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Diabetes Mellitus, Type 1Phase 2PRIFENNCT05909800

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

A Phase 2 study testing Phenofibrate in people with Diabetes Mellitus, Type 1, run by Medical University of Warsaw at 2 sites in Poland.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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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. Read more about this study

Who is it for?
Ages 10 to 17 years
Conditions the study is for
Diabetes Mellitus, Type 1
What is being tested?
Phenofibrate
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
102 participants
Where
2 sitesPoland
Study dates
29 Sep 2022to Jul 2024
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 10 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.
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  • 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.
  • Females (menstruating) must have a negative urine beta-human chorionic gonadotropin hormone (hCG) pregnancy test at Visit 0.

Who cannot join

  • 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.
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  • 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.
  • The presence of circumstances that the Investigator considers problematic when obtaining informed consent or meeting the study guidelines, or that may invalidate the interpretation of test results or expose Subjects to unnecessary risk.
  • Inability or unwillingness to comply with study procedures.
  • Any medical condition or treatment the Investigator believes may expose the Subject to unnecessary risk during the study.
  • Participation in interventional or other drug research studies which could affect the objectives of this study.

Trial locations and contacts

2 sites in 1 country.

Agnieszka Szypowska, MD, PhD,Prof contact

+48223179426

agnieszka.szypowska@uckwum.pl

Agnieszka Kowalska, MD, PhD contact

+48223179426

agnieszka.kowalska@uckwum.pl

Poland2 sites, 2 recruiting
  • Clinical department of pediatric diabetology and paediatrics, DSK UCKWUM

    Warsaw, Masovian Voivodeship, 02-091

    Agnieszka Szypowska, MD, PhD, Professor, +480223179425, agnieszka.szypowska@uckwum.pl

    Agnieszka Kowalska, MD, PhD, +480223179425, agnieszka.kowalska@uckwum.pl

    Recruiting
  • Diabetology Department, Children's Memorial Health Institute

    Warsaw, Masovian Voivodeship, 04-730

    Marta Wysocka- Mincewicz, MD, PhD, +48 22 815 75 78, oddzial.diabetologia@ipczd.pl

    Marta Wysocka- Mincewicz, MD, PhD

    Recruiting

Other trials in Poland

About this study

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.

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.

Read the detailed 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.
Being tested
Phenofibrate, Placebo
Keywords
Diabetes Mellitus, sulfatide, phenofibrate, children, adolescents, randomized controlled trial, fenofibrate
Medical subject headings
Diabetes Mellitus, Type 1, Diabetes Mellitus

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PhenofibrateDrug

    Also called Fenofibrate, Fenofibratum, SUB07576MIG, CAS no. 49562-28-9

    Administred orally, once daily, for 12 months.

  • PlaceboDrug

    Also called inactive drug, inactive medicine, inactive substance.

    Administred orally, once daily, for 12 months.

Study groups in NCT05909800
Study groupRoleWhat they receive
PhenofibrateExperimentalPhenofibrate in capsules received orally, daily, for 12 months.
Receives: Drug: Phenofibrate
PlaceboPlacebo comparatorCapsules 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.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 12 months

Secondary outcomes (11)
  1. Differences in C-peptide concentration in the stimulation test: change in the insulin secretion measured on the basis of the fasting C-peptide concentration

    Fasting C-peptide concentration in the stimulation test

    Measured: 0, 6, 12 months

  2. Differences in parameters of diabetes control

    HbA1c

    Measured: 0,3,6,9,12 months

  3. Daily insulin requirement

    Daily insulin requirement/kg of body mass

    Measured: 0,3,6,9,12 months

  4. Interleukins

    IL1, IL2, IL10, TNF alpha, IFN gamma

    Measured: 0,6,12 months

  5. Adverse Events occurence

    Safety will be evaluated through assessment of AEs, vital signs, physical examinations, USG findings, and laboratory evaluations. Any clinically significant laboratory occurring after study drug initiation must be reported by the investigator as an AE and/or SAE, as appropriate, and must be followed by additional laboratory evaluations until they return to normal range, stabilize, or until the change is no longer clinically relevant. All safety analyses will be conducted using the Safety Analysis Set. Adverse event data will be presented and tabulated according to MedDRA classification.

    Measured: 0,3,6,9,12 months

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

    Maximum C-peptide concentration

    Measured: 0,6,12 months

  7. Diabetes control and glucose fluctuations

    Mean blood glucose with standard deviation

    Measured: 0,3,6,9,12 months

  8. Differences in glucose fluctuations

    Glucose variability index (CV%)

    Measured: 0,3,6,9,12 months

  9. Parameter of glucose fluctuations

    Time in range 70-180mg/dl

    Measured: 0,3,6,9,12 months

  10. Difference in autoantibodies

    Difference in anti-insulin IAA antibodies, antibodies against glutamic acid decarboxylase (GADA), antibodies to tyrosine phosphatase (IA2A), anti-zinc transporter antibodies 8 determination.

    Measured: 0,6,12 month

  11. Genetical analysis

    WES Whole Exome Sequencing and HLA

    Measured: 1 per study

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jun 2023
  2. Study start29 Sep 2022
  3. Primary completionJul 2024 (estimated)
  4. Study completionJul 2024 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
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.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
102 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05909800
Sponsor's protocol ID
PRIFEN-01
Other IDs
2020-003916-28
Acronym
PRIFEN
Status last verified
Oct 2023
First submitted
29 Nov 2022
Last updated
26 Oct 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: The data relevant to the study will be included in the article or uploaded as supplementary information

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Phenofibrate in people with Diabetes Mellitus, Type 1, run by Medical University of Warsaw at 2 sites in Poland.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 26 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Agnieszka Szypowska, MD, PhD,Prof (+48223179426, agnieszka.szypowska@uckwum.pl), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Differences in AUC in C-peptide stimulation test (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2023.

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