CompletedPhase 2/3

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

United States59 participantsStarted 2018-09-14

Plain-language summary

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Who can participate

Age range12 Years – 35 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning
✓. History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening

What they're measuring

Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12)

Timeframe: From 60 days after last RBC transfusion up to 24 months post-CTX001 infusion]

Proportion of participants with engraftment (first day of 3 consecutive measurements of absolute neutrophil count [ANC] ≥500/µL on three different days)

Timeframe: Within 42 days after CTX001 infusion

Time to neutrophil and platelet engraftment

Timeframe: Days post-infusion to engraftment

Frequency and severity of collected adverse events (AEs)

Timeframe: Signing of informed consent through Month 24 visit

Incidence of transplant-related mortality (TRM)

Timeframe: Baseline (pre-transfusion) to 100 days and 1 year post-CTX001 infusion

All-cause mortality

Timeframe: Signing of informed consent through Month 24 visit

Trial details

NCT IDNCT03655678

SponsorVertex Pharmaceuticals Incorporated

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-11-13

View on ClinicalTrials.gov More Beta-Thalassemia trials

Who can participate

Age range12 Years – 35 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning
✓. History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening

What they're measuring

Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12)

Timeframe: From 60 days after last RBC transfusion up to 24 months post-CTX001 infusion]

Proportion of participants with engraftment (first day of 3 consecutive measurements of absolute neutrophil count [ANC] ≥500/µL on three different days)

Timeframe: Within 42 days after CTX001 infusion

Time to neutrophil and platelet engraftment

Timeframe: Days post-infusion to engraftment

Frequency and severity of collected adverse events (AEs)

Timeframe: Signing of informed consent through Month 24 visit

Incidence of transplant-related mortality (TRM)

Timeframe: Baseline (pre-transfusion) to 100 days and 1 year post-CTX001 infusion

All-cause mortality

Timeframe: Signing of informed consent through Month 24 visit