Tests a one-time gene-edited cell treatment in beta-thalassemia and sickle cell disease
Tests CTX001, gene-edited blood stem cells, in people with beta-thalassemia or sickle cell disease.
Plain-language title generated from the registry record
Registry title: Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Key facts
- Where
- Charlotte, Nashville, and New York, United StatesSee the 3 sites
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 12 and 35 years old?
Source: NCT05477563, who can take part
Where in United States
- Levine Children's Hospital - HematologyCharlotte, United StatesTaking people
- TriStar Medical Group Children's Specialists - Pediatric OncologyNashville, United StatesTaking people
- New York Presbyterian Hospital - Morgan Stanley Children's HospitalNew York, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Vertex Pharmaceuticals Incorporated
- Trial code
- NCT05477563EU 2024-514641-12-00
- Record updated
- 23 March 2026
- Original description
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.Read full original descriptionShow less
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
Inclusion: Participants with TDT and SCD:; Eligible for autologous stem cell transplant as per investigator's judgment; Participants with TDT:; Diagnosis of TDT as defined by:; 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 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening; Participants with SCD:; Diagnosis of severe SCD as defined by:; Documented SCD genotypes; History of at least two severe VOCs events per year for the previous two years prior to enrollment.
Exclusion: Prior hematopoietic stem cell transplant (HSCT); Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator; History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening; Other protocol defined Inclusion/Exclusion criteria may apply; A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement; Participants with TDT and SCD:; Participants with TDT:; Participants with associated α-thalassemia and >1 alpha deletion, or alpha multiplications; Participants with sickle cell β-thalassemia variant; Participants with SCD:.