Active not recruitingPHASE3INTERVENTIONAL

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

This research study is looking at a new treatment called CTX001 for children between 2 and 11 years old who have severe sickle cell disease. This treatment is being tested for children whose current medicine, hydroxyurea, either hasn't worked well enough or caused difficult side effects. CTX001 is a type of gene therapy where a child's own cells are collected, modified in a lab, and then given back to them. The main goals of this study are to find out if CTX001 is safe for children and if it can help improve their sickle cell disease symptoms. Researchers want to see if this one-time treatment can make a lasting difference.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Vertex Pharmaceuticals Incorporated

Enrolment target

Start

02 May 2022

Estimated completion

31 May 2026

Sickle Cell Disease Hydroxyurea Failure Hydroxyurea Intolerance Hemoglobinopathies Hematological Diseases

What is this study about?

Sickle cell disease is a serious condition that affects red blood cells. These cells, which are usually round and flexible, become stiff and C-shaped, like a farm tool called a sickle. This can cause blockages in blood vessels, leading to pain, damage to organs, and other health problems. Many children with severe sickle cell disease are treated with a medicine called hydroxyurea, but for some, it doesn't work well enough or causes side effects that make it hard to take.

This study is exploring a new approach called gene therapy, specifically using a treatment named CTX001. Gene therapy aims to correct the underlying problem that causes sickle cell disease. In this trial, doctors will collect some of the child's own blood-forming cells. These cells are then sent to a lab where they are modified using a special technique called CRISPR-Cas9 to help them produce healthy red blood cells.

After modification, these changed cells (CTX001) are given back to the child through an infusion, similar to a blood transfusion. The hope is that these new cells will take root and start making healthy red blood cells, reducing the symptoms and complications of sickle cell disease. This is a "Phase 3" study, which means it's one of the final steps in testing a treatment before it can be considered for wider use, and it will involve a single dose of the treatment.

Key takeaways

This study is testing a new gene therapy (CTX001) for severe sickle cell disease.
It's for children aged 2-11 whose current treatment (hydroxyurea) isn't working well.
The treatment uses the child's own cells, modified in a lab, then given back to them.
The main goals are to check how safe and effective CTX001 is.
There will be careful medical monitoring for many years after the single treatment.

Who may be eligible?

This study is looking for children between 2 and 11 years old who have been diagnosed with severe sickle cell disease. To be considered severe, they must have experienced at least two serious pain crises or other severe events related to their sickle cell disease each year for the past two years. Importantly, they must also be in a situation where their current medicine, hydroxyurea, isn't working well enough to control their symptoms, or they can't take it due to side effects. Their doctor will also need to confirm that they are suitable for a specific type of cell transplant.

There are also some reasons why a child might not be able to join the study. For example, if they have a healthy family member who is a perfect match for a different type of stem cell transplant, or if they have already had a stem cell transplant before, they wouldn't be eligible. Also, if a child has a significant infection at the time of screening, they wouldn't be able to participate. The study team will carefully review all medical information to ensure it's safe and appropriate for a child to join.

Quick self-check

Is your child between 2 and 11 years old?
Does your child have severe sickle cell disease?
Has hydroxyurea not worked well for your child, or can they not tolerate it?
Has your child had at least two serious sickle cell crises in each of the last two years?
Has your child NOT had a stem cell transplant before?
Does your child NOT have a perfectly matched family donor for a stem cell transplant?

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

What does participation involve?

Taking part in this study would involve several steps over a period of time. First, there will be screening visits to confirm your child meets all the requirements. If eligible, your child's blood-forming stem cells will be collected. This is a procedure that may involve hospitalisation. These cells will then be sent away to be modified to create the CTX001 treatment. Before returning these modified cells, your child will receive a strong medicine called chemotherapy to prepare their body. Then, they will receive the CTX001 infusion, which is a one-time treatment. After this, there will be careful monitoring in the hospital, followed by regular check-ups and assessments at the clinic, which will continue for many years to track their progress and safety. The full duration of follow-up for this therapy is typically 15 years.

Potential risks and benefits

The potential benefit of this study is that CTX001 could significantly reduce the severe symptoms and complications of sickle cell disease, potentially offering a long-term solution. However, like all new treatments, there are potential risks, including side effects from the chemotherapy given before the treatment, risks associated with the cell collection procedure, and potential unknown side effects from the gene therapy itself. You will receive detailed information about all possible risks and benefits before deciding. Remember, joining a study is always voluntary, and you are free to withdraw your child at any time without affecting their future medical care.

Locations (7)

Levine Children's Hospital - Hematology
Charlotte, United States
The Children's Hospital of Philadelphia - Hematology
Philadelphia, United States
St. Jude Children's Research Hospital
Memphis, United States
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, United States
University Hospital Duesseldorf - 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
St.Mary's Hospital - Haematology Dept
London, United Kingdom

Common questions

What is gene therapy?

Gene therapy is a new type of treatment that tries to fix the root cause of a disease by changing or replacing faulty genes in your body's cells.

What is 'autologous' CTX001?

'Autologous' means the treatment is made using your child's own cells, so there's no risk of their body rejecting cells from another person.

What does 'severe SCD' mean in this study?

In this study, 'severe SCD' means your child has had at least two serious pain crises or other major sickle cell related events each year for the past two years.

What is 'hydroxyurea failure'?

This means that the medicine hydroxyurea hasn't been effective enough in controlling your child's sickle cell disease, or they couldn't take it due to side effects.

How long will my child be followed after treatment?

Children who receive this treatment will be closely monitored for safety and effectiveness for a long time, typically up to 15 years.