Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humansInterventional

A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

This research is looking at a new treatment called CTX001 for people with severe sickle cell disease. This treatment involves using your own blood cells, specially modified, and given back to you. The main goal is to find out if this treatment is safe and effective. Researchers will monitor several things, including how well your body accepts the new cells and if you have fewer painful sickle cell crises. They will also look generally at your health and any side effects. This is a combined early-stage study, meaning it's one of the first times this treatment is being directly tested in people to see how it works and if it could be a benefit for those with this condition.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Vertex Pharmaceuticals Inc.

Enrolment target

Start

28 Aug 2024

Severe sickle cell disease (SCD)

What is this study about?

This is a clinical trial investigating an experimental treatment called CTX001 for people living with severe sickle cell disease. Sickle cell disease is a lifelong condition that can cause severe pain, organ damage, and other serious health problems. The aim of this study is to see if this new treatment can make a real difference to people's lives.

The CTX001 treatment involves taking some of your own blood stem cells, which are special cells that can develop into different types of blood cells. These cells are then changed in a laboratory using a technology called CRISPR-Cas9 before being given back to you, often after you've had some other treatments to prepare your body. The hope is that these modified cells will help your body produce healthy red blood cells, reducing the symptoms of sickle cell disease.

Throughout the study, the research team will keep a close eye on your health. They will be measuring several important things, like how well your body accepts the new cells, if you have fewer painful sickle cell crises (also known as 'VOCs'), and how often you need to go into hospital. They will also carefully watch for any side effects to make sure the treatment is safe.

Key takeaways

This study is testing a new gene therapy (CTX001) for severe sickle cell disease.
It uses your own modified stem cells to try and improve your condition.
The main goals are to check the treatment's safety and how effectively it reduces painful crises and hospital stays.
Participation involves preparing your body, receiving the modified cells, and long-term health monitoring.
This is an early and combined stage of research to understand the treatment's potential.
You must be at least 18 years old to be considered for this study.

Who may be eligible?

To be considered for this study, you must be at least 18 years old. There isn't an upper age limit mentioned, so older adults may also be able to take part.

Since this is a study for severe sickle cell disease, potential participants will need to have a confirmed diagnosis of severe sickle cell disease to be eligible. The clinical team will check your medical history to make sure the study is suitable for you.

Generally, participants should be in good enough health to undergo the treatment procedures and follow-up care required by the study. The study team will review all your health information to ensure your safety and that the treatment is appropriate for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of severe sickle cell disease?
Are you generally in good health, aside from your sickle cell disease?
Are you willing and able to attend regular hospital appointments for monitoring?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve several steps. Initially, you would undergo a thorough health check-up to ensure you are suitable. If eligible, you would likely receive some medication like PLERIXAFOR and BUSULFAN to prepare your body for the treatment. Your own stem cells would be collected, modified to create CTX001 (which is given as an infusion of 4-13 million cells per ml), and then returned to you.

After receiving CTX001, you would have regular hospital visits for medical checks, blood tests, and to monitor any side effects or improvements in your condition. These visits would be frequent at first and then might become less often over time. The team will be watching closely for how well your body's cells are working and if your sickle cell symptoms improve. The total duration of your participation, including follow-up, could be quite long, potentially several years, to fully understand the long-term effects of the treatment.

Potential risks and benefits

Participating in a clinical trial like this could offer potential benefits, such as access to a new and experimental treatment that might help improve your severe sickle cell disease symptoms or even offer a long-term solution where current treatments haven't been fully effective. However, it's also important to understand there are potential risks involved. These might include side effects from the medications used, risks associated with the cell collection and infusion procedures, and uncertainty about how your body will react to the modified cells. There's also a possibility the treatment may not work for you, or could even lead to unexpected health problems. You will have full rights to withdraw from the study at any time without giving a reason, and this decision will not affect your usual medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Belgium
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Unverified
Italy

Common questions

What is CTX001?

CTX001 is a new, experimental gene therapy designed to treat severe sickle cell disease by modifying your own blood stem cells.

What does a 'Phase 1/2/3 Study' mean?

This means the study is combining early stages (Phase 1 and 2) to see if the treatment is safe and how well it works, and also later stages (Phase 3) to confirm its effectiveness and compare it to existing treatments.

What are 'painful crises' and how does the study track them?

Painful crises, or VOCs, are severe pain episodes in sickle cell disease. The study tracks how often they happen and if people remain free from them.

Will I need to stay in the hospital?

It's likely you would need hospital stays for the cell collection and infusion parts of the treatment, and for close monitoring afterwards.

What is 'neutrophil engraftment'?

Neutrophil engraftment means that the new, modified blood cells have successfully settled into your bone marrow and started making important infection-fighting white blood cells (neutrophils).