NCT05477563 Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
| NCT ID | NCT05477563 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Condition | Beta-Thalassemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 26 participants |
| Start Date | 2022-08-02 |
| Primary Completion | 2027-06-09 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 26 participants in total. It began in 2022-08-02 with a primary completion date of 2027-06-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05477563 clinical trial?
This trial is open to participants of all sexes, aged 12 Years or older, up to 35 Years, studying Beta-Thalassemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05477563 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 26 participants.
Is NCT05477563 currently recruiting?
Yes, NCT05477563 is actively recruiting participants. Contact the research team at medicalinfo@vrtx.com for enrollment information.
Where is the NCT05477563 trial being conducted?
This trial is being conducted at New York, United States, Charlotte, United States, Nashville, United States, Düsseldorf, Germany and 2 additional locations.
Who is sponsoring the NCT05477563 clinical trial?
NCT05477563 is sponsored by Vertex Pharmaceuticals Incorporated. The trial plans to enroll 26 participants.