennessee | United States | 37203 |
5 | Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital | San Antonio | Texas | United States | 78229 |
6 | The Hospital for Sick Children | Toronto | Canada | ||
7 | Toronto General Hospital, University Health Network | Toronto | Canada | ||
8 | St. Paul's Hospital | Vancouver | Canada | ||
9 | Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine | Regensburg | Germany | ||
10 | Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin | Tuebingen | Germany | ||
11 | Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS | Rome | Italy | ||
12 | Imperial College Healthcare NHS Trust, Hammersmith Hospital | London | United Kingdom |
Sponsors and Collaborators
- Vertex Pharmaceuticals Incorporated
- CRISPR Therapeutics
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CTX001-131
- 2018-002935-88