Beta-Thalassemia study: CTX001
Official title Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
ClinicalTrials.gov ID: NCT05477563
What this study is testing
What is CTX001?
CTX001 is an investigational biological treatment being studied for transfusion‑dependent beta‑thalassemia and severe sickle cell disease.
Also referred to as Exagamglogene autotemcel, Exa‑cel.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study will look at how safe and effective the treatment is.
- Who it's for
- People who need regular blood transfusions because of beta‑thalassemia or have severe sickle cell disease.
- What's involved
- Participants receive a one‑time infusion of their own stem cells that have been edited with CRISPR‑Cas9 to produce CTX001.
- Time commitment
- The study lasts about a year with several clinic visits for monitoring.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 35
You may be able to join if
- Eligible for autologous stem cell transplant as per investigator's judgment.
- Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE).
- 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...
- Documented SCD genotypes
- History of at least two severe VOCs events per year for the previous two years prior to enrollment Key
You likely can't join if
- 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 associated α-thalassemia and \>1 alpha deletion, or alpha multiplications
- Participants with sickle cell β-thalassemia variant
- History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening Other protocol defined Inclusion/Exclusion criteria may apply.
The study team makes the final eligibility decision.
Where it is enrolling
- New York, New York, United States
- Charlotte, North Carolina, United States
- Nashville, Tennessee, United States
- Düsseldorf, Germany
- Rome, Italy
- Al Mathar Ash Shamali, Saudi Arabia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at New York, New York, United States; Charlotte, North Carolina, United States; Nashville, Tennessee, United States; Düsseldorf, Germany; Rome, Italy; Al Mathar Ash Shamali, Saudi Arabia. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.