PMID- 38341215
OWN - NLM
STAT- MEDLINE
DCOM- 20240214
LR  - 20240215
IS  - 2044-6055 (Electronic)
IS  - 2044-6055 (Linking)
VI  - 14
IP  - 2
DP  - 2024 Feb 10
TI  - Prolonged Remission Induced by FENofibrate in children with newly diagnosed type 
      1 diabetes (PRIFEN): protocol of a randomised controlled trial.
PG  - e076882
LID - 10.1136/bmjopen-2023-076882 [doi]
LID - e076882
AB  - INTRODUCTION: Sphingolipids regulate proinsulin folding, insulin secretion and 
      control beta cells apoptosis. Recent evidence has demonstrated that, among other 
      factors, reduced amounts of sulfatide may be relevant in the development of type 
      1 diabetes (T1D). Thus, fenofibrate, which activates sulfatide biosynthesis, may 
      prolong remission in subjects with T1D. The aim of the study is to evaluate 
      clinical efficacy of fenofibrate on the maintenance of residual beta-cell 
      function in children with newly diagnosed T1D. METHODS AND ANALYSIS: A total of 
      102 children aged 10-17 years with newly diagnosed T1D will be enrolled in a 
      double-blind, two-centre randomised, non-commercial, placebo-controlled trial. 
      Subjects who will meet all inclusion criteria will be randomly assigned to 
      receive fenofibrate at a dose of 160 mg or an identically appearing placebo, 
      orally, once daily, for 12 months. The primary endpoint will be the area under 
      the curve of the C-peptide level during 2-hour responses to a mixed-meal 
      tolerance test (MMTT). Secondary endpoints include fasting and maximum C-peptide 
      concentration in the MMTT, parameters of diabetes control and glucose 
      fluctuations, daily insulin requirement, inflammation markers, genetic analysis, 
      safety and tolerance of the fenofibrate ETHICS AND DISSEMINATION: The study 
      protocol was approved by the Bioethics Committee. The results of this study will 
      be submitted to a peer-reviewed diabetic journal. Abstracts will be submitted to 
      international and national conferences. TRIAL REGISTRATION NUMBER: EnduraCT 
      2020-003916-28.
CI  - (c) Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No 
      commercial re-use. See rights and permissions. Published by BMJ.
FAU - Groele, Lidia
AU  - Groele L
AD  - Department of Paediatrics, Medical University of Warsaw, Warsaw, Poland.
FAU - Dzygalo, Katarzyna
AU  - Dzygalo K
AD  - J.P. Brudzinski Children's Clinical Hospital, University Clinical Centre of 
      Warsaw Medical University, Warsaw, Poland.
FAU - Kowalska, Agnieszka
AU  - Kowalska A
AUID- ORCID: 0000-0002-9836-2987
AD  - J.P. Brudzinski Children's Clinical Hospital, University Clinical Centre of 
      Warsaw Medical University, Warsaw, Poland.
FAU - Szypowska, Agnieszka
AU  - Szypowska A
AUID- ORCID: 0000-0002-3407-3174
AD  - Department of Paediatrics, Medical University of Warsaw, Warsaw, Poland 
      agnieszka.szypowska@wum.edu.pl.
AD  - J.P. Brudzinski Children's Clinical Hospital, University Clinical Centre of 
      Warsaw Medical University, Warsaw, Poland.
LA  - eng
PT  - Clinical Trial Protocol
PT  - Journal Article
DEP - 20240210
PL  - England
TA  - BMJ Open
JT  - BMJ open
JID - 101552874
RN  - U202363UOS (Fenofibrate)
RN  - 0 (C-Peptide)
RN  - 0 (Sulfoglycosphingolipids)
RN  - 0 (Insulin)
SB  - IM
MH  - Child
MH  - Humans
MH  - *Diabetes Mellitus, Type 1/drug therapy
MH  - *Fenofibrate/therapeutic use
MH  - C-Peptide
MH  - Sulfoglycosphingolipids/therapeutic use
MH  - Insulin/therapeutic use
MH  - Double-Blind Method
MH  - Randomized Controlled Trials as Topic
PMC - PMC10862295
OTO - NOTNLM
OT  - Child protection
OT  - Chronic Disease
OT  - Clinical Trial
OT  - DIABETES & ENDOCRINOLOGY
OT  - PREVENTIVE MEDICINE
OT  - Paediatric endocrinology
COIS- Competing interests: None declared.
EDAT- 2024/02/11 07:42
MHDA- 2024/02/11 16:43
PMCR- 2024/02/10
CRDT- 2024/02/10 20:53
PHST- 2024/02/11 16:43 [medline]
PHST- 2024/02/11 07:42 [pubmed]
PHST- 2024/02/10 20:53 [entrez]
PHST- 2024/02/10 00:00 [pmc-release]
AID - bmjopen-2023-076882 [pii]
AID - 10.1136/bmjopen-2023-076882 [doi]
PST - epublish
SO  - BMJ Open. 2024 Feb 10;14(2):e076882. doi: 10.1136/bmjopen-2023-076882.