Actively Recruiting
First-in-human Safety Study of Hypoimmune Pancreatic Islet Transplantation in Adult Subjects With Type 1 Diabetes
Led by Per-Ola Carlsson · Updated on 2024-12-11
2
Participants Needed
1
Research Sites
64 weeks
Total Duration
On this page
Sponsors
P
Per-Ola Carlsson
Lead Sponsor
S
Sana Biotechnology
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
First-in-human Safety Study of Hypoimmune Pancreatic Islet Transplantation in Adult Subjects With Type 1 Diabetes
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent for participation in the study
- Diagnosis of type 1 diabetes mellitus for at least 5 years
- Diabetes diagnosed before age 18
- At least one HbA1c measurement 50 mmol/mol during the last five years
- Intensive diabetes management with frequent glucose monitoring and multiple daily insulin injections or pump therapy under specialist care for 12 months
- C-peptide negative response to mixed meal tolerance test (less than 0.01 nmol/l)
- Positive for antibodies to either GAD or IA2 at screening
- Age between 30 and 45 years at enrollment
- HbA1c 50 mmol/mol
- Exogenous insulin needs less than 1 IU/kg
- Body weight less than 80 kg
- Female participants of childbearing potential must use adequate contraception until one year after UP421 administration
- Male participants must use effective contraception during the trial period
You will not qualify if you...
- Previous organ transplantation
- Use of systemic immunosuppressive medication for other diseases
- History of malignancy
- Use of investigational agents within 4 weeks before enrollment
- Use of anti-diabetic medication other than insulin within 4 weeks before enrollment
- Active infections including Tuberculosis, HIV, HBV, or HCV
- Liver function test values exceeding normal limits
- Infection with HTLVI or HTLVII
- Pregnancy, nursing, or intention to become pregnant
- Chronic kidney disease grade 3 or worse (GFR <60 ml/min)
- History or symptoms of cardiac disease
- HLA or MIC A/B immunization
- Known autoimmune diseases other than type 1 diabetes
- Live attenuated vaccines within 6 months before transplant
- High islet antibody levels or positive ZnT8 auto-antibodies
- Untreated proliferative diabetic retinopathy
- Major psychiatric illness increasing risk of noncompliance or unsafe participation
- Ongoing substance abuse or recent noncompliance
- Known hypersensitivity to ciprofloxacin, gentamicin, or amphotericin
- Any other condition judged by the Principal Investigator as unsafe for participation
AI-Screening
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Trial Site Locations
Total: 1 location
1
Uppsala University Hospital
Uppsala, Sweden, 75185
Actively Recruiting
Research Team
P
Per-Ola Carlsson, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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