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Trial · NCT05329649

Registered outcomes31

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

ListOutcomeTime frameDescription
PrimaryProportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months (VF12)Up to 24 Months After CTX001 Infusion
SecondaryProportion of Participants Free from Inpatient Hospitalization for Severe VOCs for at Least 12 Months (HF12)Up to 24 Months After CTX001 Infusion
SecondaryRelative Reduction in Annualized Rate of Severe VOCsFrom Baseline up to 24 Months After CTX001 Infusion
SecondaryDuration of Severe VOC Free in Participants who Have Achieved VF12Up to 24 Months After CTX001 Infusion
SecondaryRelative Reduction in Annualized Rate of Inpatient Hospitalizations for Severe VOCsFrom Baseline up to 24 Months After CTX001 Infusion
SecondaryProportion of Participants With Sustained Fetal Hemoglobin (HbF) ≥20 Percent (%) for at Least 3 MonthsUp to 24 Months After CTX001 Infusion
SecondaryProportion of Participants With Sustained HbF ≥20% for at Least 6 MonthsUp to 24 Months After CTX001 Infusion
SecondaryProportion of Participants With Sustained HbF ≥20% for at Least 12 MonthsUp 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
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 Duration of Hospitalization for Severe VOCsFrom Baseline up to 24 Months After CTX001 Infusion
SecondaryProportion of Participants With Sustained HbF ≥30% for at Least 3 MonthsUp to 24 Months After CTX001 Infusion
SecondaryProportion of Participants With Sustained HbF ≥30% for at Least 6 MonthsUp to 24 Months After CTX001 Infusion
SecondaryProportion of Participants With Sustained HbF ≥30% for at Least 12 MonthsUp 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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