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

NCT05909800 | Unknown Phase 2 DMC

• 2 other identifiers: PRIFEN-012020-003916-28
• Study Type: interventional
• Target: 102
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Diabetes Mellitus, Type 1

Keywords

Diabetes Mellitus; sulfatide; phenofibrate; children; adolescents; randomized controlled trial; fenofibrate

Outcome Measures

Primary Outcomes (1)

• Differences in AUC in C-peptide stimulation test

  Assessment of pancreatic beta cell function by comparing the area under the curve (AUC) in the C-peptide stimulation test: Change in the mean insulin secretion measured on the basis of the C-peptide area under the curve in the stimulation test

  12 months

Secondary Outcomes (11)

• Differences in C-peptide concentration in the stimulation test: change in the insulin secretion measured on the basis of the fasting C-peptide concentration

  0, 6, 12 months

• Differences in parameters of diabetes control

  0,3,6,9,12 months

• Daily insulin requirement

  0,3,6,9,12 months

• Interleukins

  0,6,12 months

• Adverse Events occurence

  0,3,6,9,12 months

Study Arms (2)

Phenofibrate

EXPERIMENTAL

Phenofibrate in capsules received orally, daily, for 12 months.

Drug: Phenofibrate

Placebo

PLACEBO COMPARATOR

Capsules containing Microcrystalline cellulose 102,594 mg (99%) and Magnesium stearate 6 mg (1%) identical to those of the active product received orally, daily, for 12 months.

Drug: Placebo

Interventions

Phenofibrate DRUG

Administred orally, once daily, for 12 months.

Also known as: Fenofibrate, Fenofibratum, SUB07576MIG, CAS no. 49562-28-9

Phenofibrate

Placebo DRUG

Administred orally, once daily, for 12 months.

Also known as: inactive drug, inactive medicine, inactive substance.

Placebo

Eligibility Criteria

• Age: 10 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subjects who meet all of the following criteria are eligible to participate in this study:
• Subject or Legally accepted representative (LAR) able to understand and provide signed informed consent. Assent is also required of adolescents and children.
• LAR of subjects ≤ 17 years sign the "Information Leaflet and ICF for the Parent/Legal Guardian of Minor Subject".
• Adolescents from 10-15 years sign "Children Assent form".
• Adolescents from 16-17 years sign "Adolescent Assent form".
• Age ≥10 and ≤ 17 years.
• Diagnosis of type 1 diabetes within 8 weeks before randomization (V0 visit) based on positive autoantibody (minimum 1 among: GADA, IA2A, ZnT4, IAA) and symptoms of type 1 diabetes according to the criteria of the Polish Diabetes Association (1 of the following):
• symptoms of diabetes and blood glucose ≥ 200 mg / dl (≥ 11.1 mmol/l),
• when no symptoms or when diabetes symptoms are present and random glucose \<200 mg/dl (\<11.1 mmol/l) - then confirmation of the diagnosis is fasting blood glucose in 2 measurements ≥ 126 mg/dl (≥ 7.0 mmol/l); each test must be performed on a different day,
• in the absence of symptoms of hyperglycaemia and random glycaemia ≥ 200 mg/dl (11.1 mmol/l), fasting glucose ≥ 126 mg/dl (7.0 mmol/l) is a confirmation of the diagnosis,
• if once or twice fasting blood glucose is 100-125 mg / dl (5.6-6.9 mmol/l), or if fasting blood glucose is below 100 mg/dl (5.6 mmol/l) ) exists, If there is a reasonable suspicion of impaired glucose tolerance or diabetes mellitus, an oral glucose tolerance test (OGTT) should be performed. At the 120th minute of the OGTT, blood glucose ≥ 200 mg/dl (11.1 mmol/l) confirms the diagnosis of diabetes.
• Male or nonpregnant and nonlactating female who is abstinent or agrees to use effective contraceptive methods throughout the course of the study. Acceptable birth control methods are the following:
• Intrauterine device in place for at least 3 months.
• Use of condom or diaphragm with spermicide for at least 14 days prior to the Visit 0 visit and through study completion.
• Stable hormonal contraceptive for at least 2 months prior to the Visit 0 and continuing through study completion.

You may not qualify if:

• Subjects who meet any of the following criteria are not eligible to participate in this study:
• Age under 10 or over 17.
• Lack of consent of at least one the guardian LAR to participate in the study.
• Treatment with any oral or injected anti-diabetic medications other than insulin.
• The Subject or close Subject's family history, past or present of allergic or hypersensitivity reactions to fenofibrate or any of the excipients (including patients with hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption).
• Severe hypersensitivity reaction to any other drug.
• Subjects with current or history of clinically significant renal impairment.
• Subjects with current or history of clinically significant hepatic impairment.
• Subjects with or history of significant gastrointestinal disease including celiac disease, gastroparesis, another disorder of intestinal absorption or motility.
• Subject with current or history of gall bladder disease.
• Present or history of chronic or acute pancreatitis, except acute pancreatitis due to severe hypertriglyceridaemia.
• Photosensitivity or phototoxic reactions after the use of fibrates or chemically related substances, e.g. ketoprofen.
• Subjects who tested positive for pregnancy at screening and V0 visit or who are currently breastfeeding.
• Low blood albumin defined as clinically significant by investigator.
• Patients with pre-disposing factors for myopathy and/or rhabdomyolysis, including personal and familial history of hereditary muscular disorders. Unexplained persistent elevated creatine phosphokinase levels considered clinically significant by the investigator.

Sponsors & Collaborators

• Medical University of Warsaw: lead
• Children's Memorial Health Institute, Poland: collaborator

Study Sites (2)

Clinical department of pediatric diabetology and paediatrics, DSK UCKWUM

Warsaw, Masovian Voivodeship, 02-091, Poland

RECRUITING

Diabetology Department, Children's Memorial Health Institute

Warsaw, Masovian Voivodeship, 04-730, Poland

RECRUITING

MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Diabetes Mellitus

Interventions

Fenofibrate

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Fibric Acids; Isobutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Phenyl Ethers; Ethers; Benzophenones; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Phenols; Ketones

Study Officials

• Agnieszka Szypowska, MD, PhD,Prof

  Medical University of Warsaw

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Agnieszka Szypowska, MD, PhD,Prof

CONTACT

+48223179426; agnieszka.szypowska@uckwum.pl

Agnieszka Kowalska, MD, PhD

CONTACT

+48223179426; agnieszka.kowalska@uckwum.pl

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Participants, caregivers, investigators, outcome assessors, and the person responsible for the statistical analysis will be blinded to the intervention until completion of the study.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Deputy Head of the Clinical Department of Pediatric Diabetology and Pediatrics

Model Details: Participants will be randomly assigned to two groups, receiving either phenofibrate at a dose of 160 mg or placebo, orally, once daily, for 12 months. All study participants will be followed up for up to 12 months after the start of the intervention. Study visits at month 3, 6, 9 and 12 will be coordinated with diabetes outpatient clinic visits.

Study Record Dates

• First Submitted: November 29, 2022
• First Posted: June 18, 2023
• Study Start: September 29, 2022
• Primary Completion: July 1, 2024
• Study Completion: July 1, 2024
• Last Updated: October 26, 2023

Record last verified: 2023-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Avaliable with the publication
• Access Criteria: open access - web adress will be known after the publication of the paper

Locations

PL (2)