Trial · NCT04208529
Registered outcomes41
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 41
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | New malignancies | Signing of informed consent up to 15 years post CTX001 infusion | |
| Primary | New or worsening hematologic disorders | Signing of informed consent up to 15 years post CTX001 infusion | |
| Primary | All-cause mortality | Signing of informed consent up to 15 years post CTX001 infusion | |
| Primary | Serious adverse events (SAEs) | Signing of informed consent up to 15 years post CTX001 infusion | |
| Primary | CTX001-related adverse events (AEs) | Signing of informed consent up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Total Hemoglobin (Hb) concentration over time | Up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Fetal Hemoglobin (HbF) concentration over time | Up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time | Up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time | Up to 15 years post CTX001 infusion | |
| Secondary | TDT and SCD: Change in patient-reported outcome (PRO) over time in participants ≥18 years of age assessed using EuroQol quality of life scale (EQ-5D-5L) for participants from study 111 and 121 only | Up to 5 years post CTX001 infusion | |
| Secondary | TDT and SCD: Change in PROs over time in participants ≥18 years of age assessed using functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) questionnaire for participants from study 111, 121 and 161 only | Up to 5 years post CTX001 infusion | |
| Secondary | TDT and SCD: Change in PROs over time in participants <18 years assessed using EQ-5D-Youth (EQ-5D-Y) from study 111,121,141 and 151 only | Up to 5 years post CTX001 infusion | |
| Secondary | TDT and SCD: Change in PROs over time in participants <18 years assessed using pediatric quality of life inventory (PedsQL) Core | Up to 5 years post CTX001 infusion | |
| Secondary | TDT: Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12) | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion | |
| Secondary | TDT: Proportion of participants achieving transfusion independence for at least 6 consecutive months (TI6) | From 60 days after last RBC transfusion up to 15 years post-CTX001 infusion | |
| Secondary | TDT: Proportion of participants achieving at least 95%, 90%, 85%, 75%, 50% reduction from baseline in annualized volume of RBC transfusions starting after month 10 after CTX001 infusion for participants who have not achieved TI12 | From Month 10 up to 15 years post-CTX001 infusion | |
| Secondary | TDT: Duration of transfusion free in participants who have achieved TI12 | From 60 days after last RBC transfusion up to 15 years post CTX001 infusion | |
| Secondary | TDT: Relative reduction from baseline in annualized volume of RBC transfusions starting after Month 10 after CTX001 infusion for participants who have not achieved TI12 | From Month 10 up to 15 years post-CTX001 infusion | |
| Secondary | TDT: Iron overload as measured by liver iron concentration (LIC), cardiac iron concentration (CIC), and ferritin for beta-Thalassemia participants | Up to 8 years post CTX001 infusion for LIC; up to 5 years post CTX001 infusion for CIC and up to 15 years post CTX001 infusion for ferritin | |
| Secondary | TDT: Proportion of participants receiving iron removal therapy over time | Up to 15 years post 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.
For software
Read this list from software.
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GET https://conductscience.org/api/trials/v1/NCT04208529/registered