Active not recruitingPHASE3INTERVENTIONAL

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

This study is investigating a new treatment called CTX001 for children with a serious blood disorder known as beta-thalassemia. Beta-thalassemia makes the body unable to produce enough healthy red blood cells, often requiring lifelong regular blood transfusions. CTX001 is a type of gene therapy that aims to correct the genetic fault causing the condition. This study is for children aged 2 to 11 who are dependent on blood transfusions. Doctors will carefully monitor the children who receive this one-time treatment to understand how safe it is and how well it works. The goal is to see if CTX001 can help children produce their own healthy blood cells and reduce or even eliminate their need for frequent blood transfusions.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Vertex Pharmaceuticals Incorporated

Enrolment target

Start

03 May 2022

Estimated completion

31 May 2026

Beta-Thalassemia Thalassemia Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies

What is this study about?

This study is looking at a new and advanced treatment called CTX001 for children who have a blood condition called beta-thalassemia. Beta-thalassemia is an inherited disorder where the body doesn't make enough healthy red blood cells. These cells are really important because they carry oxygen around the body. Because of this, children with severe beta-thalassemia often need regular blood transfusions, sometimes every few weeks, to stay healthy. These transfusions can be life-saving but also come with challenges over time.

CTX001 is a special type of treatment that works by changing a patient's own blood-forming stem cells. These are the powerful cells in the bone marrow that make all other blood cells. Scientists use a technique called CRISPR-Cas9, which you might have heard of as 'gene editing,' to fix the problem in these cells before putting them back into the child. The hope is that these 'edited' cells will then start making healthy red blood cells, which could greatly reduce or even stop the need for future blood transfusions.

This is a 'Phase 3' study, which means it's one of the last steps before a treatment might become more widely available if it proves safe and effective. It's an 'open-label' study, which means everyone involved (doctors, patients, and families) will know that the child is receiving the CTX001 treatment. The main goals are to carefully watch the children to understand how safe the treatment is and to see how well it works in helping them produce their own healthy blood cells.

Key takeaways

This study is testing a new gene-editing treatment (CTX001) for beta-thalassemia.
It's for children aged 2-11 who regularly need blood transfusions.
The treatment involves modifying a child's own stem cells.
The main goals are to check the treatment's safety and effectiveness.
Participation includes a single treatment and long-term follow-up.
The study aims to reduce or end the need for blood transfusions.

Who may be eligible?

To be considered for this study, children need to be between 2 and 11 years old and have a confirmed diagnosis of transfusion-dependent beta-thalassemia. This means they regularly need blood transfusions.

There are also some specific requirements about their past transfusions, for example, having received a certain amount of blood over the past two years, or needing transfusions regularly for at least six months. The doctors would also need to agree that the child is suitable for a 'stem cell transplant,' which is part of this treatment process.

Children would not be able to join the study if they have a fully matched family donor available for a traditional bone marrow transplant, or if they have had a stem cell transplant before. Other reasons for not being able to join include certain types of genetic variations that affect alpha-thalassemia, having specific forms of sickle cell beta-thalassemia, or having an active serious infection at the time of screening.

Quick self-check

Is your child between 2 and 11 years old?
Does your child have a confirmed diagnosis of transfusion-dependent beta-thalassemia?
Does your child regularly need blood transfusions?
Does your child NOT have a fully matched family donor for a standard bone marrow transplant?
Has your child NOT had a stem cell transplant before?
Is your child generally well at the moment without serious active infections?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your child joins this study, they will receive a single treatment of CTX001. Before this, they will have several appointments for tests and checks to make sure they are suitable. This will involve blood tests and other medical assessments. The treatment itself involves a process where their own blood stem cells are collected, sent away to be modified, and then given back to them through an infusion, similar to a blood transfusion. Before receiving the modified cells, they will need a conditioning treatment, which is a type of medicine given to prepare their body. After the treatment, there will be a long period of follow-up visits, which will include regular blood tests and other monitoring to check their health, how well the treatment is working, and for any side effects. The total duration of follow-up for safety and effectiveness is several years.

Potential risks and benefits

Participating in this study might offer the potential benefit of reducing or eliminating the need for regular blood transfusions, which could significantly improve a child's quality of life. However, like all medical treatments, especially new ones, CTX001 carries potential risks. These can include side effects from the conditioning treatment, reactions to the cell infusion, and other potential complications that doctors will discuss in detail. Your child's health will be closely monitored throughout the study. Remember, joining a study is always voluntary, and you have the right to withdraw your child from the study at any time, for any reason, without affecting their future medical care.

Locations (6)

TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, United States
Hospital for Sick Children - Hematology
Toronto, Canada
University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
Great Ormond Street Hospital for Children
London, United Kingdom
St.Mary's Hospital - Children's Clinical Research Facility
London, United Kingdom

Common questions

What is beta-thalassemia?

It's an inherited blood disorder where the body can't make enough healthy red blood cells, leading to anaemia and often requiring regular blood transfusions.

What is CTX001?

It's a new gene-editing treatment that aims to correct the genetic problem in a child's own blood stem cells so they can produce healthy red blood cells.

Is this a cure?

This study is investigating if CTX001 can reduce or eliminate the need for blood transfusions. While promising, it's an experimental treatment, and studies like this are crucial to understand its full impact.

How long will my child be followed after treatment?

Children in the study will be monitored carefully for several years after receiving the treatment to track its long-term safety and effectiveness.

Will my child still need blood transfusions after treatment?

The goal of CTX001 is to help children produce their own healthy blood cells and potentially reduce or stop the need for transfusions, but this is what the study aims to find out.