Prolonged Remission Induced by Phenofibrate in Children Newly Diagnosed With Type 1 Diabetes. — PRIFEN  

Diabetes Mellitus, Type 1 Clinical Trial

Official title: Randomized, Double-blind, Multicenter, Parallel-group, Placebo-controlled Study to Evaluate the Efficacy of Phenofibrate Treatment on the Functions of Beta Cells in Children and Adolescents With Newly Diagnosed of Type 1 Diabetes.

Status Recruiting

Clinical Trial Summary

The goal of this clinical trial is to evaluate of the effect of phenofibrate on the functions of beta cells in children with new diagnosis of type 1 diabetes. The main question it aims to answer is: whether phenofibrate may prolong residual beta-cell function therefore own insulin secretion. Participants will be asked to take a phenofibrate or identically appearing placebo (a neutral substance), orally, once daily, for 12 months with no knowledge what is administred to them. They will be invited for follow-up visits including blood tests every 3 months. Researchers will be monitoring the two groups for the safety of the phenofibrate, and at the trial end they compare the residual insulin secretion results in two groups.

Clinical Trial Description

Rationale:

Preservation of residual pancreatic beta cell function in children with newly diagnosed T1D gives a chance for better diabetes control, reduction of chronic diabetes complications, and possibly temporary insulin withdrawal. Indication of a cheap drug for secondary prevention of T1D.

Setting:

Recruitment will be through the paediatric diabetes clinics at two participating centres in Warsaw, Poland (Department of Paediatrics, the Medical University of Warsaw and Department of Endocrinology and Diabetology, Children's Memorial Health Institute).

The initiation of study treatment may be performed no later than 28 days after screening visit, and no later than in 8 weeks from diabetes diagnosis.

PICO:

Adolescent participants meeting inclusion criteria, newly diagnosed with type 1 diabetes will be randomly assigned to two groups, receiving either fenofibrate at a dose of 160 mg or placebo, and regularly assesed, every 3 months for the next year. Assuming increase by 50% of AUC of C-peptide in the test group compared to placebo, 88 subjects are needed to achieve power of 85%. If about 13% drop-out is assumed the total group size is 102 patients. Given randomization ratio 1:1, there is 51 patients in each group.

Main study procedures:

• Demographic and medical history.

• Physical examination and vital signs: heart rate and blood pressure, respiration rate, body temperature.

• Blood collection for laboratory analysis (hematology: morphology with automatic blood smear, biochemistry: ALT, AST, total bilirubin, albumin, amylase, lipase, total cholesterol, HDL, LDL, triglycerides, GGTP, HbA1c, TSH, FT4, Anti-Tg, Anti-TPO, CK, creatinine, urea, vitamin D, homocysteine, Anti-tTG IgA, IgA.

• Urine pregnancy test (in girls with childbearing potential).

• C-peptide and Glucose in MMTT stimulation assay.

• Anti-insulin IAA antibodies, Antibodies to glutamic acid decarboxylase (GADA), Antibodies to tyrosine phosphatase (IA2A), Antibodies to the zinc transporter 8 (ZnT8).

• IL1, IL2, IL10.

• TNF alpha, IFN gamma.

• Genetic study - WES genome sequencing, HLA DR3, DR4, DO8, DQ7.

• FGM Libre Free Style Glucose Monitoring System.

• Abdominal ultrasound.

• Ultrasound of the thyroid gland.

• Safety monitoring and AE collection.

• IMP administration. Compliance will be assessed by collecting empty packages as well as by direct interview and participants receiving <75% of the recommended doses will be considered as non-compliant. ;

Related Conditions & MeSH terms

• Diabetes Mellitus
• Diabetes Mellitus, Type 1

• NCT number: NCT05909800
• Study type: Interventional
• Source: Medical University of Warsaw
• Contact: Agnieszka Szypowska, MD, PhD,Prof
• Phone: +48223179426
• Email: agnieszka.szypowska@uckwum.pl
• Status: Recruiting
• Phase: Phase 2
• Start date: September 29, 2022
• Completion date: July 2024