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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

Tracking Information
First Submitted Date December 20, 2019
First Posted Date December 23, 2019
Last Update Posted Date April 20, 2023
Actual Study Start Date January 20, 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: April 29, 2022)
  • TDT and SCD: Hemoglobin (Hb) concentration over time [ Time Frame: Up to 15 years post CTX001 infusion ]
  • TDT and SCD: HbF concentration over time [ Time Frame: Up to 15 years post CTX001 infusion ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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)] ]
  • TDT: Proportion of subjects receiving iron chelation therapy over time [ Time Frame: 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) [ Time Frame: 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) [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Up to 15 years post CTX001 infusion ]
  • SCD: Change from baseline in reticulocytes/erythrocytes over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  • SCD: Change from baseline in lactate dehydrogenase (LDH) over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  • SCD: Change from baseline in haptoglobin over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  • SCD: Change from baseline in total bilirubin over time [ Time Frame: From baseline up to 15 years post CTX001 infusion ]
  • SCD: Change from baseline in indirect bilirubin over time [ Time Frame: 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) [ Time Frame: 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 [ Time Frame: Up to 5 years post CTX001 infusion ]
  • SCD: Change in PRO over time assessed using 11-point numerical rating scale (NRS) [ Time Frame: Up to 5 years post CTX001 infusion ]
  • SCD: Change in PROs over time assessed using Wong Baker FACES pain scale [ Time Frame: Up to 5 years post CTX001 infusion ]
  • 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 ]
Original 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 ]
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 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).
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 or VX21-CTX001-141 or VX21-CTX001-151) 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.
Other Names:
  • Exagamglogene autotemcel
  • Exa-cel
Study Groups/Cohorts 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.
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: April 29, 2022)		 114
Original Estimated Enrollment
 (submitted: December 20, 2019)		 90
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 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
Sex/Gender
Sexes Eligible for Study: All
Ages 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Canada,   Germany,   Italy,   United Kingdom,   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
Current Responsible Party Vertex Pharmaceuticals Incorporated
Original Responsible Party Same as current
Current Study Sponsor Vertex Pharmaceuticals Incorporated
Original Study Sponsor Same as current
Collaborators CRISPR Therapeutics
Investigators Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date April 2023