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Trial · NCT05356195

Registered outcomes14

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

ListOutcomeTime frameDescription
PrimaryProportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)Up to 24 Months After CTX001 Infusion
SecondaryProportion of Participants Achieving at Least 95 Percent (%), 90%, 85%, 75% and 50% Reduction in Annualized TransfusionsFrom Baseline up to 24 Months After CTX001 Infusion
SecondaryTransfusion Free Duration for Participants who Achieve TI12Up to 24 Months After CTX001 Infusion
SecondaryProportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over TimeUp to 24 Months After CTX001 Infusion
SecondaryProportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over TimeUp to 24 Months After CTX001 Infusion
SecondaryChange in Fetal Hemoglobin Concentration Over TimeFrom Baseline (Pre-transfusion) up to 24 Months After CTX001 Infusion
SecondaryChange in Total Hemoglobin Concentration Over TimeFrom Baseline (Pre-transfusion) up to 24 Months After CTX001 Infusion
SecondaryNumber of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)From Signing of Informed Consent up to 24 Months After CTX001 Infusion
SecondaryProportion of Participants With Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count [ANC] ≥500 per Microliter [mcgL] on 3 Different Days)Within 42 Days After CTX001 Infusion
SecondaryTime to EngraftmentUp to 24 Months After CTX001 Infusion
SecondaryIncidence of Transplant-related Mortality (TRM) Within 100 Days After CTX001 InfusionWithin 100 Days After CTX001 Infusion
SecondaryIncidence of TRM Within 12 Months After CTX001 InfusionWithin 12 Months After Infusion
SecondaryIncidence of All-cause MortalityFrom Signing of Informed Consent up to 24 Months After CTX001 Infusion
SecondaryRelative Reduction in Annualized Volume and Episodes of RBC Transfusions starting Month 10 After CTX001 infusionFrom Baseline up to 24 Months After CTX001 Infusion

Source ClinicalTrials.govtrials-2026-11-12-0.5.0

Sources 3

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ClinicalTrials.govNo license stated
  • NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ClinicalTrials.govnone stated
  • NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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