Making stem cells to create insulin-producing pancreatic beta cells

Production Of Induced Pluripotent Stem Cells (iPSCs) For The Generation Of Insulin-Producing β Cells

This project will try to turn a small skin, blood, or urine sample from people with diabetes and healthy volunteers into stem cells that can be made into insulin-producing pancreatic beta cells.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 12 Years to 70 Years
• Sex: All
• Sponsor: Ospedale San Raffaele (other)
• Locations: 1 site (Milan)
• Trial ID: NCT07479134 on ClinicalTrials.gov

What this trial studies

Participants provide a one-time biological sample (3 mm skin punch biopsy, blood draw, or urine) collected at Ospedale San Raffaele in Milan. Somatic cells from those samples will be reprogrammed into induced pluripotent stem cells (iPSCs) and then differentiated in the lab into insulin-producing β cells. Researchers will compare iPSC-derived β cells from patients with metabolic or genetic pancreatic disorders (including type 1 and type 2 diabetes, MODY, Wolfram syndrome, and pancreatogenic diabetes) to cells from healthy donors to identify disease-specific cellular and molecular features. The primary aim is to establish a reliable, reproducible protocol for producing functional β cells to support disease modeling and future cell-based therapies.

Who should consider this trial

Good fit: Ideal participants are people aged 12–70 with pancreatic β cell disorders (such as type 1 or type 2 diabetes, MODY, Wolfram syndrome, or pancreatogenic diabetes) or healthy volunteers willing to provide a one-time skin, blood, or urine sample and informed consent.

Not a fit: People with active cancer, known HIV/HBV/HCV infection, severely unstable blood glucose at sampling, or on medications that interfere with iPSC generation are excluded and are unlikely to benefit from participating.

Why it matters

Potential benefit: If successful, this work could provide patient-specific beta cells for lab research and help lay the groundwork for future cell-replacement therapies for diabetes.

How similar studies have performed: Similar laboratory programs have successfully generated iPSC-derived insulin-producing cells for disease modeling, but translating these results into safe, effective cell therapies for patients remains experimental.

Eligibility criteria

Inclusion Criteria:

Age between 12 and 70 years.

Ability and willingness to provide informed consent (or assent with parental consent for minors).

Group 1: Individuals diagnosed with pancreatic β cell dysfunction, including but not limited to:

Type 1 Diabetes

Type 2 Diabetes

Maturity Onset Diabetes of the Young (MODY)

Wolfram Syndrome

Pancreatogenic Diabetes

Group 2: Healthy control donors without pancreatic β cell dysfunction.

Pregnant or breastfeeding women may be included if they meet the inclusion criteria.

Exclusion Criteria:

Age below 12 or above 70 years.

Health condition too compromised to allow safe tissue collection (e.g., acute hypoglycemia \<70 mg/dL or hyperglycemia \>140 mg/dL at sampling).

Inability or unwillingness to provide informed consent/assent.

Active malignancy or current cancer treatment.

Known infection with HIV, Hepatitis B, or Hepatitis C.

Use of medications that may interfere with iPSC generation (e.g., high-dose corticosteroids, immunomodulators), unless approved by investigators.

For participants undergoing skin biopsy: bleeding disorders, local skin infection, allergy to anesthetics, or use of anticoagulants.

Where this trial is running

Milan

• Diabetes Research Institute — Milan, Italy (RECRUITING)

Study contacts

• Study coordinator: Valeria Sordi, PhD

Conditions: Diabetes, diabetes, stem cells, induced pluripotent stem cells