Ignite Creation Date:
2026-03-26 @ 3:19 PM
Ignite Modification Date:
2026-03-31 @ 12:42 PM
Study NCT ID:
NCT07479134
Status:
RECRUITING
Last Update Posted:
2026-03-18
First Post:
2026-03-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Production of Stem Cells for the Generation of Pancreatic Cells
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003920', 'term': 'Diabetes Mellitus'}], 'ancestors': [{'id': 'D044882', 'term': 'Glucose Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D001800', 'term': 'Blood Specimen Collection'}, {'id': 'D059349', 'term': 'Urine Specimen Collection'}], 'ancestors': [{'id': 'D013048', 'term': 'Specimen Handling'}, {'id': 'D019411', 'term': 'Clinical Laboratory Techniques'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}, {'id': 'D011677', 'term': 'Punctures'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'BASIC_SCIENCE', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2026-03-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2036-02-28', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-03-13', 'studyFirstSubmitDate': '2026-03-13', 'studyFirstSubmitQcDate': '2026-03-13', 'lastUpdatePostDateStruct': {'date': '2026-03-18', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-03-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2035-02-28', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'generation of iPSC', 'timeFrame': 'within 7-60 days after culture establishment', 'description': 'Successful generation of iPSCs from somatic cells, defined by \\>80% expression of pluripotency markers (SSEA4, OCT4, NANOG) within 7-60 days after culture establishment.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['diabetes; stem cells, induced pluripotent stem cells'], 'conditions': ['Diabetes']}, 'descriptionModule': {'briefSummary': 'The goal of this interventional study is to generate induced pluripotent stem cells (iPSCs) from somatic cells and differentiate them into insulin-producing β cells in patients with metabolic and genetic pancreatic diseases and in healthy controls. The main questions it aims to answer are:\n\nCan somatic cells from healthy individuals and patients with diabetes be successfully reprogrammed into iPSCs?\n\nCan these iPSCs be differentiated into functional insulin-producing β cells suitable for studying disease mechanisms and developing cell-based therapies?\n\nParticipants will provide a single biological sample (either a 3 mm skin punch biopsy, a blood sample, or a urine sample) collected under sterile conditions. The samples will be used to derive somatic cells, which will then be reprogrammed into iPSCs and differentiated into β cells for laboratory analyses.\n\nParticipants will:\n\nUndergo a one-time sample collection (skin biopsy, blood draw, or urine collection) at Ospedale San Raffaele\n\nReceive standard post-procedure care (if applicable)\n\nThis research aims to improve understanding of β cell function and dysfunction in diabetes and to advance personalized regenerative therapies for β cell replacement.', 'detailedDescription': "The goal of this interventional study is to investigate how induced pluripotent stem cells (iPSCs) can be efficiently generated from somatic cells and subsequently differentiated into insulin-producing β cells, with the broader aim of improving the understanding of pancreatic β cell biology and supporting the development of future cell-based therapies for diabetes. The study involves the collection of biological samples from both healthy donors and patients affected by metabolic or genetic disorders that impact pancreatic β cell function, such as type 1 diabetes, type 2 diabetes, Maturity Onset Diabetes of the Young (MODY), Wolfram syndrome, and pancreatogenic diabetes.\n\nThe primary objective is to establish a reliable and reproducible protocol for the production of iPSCs from somatic cells obtained from different donor groups and to evaluate their potential to differentiate into functional β cells. By comparing iPSC-derived β cells from patients with those derived from healthy controls, the study aims to identify disease-specific cellular and molecular characteristics that may contribute to β cell dysfunction and diabetes pathogenesis. These findings will serve as a foundation for improving strategies for β cell replacement and regenerative medicine.\n\nParticipants enrolled in the study will provide a single biological sample, collected at Ospedale San Raffaele in Milan. Depending on the laboratory's technical requirements and the participant's clinical condition, the sample may consist of a 3 mm skin punch biopsy, a small volume of peripheral blood (up to 20 mL), or a urine sample (up to 300 mL). These procedures are all standard clinical techniques performed under sterile conditions by trained healthcare personnel. The risks associated with the procedures are minimal and limited to mild pain, bruising, or, in the case of skin biopsy, a small scar. Participants will receive post-procedure care instructions and will not be required to attend follow-up visits after the sample collection.\n\nOnce collected, the somatic cells (such as fibroblasts, peripheral blood mononuclear cells, or urine-derived epithelial cells) will be isolated, expanded, and cryopreserved. These cells will then be reprogrammed into iPSCs using a non-integrating RNA-based reprogramming system (StemRNA™ 3rd Gen Reprogramming Kit, Reprocell). The success of the reprogramming process will be assessed by measuring cell viability and the expression of specific pluripotency markers, including SSEA4, OCT4, and NANOG, through flow cytometry. Only iPSC lines that meet predefined quality criteria-viability above 60% and pluripotency marker expression above 80%-will be considered successful and preserved for further use.\n\nSubsequently, the established iPSC lines will be differentiated into insulin-producing β cells through a stepwise process that mimics pancreatic development. The resulting β-like cells will be analyzed for their ability to produce and secrete insulin in response to glucose stimulation, as well as for other molecular and functional properties. The comparison between β cells derived from healthy donors and those from patients with different forms of diabetes will help elucidate the mechanisms underlying β cell dysfunction and loss in these diseases.\n\nThe study will include up to 100 participants, both male and female, aged between 12 and 70 years. At least 30% of participants will be healthy controls, while the remaining will represent patients with various types of pancreatic dysfunction. This approach allows the generation of a diverse biobank of patient-specific iPSC lines, which can be used not only for this study but also for future research projects approved by the Ethics Committee. The total duration of the study is estimated at 10 years, reflecting the long-term nature of iPSC generation, differentiation, and characterization.\n\nAlthough there are no direct medical benefits for participants, the potential societal benefits of this study are significant. The generation of patient-specific iPSCs provides a powerful platform for studying the pathophysiology of diabetes in vitro, enabling the identification of disease mechanisms and testing of new therapeutic strategies in a personalized way. These cells can serve as models to evaluate how genetic background, environmental factors, and disease states affect β cell development and function. In the long term, this research could contribute to the development of advanced regenerative and transplantation-based treatments for diabetes, potentially reducing or eliminating the need for exogenous insulin therapy.\n\nIn summary, this study represents an important step toward bridging fundamental stem cell biology with translational applications in diabetes research. By combining patient-specific iPSC technology with cutting-edge differentiation protocols, it aims to create a resource that not only deepens scientific understanding but also supports the development of innovative and personalized therapies for individuals living with diabetes and related metabolic diseases."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '12 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\nAge between 12 and 70 years.\n\nAbility and willingness to provide informed consent (or assent with parental consent for minors).\n\nGroup 1: Individuals diagnosed with pancreatic β cell dysfunction, including but not limited to:\n\nType 1 Diabetes\n\nType 2 Diabetes\n\nMaturity Onset Diabetes of the Young (MODY)\n\nWolfram Syndrome\n\nPancreatogenic Diabetes\n\nGroup 2: Healthy control donors without pancreatic β cell dysfunction.\n\nPregnant or breastfeeding women may be included if they meet the inclusion criteria.\n\nExclusion Criteria:\n\nAge below 12 or above 70 years.\n\nHealth condition too compromised to allow safe tissue collection (e.g., acute hypoglycemia \\<70 mg/dL or hyperglycemia \\>140 mg/dL at sampling).\n\nInability or unwillingness to provide informed consent/assent.\n\nActive malignancy or current cancer treatment.\n\nKnown infection with HIV, Hepatitis B, or Hepatitis C.\n\nUse of medications that may interfere with iPSC generation (e.g., high-dose corticosteroids, immunomodulators), unless approved by investigators.\n\nFor participants undergoing skin biopsy: bleeding disorders, local skin infection, allergy to anesthetics, or use of anticoagulants.'}, 'identificationModule': {'nctId': 'NCT07479134', 'briefTitle': 'Production of Stem Cells for the Generation of Pancreatic Cells', 'organization': {'class': 'OTHER', 'fullName': 'Ospedale San Raffaele'}, 'officialTitle': 'Production Of Induced Pluripotent Stem Cells (iPSCs) For The Generation Of Insulin-Producing β Cells', 'orgStudyIdInfo': {'id': 'iBETA'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Biospecimen Collection for iPSC Generation and β Cell Differentiation', 'description': 'All participants will provide a single biological sample (skin biopsy, blood draw, or urine collection) for isolation of somatic cells. These cells will be reprogrammed into induced pluripotent stem cells (iPSCs) and differentiated into insulin-producing β cells to study pancreatic function and disease mechanisms. No therapeutic intervention is administered.', 'interventionNames': ['Procedure: Biological - skin biopsy, blood draw, or urine collection for iPSC generation']}], 'interventions': [{'name': 'Biological - skin biopsy, blood draw, or urine collection for iPSC generation', 'type': 'PROCEDURE', 'description': 'This study involves a single, minimally invasive biological sampling to obtain somatic cells for the generation of induced pluripotent stem cells (iPSCs). Each participant will undergo only one procedure: a 3 mm skin punch biopsy under local anesthesia, a peripheral blood draw (up to 20 mL), or a urine collection (up to 300 mL), depending on laboratory needs. Samples will be processed to isolate fibroblasts, blood cells, or urine-derived epithelial cells, which will be reprogrammed into iPSCs using a non-integrating RNA-based system . The resulting iPSCs will be characterized for pluripotency and differentiated into insulin-producing β cells.', 'armGroupLabels': ['Biospecimen Collection for iPSC Generation and β Cell Differentiation']}]}, 'contactsLocationsModule': {'locations': [{'zip': '20132', 'city': 'Milan', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Valeria Sordi, PhD', 'role': 'CONTACT', 'email': 'sordi.valeria@hsr.it', 'phone': '+390226432643'}, {'name': 'Lorenzo Piemonti, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Valeria Sordi, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Graziano Barera, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Diabetes Research Institute', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}], 'centralContacts': [{'name': 'Valeria Sordi, PhD', 'role': 'CONTACT', 'email': 'sordi.valeria@hsr.it', 'phone': '+390226432643'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ospedale San Raffaele', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor of Endocrinology Director, Diabetes Research Institute, Director, Regenerative Medicine and Transplant Unit', 'investigatorFullName': 'Lorenzo Piemonti', 'investigatorAffiliation': 'Ospedale San Raffaele'}}}}