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Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

StatusRecruiting
PhasePhase 3
Started
View on ClinicalTrials.gov ↗

Record History

  1. Re-verified, no change to tracked fieldsv14
  2. Study sites revised (6)v13 See this version in full on ClinicalTrials.gov ↗
  3. Primary and study completion dates: Feb 2025 to Jun 9, 2027 Study sites revised (6) · Collaborators shortened, about 2 wordsv12 Show 2 more changesShow less
    • Study sites revised (6)
    • Collaborators shortened, about 2 words

    See this version in full on ClinicalTrials.gov ↗

  4. Study sites: 5 to 6v11
  5. Enrollment target: 18 to 26v10
Show 8 earlier versionsHide 8 earlier versions
  1. Re-verified, no change to tracked fieldsv9
  2. Site details revised at 1 of 5 sitesv8
  3. Enrollment target: 12 to 18v7
  4. Study sites: 4 to 5v6
  5. Secondary endpoints revised (17)v5 See this version in full on ClinicalTrials.gov ↗
  6. Study sites: 3 to 4v4
  7. Study sites: 1 to 3v3
  8. Trial status: Not Yet Recruiting to Recruiting Study sites: 0 to 1 · Start date changed from estimate of Jul 2022 to Aug 2, 2022v2 Show 2 more changesShow less
    • Study sites: 0 to 1
    • Start date changed from estimate of Jul 2022 to Aug 2, 2022
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
Trial Details
NCT Number NCT05477563
Lead Sponsor Vertex Pharmaceuticals Incorporated
Conditions Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Enrollment 26 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov