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Trial · NCT05951205

Registered outcomes32

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

ListOutcomeTime frameDescription
PrimaryProportion of Participants with an Average Fetal Hemoglobin (HbF) Greater Than or Equal To (>=) 20 percent (%) on or After 6 MonthsFrom 60 Days after Last Red Blood Cell (RBC) transfusion up to 24 Months after exa-cel infusion
SecondaryNumber of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)From Signing of Informed Consent up to 24 Months After exa-cel Infusion
SecondaryProportion of Participants With Neutrophil Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count (ANC) >=500 per Microliter [mcgL] on 3 Different Days)Within 42 Days After exa-cel Infusion
SecondaryTime to Neutrophil EngraftmentUp to 24 months After exa-cel Infusion
SecondaryTime to Platelet EngraftmentUp to 24 months After exa-cel Infusion
SecondaryIncidence of Transplant-Related Mortality (TRM)Up to 100 Days After exa-cel Infusion
SecondaryIncidence of Transplant-Related Mortality (TRM)Within 12 Months After exa-cel Infusion
SecondaryIncidence of All-cause MortalityFrom Signing of Informed Consent up to 24 Months After exa-cel Infusion
SecondaryProportion of Participants With No Severe Vaso-Occlusive Crises (VOCs) for At least 12 Months (VF12)From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
SecondaryProportion of Participants Free from Inpatient Hospitalization For Severe VOCs Sustained for At least 12 Months (HF12)From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
SecondaryRelative Reduction in Annualized Rate of Severe VOCsFrom Baseline up to 24 Months After exa-cel Infusion
SecondaryDuration of Severe VOC Free in Participants who Have Achieved VF12From 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
SecondaryRelative Reduction in Rate of Inpatient Hospitalizations for Severe VOCsFrom Baseline up to 24 Months After exa-cel Infusion
SecondaryRelative Reduction in Annualized Duration of Hospitalization for Severe VOCsFrom Baseline up to 24 Months After exa-cel Infusion
SecondaryProportion of Participants With Sustained HbF >= 20 % for At least 3, 6, or 12 monthsFrom 60 Days after Last RBC transfusion up to 24 Months After exa-cel Infusion
SecondaryRelative Reduction in Annualized Volume of RBC TransfusionsFrom Baseline Up To 24 Months After exa-cel Infusion
SecondaryHbF Concentration Over TimeUp To 24 Months After exa-cel Infusion
SecondaryTotal Hemoglobin (Hb) Concentration Over TimeUp To 24 Months After exa-cel Infusion
SecondaryChange In Reticulocyte Count Over TimeFrom Baseline Up To 24 Months After exa-cel Infusion
SecondaryChange in Indirect Bilirubin Over TimeFrom Baseline Up To 24 Months After exa-cel 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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