BridgeMD
Tests treatment safety and results for Beta-Thalassemia
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 medicine, given as an infusion into a vein, being studied as a potential treatment for beta-thalassemia.
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
- 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.
- 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
- 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...
You likely can't join if
- 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
See the full eligibility 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...
- 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...
- 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
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- 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 - 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?
Study sites include 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 site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.