A Safety, Tolerability, and Efficacy Study of VX-264 in Subjects With Type 1 Diabetes
Research type
Research Study
Full title
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
IRAS ID
1006715
Contact name
Elizabeth Mellen
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2022-003318-35
Clinicaltrials.gov Identifier
Research summary
Type 1 diabetes (T1D) is a chronic condition that affects nearly 1.7 million people in the US and more than 1 million people in Europe. In T1D, the body’s immune system destroys insulin-producing cells called beta cells which help the body manage and use blood sugar correctly. This leads to hyperglycemia. Patients with T1D are dependent on insulin given via injection or pump to manage hyperglycemia, prevent life-threatening ketoacidosis, and delay other diabetes complications. The current standard treatments available do not always achieve and maintain blood sugar control in patients with T1D. Vertex Pharmaceuticals, Inc. is currently developing VX-264 as a new potential treatment for patients with T1D. VX-264 is comprised of cells to control blood sugars and a device that houses those cells. The VX-264 cells are a mixture of cells made from human embryonic stem cells that can make insulin and other hormones like glucagon that are usually made in the pancreatic islets. The device protects the cells from the body’s immune system but is designed to let oxygen and nutrients in to fuel the VX-264 cells, as well as allowing them to a move glucose in and insulin out. VX-264 is intended to be implanted via a surgical procedure in the abdominal wall of participants with T1D, and stay in place indefinitely. The study objective is to determine whether the Study Product can stay in place safely and tolerably, and whether it can improve blood sugar control. This is a 3-part (Part A, Part B, and Part C), open-label, multicentre study in adult participants with T1D. Which Part the participant may enroll in depends on the enrollment status of the study at the time of their screening. Participants will be in the study for approximately two years. All participants will be requested to enroll in a separate 5-year long term follow up (LTFU) study.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0163
Date of REC Opinion
15 Mar 2023
REC opinion
Further Information Favourable Opinion