First-in-human Safety Study of Hypoimmune Pancreatic Islet Transplantation in Adult Subjects With Type 1 Diabetes
This study tests whether insulin-producing cells from a donor pancreas can be safely transplanted into people with type 1 diabetes. These cells have been genetically modified to reduce the risk of rejection, so patients may not need strong immunosuppressive drugs. The goal is to help the body produce insulin again without constant insulin injections.
Key Objective: The trial is testing whether genetically modified pancreatic islet transplants can restore the body's ability to produce insulin and control blood sugar in people with type 1 diabetes.
Who to Consider: Adults with type 1 diabetes who are interested in exploring a potential cell transplant therapy should consider enrolling, particularly those willing to participate in a first-in-human safety study.
Trial Parameters
Brief Summary
The current study tests the hypothesis whether genetically modified Langerhans islet cells containing insulin-producing cells from a deceased organ donor can 1. be transplanted safely and 2. help to regain insulin production in individuals with type 1 diabetes without need in simultaneous treatment with immunosuppressive medicines. The study is an open, one-armed study where adult subjects with longstanding type 1 diabetes will receive transplantation of Langerhans islet cells (25 000 000-80 000 000) into forearm muscle. Both subjects receive active treatment. Safety is monitored with frquent follow-up visits over a year, including medical examinations, blood tests and MRI scans. Insluin producing cell function is monitored with blood samples and continuous glucose measurement. Main objective is to to investigate the safety of an intramuscular transplantation of genetically modified allogeneic human islets (study product UP421) in adult subjects diagnosed with type 1 diabetes. Secondary objectives are to study changes in beta-cell function, metabolic control and immunological response to pancreatic islets during the first year following treatment.
Eligibility Criteria
Inclusion Criteria: 1. Signed informed consent for participation in the study 2. Diagnosis of type 1 diabetes mellitus (T1D); i) for ≥ 5 years and ii) diagnosed before the age of 18 years and iii) at least one or more HbA1c documented in the subject's medical journal or Swedish National Diabetes Registry during the last five-year period must be ≥70 mmol/mol. 3. The subject must be involved in intensive diabetes management defined as self-monitoring of subcutaneous glucose level by continuous glucose monitoring or by intermittent scanning glucose monitoring no less than a mean of three times per day averaged over each week and by the administration of three or more insulin injections per day or insulin pump therapy. This management must be under the direction of an M.D specialized in endocrinology and diabetology with support of a diabetes nurse at a specialist clinic for Endocrinology and Diabetology or Internal Medicine during the 12 months prior to study enrolment. 4. C-peptide negat