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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 : July 23, 2020
Sponsor:
Collaborator:
CRISPR Therapeutics
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date December 20, 2019
First Posted Date December 23, 2019
Last Update Posted Date July 23, 2020
Estimated Study Start Date February 2021
Estimated Primary Completion Date September 2039   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: December 20, 2019)
  • New malignancies [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
  • New or worsening hematologic disorders [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
  • All-cause mortality [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
  • 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 ]
  • CTX001-related AEs [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: December 20, 2019)
  • Hemoglobin (Hb) concentration over time [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]
  • HbF concentration over time [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]
  • 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 ]
  • Transfusion dependent thalassemia (TDT) related transfusions for beta-Thalassemia subjects [ Time Frame: Signing of informed consent up to 15 years post CTX001 infusion ]
  • 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) ]
  • Proportion of subjects receiving iron chelation therapy over time [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]
  • 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 ]
  • Sickle cell disease (SCD) related transfusions for SCD subjects [ Time Frame: From Day 1 up to 15 years post CTX001 infusion ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Long-term Follow-up Study in Subjects Who Received CTX001
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)
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).
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition
  • Beta-Thalassemia
  • Thalassemia
  • Sickle Cell Disease
  • Hematologic Diseases
  • Hemoglobinopathies
  • Genetic Diseases, Inborn
  • Sickle Cell Anemia
Intervention Biological: CTX001
CTX001 infusion
Study Groups/Cohorts 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.
Intervention: Biological: CTX001
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Enrolling by invitation
Estimated Enrollment
 (submitted: December 20, 2019)
90
Original Estimated Enrollment Same as current
Estimated Study Completion Date September 2039
Estimated Primary Completion Date September 2039   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Germany,   Italy,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04208529
Other Study ID Numbers CTX001-131
2018-002935-88 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Product Manufactured in and Exported from the U.S.: Yes
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
Responsible Party Vertex Pharmaceuticals Incorporated
Study Sponsor Vertex Pharmaceuticals Incorporated
Collaborators CRISPR Therapeutics
Investigators Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date February 2020