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Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

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

Record History

  1. Primary and study completion pushed from May 31, 2026 to Jun 6, 2027v16
  2. Trial status: Recruiting to Active, Not Recruiting Enrollment target: estimated 15 to confirmed 13 · Site details revised at 7 of 7 sitesv15 Show 2 more changesShow less
    • Enrollment target: estimated 15 to confirmed 13
    • Site details revised at 7 of 7 sites
  3. Primary and study completion dates: May 2026 to May 31, 2026 Study sites revised (7)v14 Show 1 more changeShow less
    • Study sites revised (7)

    See this version in full on ClinicalTrials.gov ↗

  4. Site details revised at 3 of 7 sitesv13
  5. Secondary endpoints revised (30)v12 See this version in full on ClinicalTrials.gov ↗
Show 10 earlier versionsHide 10 earlier versions
  1. Re-verified, no change to tracked fieldsv11
  2. Study sites: 6 to 7v10
  3. Site details revised at 3 of 6 sitesv9
  4. Enrollment target: 12 to 15 Secondary endpoints: 31 to 30 · Eligibility criteria shortened, about 5 wordsv8 Show 2 more changesShow less
    • Secondary endpoints: 31 to 30
    • Eligibility criteria shortened, about 5 words

    See this version in full on ClinicalTrials.gov ↗

  5. Study sites: 5 to 6v7
  6. Study sites: 4 to 5v6
  7. Study sites: 2 to 4v5
  8. Site details revised at 1 of 2 sitesv4
  9. Study sites: 1 to 2v3
  10. Trial status: Not Yet Recruiting to Recruiting Study sites: 0 to 1 · Start date changed from estimate of Apr 2022 to May 2, 2022v2 Show 2 more changesShow less
    • Study sites: 0 to 1
    • Start date changed from estimate of Apr 2022 to May 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  • Stopped: an ongoing trial was terminated, suspended or withdrawn.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 pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. 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 NCT05329649
Lead Sponsor Vertex Pharmaceuticals Incorporated
Collaborators: CRISPR Therapeutics
Conditions Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
Enrollment 13 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov