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RecruitingPhase 3

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
12 Years to 35 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests how safe a single dose of CTX001 is and how well it works. CTX001 uses a person's own blood-forming stem cells, with genes changed using a tool called CRISPR-Cas9. It is for people with beta-thalassemia who need regular blood transfusions or people with 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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • For either disease: The study doctor must decide you can have a stem cell transplant using your own cells.
  • For beta-thalassemia: You must have a confirmed inherited type listed in the study, including beta-thalassemia with hemoglobin E. The study lab must confirm your gene type before transplant preparation treatment.
  • For beta-thalassemia: You must have received at least 100 milliliters of red blood cells per kilogram of body weight, or 10 units of red blood cells, each year for the past 2 years.
  • For sickle cell disease: You must have a documented sickle cell gene type.
  • For sickle cell disease: You must have had at least 2 severe crises caused by blocked blood flow each year for the past 2 years.
  • Other study rules may also apply.

Who can't join

  • For either disease: A healthy relative is willing to donate stem cells and is a full tissue match, as judged by the study doctor.
  • For either disease: You have already had a blood-forming stem cell transplant.
  • For either disease: You have an active, medically important infection, as judged by the study doctor.
  • For beta-thalassemia: You also have alpha-thalassemia with more than one missing alpha gene, or extra copies of alpha genes.
  • For beta-thalassemia: You have the sickle cell beta-thalassemia type.
  • For sickle cell disease: You have a history of untreated moyamoya syndrome, which narrows blood vessels in the brain, or have it at screening.
  • Other study rules may also apply.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Key Inclusion Criteria: * 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 Key Exclusion Criteria: * Participants with TDT and SCD: * A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement * Prior hematopoietic stem cell transplant (HSCT) * Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator * 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: * History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening Other protocol defined Inclusion/Exclusion criteria may apply.

Study sites (3)

NC

NY

TN