Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-880 in T1D Subjects

  • Research type

    Research Study

  • Full title

    A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-880 in Subjects Who Have Type 1 Diabetes Mellitus With Impaired Hypoglycemic Awareness and Severe Hypoglycemia

  • IRAS ID

    1006463

  • Contact name

    Kari Forsaith

  • Contact email

    Kari_Forsaith@vrtx.com

  • Sponsor organisation

    Vertex Pharmaceuticals Incorporated

  • Eudract number

    2022-002292-11

  • Clinicaltrials.gov Identifier

    NCT04786262

  • Research summary

    Type 1 diabetes (T1D) is a chronic condition that affects nearly 1.6 million people in the US and more than an estimated 900,000 people in Europe. T1D results from the destruction of insulin-producing pancreatic β-cells by an autoimmune process leading to insulin deficiency and hyperglycemia. Patients with T1D are dependent on insulin replacement therapy to manage hyperglycemia, prevent life-threatening ketoacidosis, and delay other severe diabetes complications. However, despite best treatment and clinical management, many patients with T1D suffer from long-term vascular complications, including diabetic retinopathy and diabetic nephropathy with a reduced life expectancy of more than 10 years.
    Vertex Pharmaceuticals, Inc is currently developing VX-880 as a new potential treatment for subjects who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 is composed of allogeneic human stem cell derived islets. VX-880 is intended to be transplanted into subjects with T1D via infusion into the portal vein in the context of a steroid-free immunosuppressive regimen.
    Subjects will be followed in Study VX20-880-101 for 5 years after VX-880 infusion.

  • REC name

    London - West London & GTAC Research Ethics Committee

  • REC reference

    22/LO/0839

  • Date of REC Opinion

    25 Jan 2023

  • REC opinion

    Further Information Favourable Opinion