Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in subjects with Transfusion-Dependent β-Thalassemia

This research trial is investigating a new gene therapy, called Casgevy, for individuals living with transfusion-dependent beta-thalassemia. This condition means people need frequent blood transfusions to stay healthy. The trial aims to find out how safe and effective Casgevy is. Specifically, researchers want to see if this treatment can help the body produce healthier red blood cells, potentially reducing or even stopping the need for ongoing blood transfusions. To do this, they will carefully monitor participants for safety, check how well their blood cells recover after treatment, and measure their haemoglobin levels to see if they can live without transfusions for long periods. This study is in its final stages, meaning it builds on earlier research that showed promising results.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Vertex Pharmaceuticals Inc.

Enrolment target

Start

13 Sep 2024

Transfusion-dependent β-thalassemia (TDT)

What is this study about?

This research trial is looking into a new treatment called Casgevy for a serious blood condition called transfusion-dependent beta-thalassemia. People with this condition have a problem with their red blood cells and need regular blood transfusions to live and stay well. Without these transfusions, they become very anaemic, meaning they don't have enough healthy red blood cells to carry oxygen around their body.

Casgevy is a type of gene therapy. It works by taking some of a patient's own blood stem cells, which are special cells that can develop into any type of blood cell. These cells are then changed in a laboratory using a clever gene-editing tool called CRISPR-Cas9. The goal is to correct the problem that causes beta-thalassemia, so the patient's body can start making healthy red blood cells on its own. After the cells are modified, they are given back to the patient through an infusion.

The main goal of this study is to see if Casgevy is safe and if it can help people with beta-thalassemia reduce or even stop their need for blood transfusions. Researchers will be carefully checking many things, including how well the new cells settle in the body, any side effects, and if patients can maintain healthy blood levels for long periods without needing transfusions. This is a crucial step in understanding if Casgevy could be a valuable new treatment option for those living with this condition.

Key takeaways

This study evaluates a new gene therapy, Casgevy, for severe beta-thalassemia.
The goal is to reduce or eliminate the need for blood transfusions.
It uses your own modified stem cells.
The study focuses on safety and how well the treatment works.
Participants will be closely monitored for several years.

Who may be eligible?

This study is open to adults aged 18 years and older. Both men and women can take part.

To be considered for this trial, you would need to have a diagnosis of transfusion-dependent beta-thalassemia. This means you currently receive regular blood transfusions for your condition.

There will be other detailed medical checks to make sure this treatment is suitable for you and that you meet all the health requirements to safely participate in the study. Your doctor will be able to discuss these with you further.

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 transfusion-dependent beta-thalassemia?
Do you currently receive regular blood transfusions?
Are you able to commit to long-term follow-up visits?
Are you willing to undergo various medical tests and procedures?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, the process involves several steps. First, some of your own blood stem cells would be collected. These cells would then be sent to a lab to be modified with the Casgevy treatment. Before you receive your modified cells, you will get some chemotherapy (busulfan) to prepare your body for the new cells. You will also receive other medications (filgrastim, plerixafor) to help your body produce and recover healthy blood cells after the treatment.

After receiving the Casgevy infusion, you would need regular check-ups and monitoring by the study team. These visits will involve blood tests to check your blood counts, haemoglobin levels, and how well the new cells are working. They will also look for any side effects. The study aims to follow participants for a long time to understand the lasting effects of the treatment, including how long you can go without transfusions. The overall duration of your involvement in the study could be several years, with more frequent visits initially and then fewer over time.

Potential risks and benefits

Participating in a clinical trial has potential benefits and potential risks. A potential benefit of this study is that Casgevy might reduce or even eliminate your need for regular blood transfusions, which could significantly improve your quality of life. However, as with any new treatment, there are potential risks, including side effects from the chemotherapy given before the gene therapy, or reactions to the Casgevy infusion itself. The modified cells could also have unexpected effects. The research team will explain all known risks to you in detail. Remember, you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

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

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Unverified
Germany

Common questions

What is beta-thalassemia?

Beta-thalassemia is a blood condition where your body doesn't produce enough healthy red blood cells. This leads to anaemia and means you often need regular blood transfusions.

What is gene therapy?

Gene therapy is a new type of treatment that aims to fix or replace faulty genes in your body to treat a disease. In this study, it involves modifying your own cells outside the body.

What is Casgevy?

Casgevy is the name of the specific gene therapy being tested. It uses a tool called CRISPR-Cas9 to make changes to your stem cells so they can produce healthy red blood cells.

Will I still need blood transfusions after this treatment?

The main goal of the treatment is to reduce or stop your need for blood transfusions. The study will carefully monitor if you can maintain healthy blood levels without them for a long time.

What are the main things researchers are checking in this study?

Researchers are mainly checking if the treatment is safe, if your blood cells recover well, and if you can go for long periods without needing blood transfusions.