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A Long-term Follow-up Study in Subjects Who Received CTX001

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04208529
Recruitment Status : Enrolling by invitation
First Posted : December 23, 2019
Last Update Posted : April 20, 2023
Sponsor:
Collaborator:
CRISPR Therapeutics
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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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
Beta-Thalassemia Thalassemia Sickle Cell Disease Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Sickle Cell Anemia Biological: CTX001

Study Design
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Study Type : Observational
Estimated Enrollment : 114 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)
Actual Study Start Date : January 20, 2021
Estimated Primary Completion Date : September 2039
Estimated Study Completion Date : September 2039

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Groups and Cohorts
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Group/Cohort Intervention/treatment
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:
  • Exagamglogene autotemcel
  • Exa-cel


Outcome Measures
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Primary Outcome Measures :
  1. New malignancies [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
  2. New or worsening hematologic disorders [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
  3. All-cause mortality [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
  4. Serious adverse events (SAEs) occurring up to 5 years after CTX001 infusion [ Time Frame: Signing of informed consent up to 5 years post CTX001 infusion ]
  5. CTX001-related AEs [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]

Secondary Outcome Measures :
  1. TDT and SCD: Hemoglobin (Hb) concentration over time [ Time Frame: Up to 15 years post CTX001 infusion ]
  2. TDT and SCD: HbF concentration over time [ Time Frame: Up to 15 years post CTX001 infusion ]
  3. 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 ]
  4. 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 [ Time Frame: Up to 5 years post CTX001 infusion ]
  5. 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 [ Time Frame: Up to 5 years post CTX001 infusion ]
  6. TDT and SCD: Change in PROs over time in subjects <18 years assessed using EQ-5D-Youth (EQ-5D-Y) [ Time Frame: Up to 5 years post CTX001 infusion ]
  7. TDT and SCD: Change in PROs over time in subjects <18 years assessed using pediatric quality of life inventory (PedsQL) Core [ Time Frame: Up to 5 years post CTX001 infusion ]
  8. TDT: Proportion of subjects 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 ]
  9. TDT: Proportion of subjects 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 ]
  10. TDT: Proportion of subjects achieving at least 95%, 90%, 85%, 75%, 50% reduction from baseline in annualized transfusions starting 60 days after CTX001 infusion [ Time Frame: From Day 60 up to 15 years post-CTX001 infusion ]
  11. TDT: Duration of transfusion free in subjects who have achieved TI12 [ Time Frame: From 60 days after last RBC transfusion up to 15 years post CTX001 infusion ]
  12. TDT: Relative change from baseline in transfusions starting 60 days after CTX001 infusion [ Time Frame: From Day 60 up to 15 years post-CTX001 infusion ]
  13. TDT: Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin for beta-Thalassemia subjects [ Time Frame: From Up to 5 years post CTX001 infusion (for LIC and CIC) and up to 15 years post CTX001 infusion (for ferritin)] ]
  14. TDT: Proportion of subjects receiving iron chelation therapy over time [ Time Frame: Up to 15 years post CTX001 infusion ]
  15. SCD: Proportion of subjects 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 ]
  16. SCD: Proportion of subjects 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 ]
  17. SCD: Proportion of subjects 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 ]
  18. 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 ]
  19. SCD: Duration of severe VOC free in subjects who have achieved VF12 [ Time Frame: From 60 days after last RBC transfusion up to 15 years post CTX001 infusion ]
  20. 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 ]
  21. 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 ]
  22. SCD: Proportion of subjects 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 ]
  23. SCD: Proportion of subjects 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 ]
  24. SCD: Proportion of subjects 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 ]
  25. SCD: Change in volume of RBCs transfused for SCD-related indications over time [ Time Frame: Up to 15 years post CTX001 infusion ]
  26. SCD: Change from baseline in reticulocytes/erythrocytes over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  27. SCD: Change from baseline in lactate dehydrogenase (LDH) over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  28. SCD: Change from baseline in haptoglobin over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  29. SCD: Change from baseline in total bilirubin over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  30. SCD: Change from baseline in indirect bilirubin over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  31. 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) [ Time Frame: Up to 5 years post CTX001 infusion ]
  32. SCD: Change in SCD-specific PROs over time in subjects <18 years of age assessed using PedsQL SCD module [ Time Frame: Up to 5 years post CTX001 infusion ]
  33. SCD: Change in PRO over time assessed using 11-point numerical rating scale (NRS) [ Time Frame: Up to 5 years post CTX001 infusion ]
  34. SCD: Change in PROs over time assessed using Wong Baker FACES pain scale [ Time Frame: Up to 5 years post CTX001 infusion ]
  35. 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 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
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 enrolled in the long-term follow-up study.
Criteria

Inclusion Criteria:

  • 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
  • Subjects must have received CTX001 infusion in a parent study

Exclusion Criteria:

  • There are no exclusion criteria
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04208529


Locations
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United States, New York
Columbia University Medical Center (21+ years)
New York, New York, United States, 10032
Columbia University Medical Center
New York, New York, United States, 10032
United States, North Carolina
Atrium Health Levine Children's Hospital
Charlotte, North Carolina, United States, 28203
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers
Nashville, Tennessee, United States, 37203
United States, Texas
Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital
San Antonio, Texas, United States, 78229
Canada
The Hospital for Sick Children
Toronto, Canada
Toronto General Hospital, University Health Network
Toronto, Canada
St. Paul's Hospital
Vancouver, Canada
Germany
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
Regensburg, Germany
Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin
Tuebingen, Germany
Italy
Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS
Rome, Italy
United Kingdom
Imperial College Healthcare NHS Trust, Hammersmith Hospital
London, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT04208529    
Other Study ID Numbers: CTX001-131
2018-002935-88 ( EudraCT Number )
First Posted: December 23, 2019    Key Record Dates
Last Update Posted: April 20, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

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Studies a U.S. FDA-regulated Drug Product: Yes
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Thalassemia
Hematologic Diseases
beta-Thalassemia
Hemoglobinopathies
 Genetic Diseases, Inborn
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia