Urine Extracellular Vesicles: Non-invasive Biomarkers of Β-cell Function and Novel Therapeutic Agents in Diabetes

NCT06832215 | Active Not Recruiting

• 1 other identifier: OBS.SF.05.2023
• Study Type: observational
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

Diabetes mellitus is a common chronic disease with a huge socioeconomic burden worldwide. Type 1 Diabetes(T1D) accounts for nearly 95% of diabetes in pediatric age and a lifelong dependence on exogenous insulin. Its diagnosis is based on symptoms and/or autoantibodies, both identified too late to avoid the disease progress. Ideally, children should be screened whilst assymptomatic, when there is endogenous insulin production, but C-peptide and beta-cell function are starting to decline. Early diagnosis would allow interventions capable of preventing disease progress and/or to preserve beta-cell function, ultimately delaying/avoiding insulin dependence. Given their association with pathogenesis of diabetes, Extracellular Vesicles have emerged as potential biomarkers for diagnosis and progression of diabetes. This project proposes the development of a non-invasive biomarker of preclinical T1D, based on miRNA characterization in urine, allowing a timely identification of children that can benefit from preventive therapies and, in the future, to cure T1D.

Conditions

Diabetes Mellitus, Type 1; Diabetes Mellitus

Keywords

Non-invasive biomarker of beta cell function in urine

Outcome Measures

Primary Outcomes (2)

• Urine miRNAs extracellular vesicles molecular signatures for the study groups

  Characterize urine EV-derived miRNAs in the three study groups

  October/23 until July/2024

• Blood miRNAs extracellular vesicles molecular signatures for the study groups

  Characterize blood miRNAs extracellular vesicles molecular signatures for the study groups

  October/23 until July/2024

Study Arms (3)

Type 1 Diabetes Group

Type 1 Diabetes people under 18 years´old

Diagnostic Test: Blood and urine samples collection

Genetic-related Group

Brothers/sisters of T1D children and adolescents (without disease)

Diagnostic Test: Blood and urine samples collection

Control group

Not genetic related and no T1D people under 18 years' old

Diagnostic Test: Blood and urine samples collection

Interventions

Blood and urine samples collection DIAGNOSTIC_TEST

Blood and urine were collected at a single time point for all participants, from the tree study groups. Fasting for blood collection and the first urine in the morning were required.

Control group; Genetic-related Group; Type 1 Diabetes Group

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Children and adolescents under follow-up at Coimbra Pediatric Hospital - Coimbra University and Hospital Centre (CHUC), either in specific clinic (Diabetes - for T1D group) or that have been observed at a general endocrine or pediatrics clinics for a suspition of disease that was not confirmed (for control group). For the genetic related group, children and adolescents were recruited within T1D families and were observed in a single time point, at the time of their brother/sister.

You may qualify if:

• T1D Group: children diagnosed with T1D, according to internationally defined criteria, with at least one positive pancreatic antibody and under functional insulin.
• Genetic-related group: children without T1D, age- and sex-matched with T1D group, recruited among T1D relatives;
• Control group: children without T1D, age- and sex-matched with T1D group, recruited from general endocrinology clinics, among children without disease or genetic relation with T1D children;

You may not qualify if:

• Obesity, according to WHO standards for pediatrics;
• Hypertension, as ≥95th percentile according to International Consensus;
• Other auto-immune diseases;
• Diabetes in the context of syndromic features/secondary to treatments;
• Hypothyroidism, adrenal insufficiency or hypercortisolism;
• Children under somatotropin/oncologic treatment;
• Under medications affecting glucose metabolism

Sponsors & Collaborators

• Unidade Local de Saúde de Coimbra, EPE: lead
• Università di Siena: collaborator

Study Sites (1)

Coimbra Pediatric Hospital - CHUC

Coimbra, 3000-602, Portugal

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Diabetes Mellitus

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, Consultant in Pediatrics (Graduated), Pediatric Endocrinologist and Diabetologist

Study Record Dates

• First Submitted: February 12, 2025
• First Posted: February 18, 2025
• Study Start: May 6, 2023
• Primary Completion: October 15, 2023
• Study Completion (Estimated): March 30, 2027
• Last Updated: February 18, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

PT (1)