Join a Type 1 Diabetes Trial Testing Insulin-Producing Cells

This study is investigating whether Vertex’s VX-264, an encapsulated stem cell therapy, can safely restore insulin-producing islet cell function to people with type 1 diabetes without the need for lifelong immunosuppressive medication.

Clinical Trials Identifier: NCT05791201

Trial Name: A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes

Diabetes Type: Adults with type 1 diabetes

Trial Sponsor: Vertex Pharmaceuticals

What is the trial researching?

This study is investigating whether VX-264, an encapsulated human stem cell therapy developed by Vertex, can safely restore insulin production in people with type 1 diabetes – without requiring lifelong immunosuppressive medication. 

Vertex has already made headlines for research outcomes from VX-880, another diabetes therapy that replaced damaged insulin-producing beta cells with healthy ones created from stem cells. In clinical trials, VX-880 has successfully restored insulin production in several patients with type 1 diabetes. Those treated with VX-880, however, must take immunosuppressants to prevent the immune system from rejecting new beta cells. 

Vertex’s VX-264 therapy keeps beta cells in an enclosed device, physically protecting them from the body's immune cells while still allowing nutrients to reach the beta cells, a strategy known as encapsulation. There is hope that, when encapsulated, the insulin-producing stem cell therapy will be safer, more convenient, and eliminate the lifelong need for immunosuppressive medication.

This phase 1 and phase 2 study, which is currently enrolling and will run through 2026, plans to enroll 17 participants. Researchers will evaluate the safety of VX-264 in participants over a period of two years. They will also measure changes in average total daily insulin dose and A1C, and observe if participants are able to reach insulin independence.

Why is this important?

Vertex has previously shown through a combination of VX-880 therapy and immunosuppressants that insulin production can be restored in people with type 1 diabetes. VX-264 is novel in that encapsulating cells in a specially designed device would eliminate the need for long-term immunosuppressive medication. 

If recruited for this study, you will receive Vertex’s stem cell therapy free of charge. It’s possible that this therapy may restore insulin production, possibly reducing insulin dosage needs and improving A1C, potentially to the point of insulin independence. 

Participating in such clinical research may also help increase your knowledge about diabetes and take a more active role in managing your health. By doing so, you are also helping researchers better understand diabetes and advance future treatments and potential cures, thus improving the lives of others with the condition.

Are you interested?

You may be eligible to participate if you:

• Are between the ages of 18-65 years old

• Were diagnosed with type 1 diabetes at least five years ago

• Have been using a continuous glucose monitor (CGM) for at least four weeks

• Have not had prior cell therapy or organ transplants

The trial is currently recruiting in Miami, Chicago, Boston, and Pittsburgh in the U.S., Edmonton and Toronto in Canada, and Leiden in the Netherlands. See more details on inclusion and exclusion criteria here.

To learn more about this study, contact Vertex’s medical information team at medicalinfo@vrtx.com or call 617-341-6777.

Photo credit: YouTube/Vertex Pharmaceuticals

About the authors

Susannah Chen

Prior to joining The diaTribe Foundation as a managing editor, Susannah Chen was a freelance writer and editorial content strategist focused on food, drink, and travel. A health journey involving... Read the full bio »