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A Long-term Follow-up Study in Participants 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 : March 6, 2024
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, 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).

Condition or disease Intervention/treatment Phase
Beta-Thalassemia Thalassemia Sickle Cell Disease Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Sickle Cell Anemia Biological: CTX001 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Long Term Safety Follow-up
Masking: None (Open Label)
Primary Purpose: Other
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

Arms and Interventions
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Arm Intervention/treatment
Experimental: 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.
 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) [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
  5. CTX001-related adverse events (AEs) [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]

Secondary Outcome Measures :
  1. TDT and SCD: Total Hemoglobin (Hb) concentration over time [ Time Frame: Up to 15 years post CTX001 infusion ]
  2. TDT and SCD: Fetal Hemoglobin (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: 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 ]
  5. 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 ]
  6. 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 ]
  7. 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 ]
  8. 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 ]
  9. 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 ]
  10. 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 ]
  11. TDT: Proportion of participants 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 ]
  12. 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 ]
  13. TDT: Relative reduction from baseline in annualized volume of RBC transfusions [ Time Frame: From Day 60 up to 15 years post-CTX001 infusion ]
  14. TDT: Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin for beta-Thalassemia participants [ Time Frame: From Up to 5 years post CTX001 infusion (for LIC and CIC) and up to 15 years post CTX001 infusion (for ferritin)] ]
  15. TDT: Proportion of participants receiving iron chelation therapy over time [ Time Frame: Up to 15 years post CTX001 infusion ]
  16. 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 ]
  17. 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 ]
  18. 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 ]
  19. 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 ]
  20. 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 ]
  21. 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 ]
  22. 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 ]
  23. 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 ]
  24. 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 ]
  25. 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 ]
  26. SCD: Change in volume of RBCs transfused for SCD-related indications over time [ Time Frame: Up to 15 years post CTX001 infusion ]
  27. SCD: Change from baseline in reticulocytes/erythrocytes over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  28. SCD: Change from baseline in lactate dehydrogenase (LDH) over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  29. SCD: Change from baseline in haptoglobin over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  30. SCD: Change from baseline in total bilirubin over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  31. SCD: Change from baseline in indirect bilirubin over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  32. 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 ]
  33. 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 ]
  34. SCD: Change in PRO over time assessed using 11-point numerical rating scale (NRS) [ Time Frame: Up to 5 years post CTX001 infusion ]
  35. SCD: Change in PROs over time assessed using Wong Baker FACES pain scale [ Time Frame: Up to 5 years post CTX001 infusion ]
  36. 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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
Criteria

Inclusion Criteria:

  • 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

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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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: March 6, 2024
Last Verified: December 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
Studies a U.S. FDA-regulated Device Product: No
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