Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

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What is this study about?

This clinical trial is focused on studying a condition known as Transfusion-Dependent β-Thalassemia, a blood disorder that requires regular blood transfusions. The study is evaluating a new treatment called CTX001, which involves using a technique known as CRISPR-Cas9 gene editing. This technique modifies the patient’s own blood stem cells to help them produce a type of hemoglobin that can reduce or eliminate the need for blood transfusions.

The purpose of the study is to assess the safety and effectiveness of a single dose of CTX001 in children with this condition. Participants will receive a dose of their own modified stem cells, which have been altered to improve their ability to produce hemoglobin. The study will monitor the participants over time to see if they can maintain healthy hemoglobin levels without needing transfusions.

In addition to CTX001, the study involves other medications such as Filgrastim, Busulfan, and Plerixafor, which are used to prepare the body for the treatment and support the process. The study will take place over several years, with regular check-ups to ensure the safety and effectiveness of the treatment. Participants will be closely monitored for any side effects or changes in their condition.

1 initial assessment

The trial begins with an initial assessment to confirm eligibility. This includes a diagnosis of transfusion-dependent thalassemia (TDT) and a history of regular red blood cell (RBC) transfusions.

A confirmation of the genotype using the study central laboratory is required before proceeding to the next step.

2 stem cell collection

Eligible participants undergo a procedure to collect their own stem cells. This is known as an autologous stem cell transplant.

The collected stem cells are modified using a technique called CRISPR-Cas9 to create CTX001, which is intended to treat TDT.

3 conditioning treatment

Before receiving CTX001, a conditioning treatment with busulfan is administered. This is done to prepare the body for the new stem cells.

Busulfan is given through an intravenous route, which means it is delivered directly into the bloodstream.

4 administration of CTX001

After conditioning, the modified stem cells (CTX001) are infused back into the participant’s body.

This is a single-dose treatment aimed at reducing the need for future RBC transfusions.

5 post-infusion monitoring

Following the infusion, participants are monitored for safety and efficacy. This includes regular check-ups and laboratory tests to assess health and response to treatment.

The primary goal is to achieve transfusion independence for at least 12 consecutive months, known as TI12.

6 follow-up assessments

Participants continue to be evaluated for secondary outcomes, such as reduction in annualized transfusions and genetic modifications in blood cells.

Safety is assessed through monitoring of adverse events, vital signs, and laboratory values.

Who Can Join the Study?

The patient must have a diagnosis of Transfusion-Dependent Thalassemia (TDT). This means they have a specific type of blood disorder that requires regular blood transfusions.
The patient must have a documented genetic condition called homozygous or compound heterozygous β-thalassemia, which includes a type called β-thalassemia/hemoglobin E (HbE). This will be confirmed by a special laboratory test before starting treatment.
The patient must have a history of receiving at least 100 milliliters of packed red blood cells (RBCs) per kilogram of body weight each year for the past two years. Packed RBCs are a type of blood transfusion.
If the patient started transfusion therapy less than two years ago, they must have needed packed RBC transfusions at least every 3 to 4 weeks for at least 6 months.
The patient must be eligible for a stem cell transplant using their own cells, as determined by the doctor in charge of the study. A stem cell transplant is a procedure where healthy cells are used to replace damaged or diseased cells.
The patient must be between the ages of 2 and 18 years old.
Both male and female patients can participate in the study.

Who Cannot Join the Study?

Patients who are not dependent on blood transfusions cannot participate.
Patients who are not children cannot participate.
Patients who do not have a condition called Thalassemia cannot participate. Thalassemia is a blood disorder that affects the body’s ability to produce hemoglobin, which is important for carrying oxygen in the blood.
Patients who are not able to receive a treatment involving their own modified stem cells cannot participate. Stem cells are special cells in the body that can develop into different types of cells.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Utpxhqjibedlzakywfmjd Drcuguilvow Atc	Duesseldorf	Germany

Trial status

Country	Status	Recruitment Start
Germany	Recruiting	01.12.2021
Italy	Recruiting	01.12.2021

Trial locations

Investigated drugs:

CTX001 is a therapy that involves using a patient’s own blood stem cells, which are modified using a technology called CRISPR-Cas9. This modification aims to help the body produce healthy blood cells, reducing or eliminating the need for blood transfusions in children with a condition called transfusion-dependent β-thalassemia.

Transfusion-Dependent Thalassemia – This is a genetic blood disorder characterized by the body’s inability to produce enough hemoglobin, which is essential for carrying oxygen in the blood. As a result, individuals with this condition often require regular blood transfusions to manage their symptoms and maintain adequate hemoglobin levels. The disease is caused by mutations in the genes responsible for hemoglobin production, leading to ineffective red blood cell formation. Over time, the need for frequent transfusions can lead to complications such as iron overload in the body. The condition is typically diagnosed in childhood and requires ongoing medical management to address the symptoms and prevent complications.

Trial ID:

2024-513349-35-00

Protocol code:

VX21-CTX001-141

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Safety and Effectiveness of CTX001 for Children with Transfusion-Dependent Beta-Thalassemia Using Exagamglogene Autotemcel and a Drug Combination

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?