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Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

StatusActive, Not Recruiting
PhasePhase 3
Started
View on ClinicalTrials.gov ↗

Record History

  1. Primary and study completion pushed from May 31, 2026 to Nov 14, 2027v18
  2. Trial status: Recruiting to Active, Not Recruiting Enrollment target: estimated 15 to confirmed 16 · Site details revised at 6 of 6 sitesv17 Show 2 more changesShow less
    • Enrollment target: estimated 15 to confirmed 16
    • Site details revised at 6 of 6 sites
  3. Primary and study completion dates: May 2026 to May 31, 2026 Study sites revised (6)v16 Show 1 more changeShow less
    • Study sites revised (6)

    See this version in full on ClinicalTrials.gov ↗

  4. Secondary endpoints: 14 to 13v15 See this version in full on ClinicalTrials.gov ↗
  5. Site details revised at 4 of 6 sitesv14
Show 12 earlier versionsHide 12 earlier versions
  1. Secondary endpoints revised (14)v13 See this version in full on ClinicalTrials.gov ↗
  2. Re-verified, no change to tracked fieldsv12
  3. Site details revised at 1 of 6 sitesv11
  4. Site details revised at 1 of 6 sitesv10
  5. Enrollment target: 12 to 15 Secondary endpoints: 15 to 14v9 Show 1 more changeShow less
    • Secondary endpoints: 15 to 14

    See this version in full on ClinicalTrials.gov ↗

  6. Re-verified, no change to tracked fieldsv8
  7. Study sites: 5 to 6v7
  8. Study sites: 3 to 5v6
  9. Study sites: 2 to 3v5
  10. Study sites: 1 to 2v4
  11. Re-verified, no change to tracked fieldsv3
  12. Trial status: Not Yet Recruiting to Recruiting Study sites: 0 to 1 · Start date changed from estimate of Apr 2022 to May 3, 2022v2 Show 2 more changesShow less
    • Study sites: 0 to 1
    • Start date changed from estimate of Apr 2022 to May 3, 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  • Stopped: an ongoing trial was terminated, suspended or withdrawn.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 pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
Trial Details
NCT Number NCT05356195
Lead Sponsor Vertex Pharmaceuticals Incorporated
Collaborators: CRISPR Therapeutics
Conditions Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Enrollment 16 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov