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CTX001 for Transfusion-Dependent Beta-Thalassemia and Severe Sickle Cell Disease

This study is looking at a single dose of CTX001 for people aged 12 to 35 with transfusion-dependent beta-thalassemia (TDT) or severe sickle cell disease (SCD). CTX001 is a treatment that uses your own modified blood stem cells. Researchers want to see how safe and effective this treatment is. To be eligible, you must be considered suitable for a stem cell transplant by your doctor. For those with TDT, you need a confirmed diagnosis of homozygous beta-thalassemia or compound heterozygous beta-thalassemia. The study will measure changes in your fetal hemoglobin (HbF) and total hemoglobin (Hb) levels for up to 12 months after receiving CTX001 to see if it helps reduce the need for blood transfusions. The study plans to enroll 26 participants, but its current status is unclear.

Study design
This is an open-label study, meaning both you and the researchers will know what treatment you are receiving. It plans to enroll 26 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
You will be followed for up to 12 months after receiving the CTX001 infusion.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05477563

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

Recruiting
PHASE3Ages 12–35InterventionalTreatment
Vertex Pharmaceuticals Incorporated
~26 participants
Updated 2026-03-23 on ClinicalTrials.gov
What's tested:CTX001

At a glance

Recruiting sites
6 of 6 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Fetal Hemoglobin (HbF) Concentration Over Time
Measured over Up to 12 Months After CTX001 Infusion
+1 more outcome measured

Conditions

Beta-Thalassemia
Thalassemia
Hematologic Diseases
Genetic Diseases, Inborn
Hemoglobinopathies
Sickle Cell Disease
Sickle Cell Anemia

Explore related trials

Beta ThalassemiaSickle Cell Disease

Where it's being run

6 sites across 6 states
New York1
North Carolina1
Tennessee1
Germany1
Italy1
Saudi Arabia1

Who to contact

Medical Information
[email protected]
6173416777
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

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

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

What this trial measures

  • Fetal Hemoglobin (HbF) Concentration Over TimeUp to 12 Months After CTX001 Infusion
  • Total Hemoglobin (Hb) Concentration Over TimeUp to 12 Months After CTX001 Infusion