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

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ClinicalTrials.gov Identifier: NCT04208529
Recruitment Status : Enrolling by invitation
First Posted : December 23, 2019
Last Update Posted : February 15, 2021
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 Study CTX001-121 (NCT03745287).

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 : 90 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 25, 2021
Estimated Primary Completion Date : September 2039
Estimated Study Completion Date : September 2039

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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) after CTX001 infusion will be asked to participate in this long-term follow-up study.
 Biological: CTX001
CTX001 infusion


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. Hemoglobin (Hb) concentration over time [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]
  2. HbF concentration over time [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]
  3. Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]
  4. Transfusion dependent thalassemia (TDT) related transfusions for beta-Thalassemia subjects [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
  5. Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin for beta-Thalassemia subjects [ Time Frame: From Day 1 up to 5 years post CTX001 infusion (for LIC and CIC) and up to 15 years post CTX001 infusion (for ferritin) ]
  6. Proportion of subjects receiving iron chelation therapy over time [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]
  7. Annualized rate of severe vaso-occlusive crises (VOC) events for severe sickle cell disease (SCD) subjects [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]
  8. Sickle cell disease (SCD) related transfusions for SCD subjects [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (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) after CTX001 infusion will be enrolled in the long-term follow-up study.
Criteria

Inclusion Criteria:

  • Subjects or legal representative or guardian (if applicable) must sign and date informed consent form (ICF)
  • Subjects must have received CTX001 infusion

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, 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
Germany
Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
Regensburg, Germany
Italy
Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS
Rome, Italy
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: February 15, 2021
Last Verified: February 2021
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