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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04208529
Recruitment Status : Not yet recruiting
First Posted : December 23, 2019
Last Update Posted : December 23, 2019
Sponsor:
Collaborator:
CRISPR Therapeutics
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

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
Beta-Thalassemia Thalassemia Sickle Cell Disease Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Sickle Cell Anemia

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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)
Estimated Study Start Date : February 2021
Estimated Primary Completion Date : September 2039
Estimated Study Completion Date : September 2039


Group/Cohort
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.



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 ]


Information from the National Library of Medicine

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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

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


Contacts
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Contact: Medical Information 6173416777 medicalinfo@vrtx.com

Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

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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: December 23, 2019
Last Verified: December 2019
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
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