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
Vertex Pharmaceuticals Incorporated
26 participants
Aug 2, 2022
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria10
- 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 Criteria9
- 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
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Interventions
Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan
Locations(6)
View Full Details on ClinicalTrials.gov
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NCT05477563