Evaluation of the effect of phenofibrate on the functions of beta cells in children with new diagnosis of type 1 diabetes

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Medical University Of Warsaw

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hormonal diseases [C19]

Trial duration

21 Sep 2022 → 31 Jan 2026

Decision date (initial)

2024-09-22

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-517483-34-00

EudraCT number

2020-003916-28

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Efficacy

Evaluation of the effectiveness of fenofibrate at a dose of 160 mg / day in maintaining residual pancreatic beta cell function in children with newly diagnosed type 1 diabetes (T1DM).

Secondary objectives 1

1. Confirm safety and tolerability of fenofibrate at a dose of 160 mg / day in subjects with newly diagnosed type 1 diabetes (T1DM).

Conditions and MedDRA coding

Evaluation of the effect of phenofibrate on the functions of beta cells in children with new diagnosis of type 1 diabetes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Subject and 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 Participant”. •Adolescents from 10-15 years sign “Children Assent form”. •Adolescents from 16-17 years sign “Adolescent Assent form”.
2. Age ≥10 and ≤17 years.
3. Diagnosis of type 1 diabetes according to the criteria of Polskie Towarzystwo Diabetologiczne within 8 weeks before randomization.
4. 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 V0 visit and through study completion. •Stable hormonal contraceptive for at least 2 months prior to the Visit 0 and continuing through study completion.
5. Females (menstruating) must have a negative blood or urine beta-human chorionic gonadotropin hormone (hCG) pregnancy test at Visit 0.

Exclusion criteria 18

1. Age under 10 or over 17.
2. Lack of consent of at least one the guardian LAR to participate in the study.
3. Treatment with any oral or injected anti-diabetic medications other than insulin.
4. The participant or close participant’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).
5. Severe hypersensitivity reaction to any other drug.
6. Subjects with current or history of clinically significant renal impairment.
7. Subjects with current or history of clinically significant hepatic impairment.
8. Subjects with current or history of significant gastrointestinal disease including celiac disease, gastroparesis, another disorder of intestinal absorption or motility.
9. Subject with current or history of gall bladder disease.
10. Present or history of chronic or acute pancreatitis, except acute pancreatitis due to severe hypertriglyceridaemia.
11. Photosensitivity or phototoxic reactions after the use of fibrates or chemically related substances, e.g. ketoprofen.
12. Subjects who tested positive for pregnancy at screening and V0 Visit or who are currently breastfeeding.
13. Low blood albumin defined as clinically significant by investigator.
14. 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.
15. The presence of circumstances that the researcher considers problematic when obtaining informed consent or meeting the study guidelines, or that may invalidate the interpretation of test results or expose participants to unnecessary risk.
16. Inability or unwillingness to comply with study procedures.
17. Any medical condition or treatment the Investigator believes may expose the Participant to unnecessary risk during the study.
18. Participation in interventional or other drug research studies which could affect the objectives of this study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Assessment of pancreatic beta cell function by comparing the AUC area under the curve in the C-peptide stimulation test: Change in the mean insulin secretion measured based on the C-peptide area under the curve in the stimulation test at individual time points in the compared groups of patients

Secondary endpoints 1

1. • Fasting c-peptide concentration and maximum c-peptide concentration in the stimulation test, • Parameters of diabetes control and glucose fluctuations (including HbA1c, mean blood glucose with standard deviation, variability index, time spent in normoglycemia), • Daily and basic insulin requirements • Inflammation markers • Safety and tolerance of the fenofibrate

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Medical University Of Warsaw

Sponsor organisation

Medical University Of Warsaw

Address

Ul. Zwirki I Wigury 61

City

Warsaw

Postcode

02-091

Country

Poland

Scientific contact point

Organisation

Medical University Of Warsaw

Contact name

Agnieszka Szypowska

Public contact point

Organisation

Medical University Of Warsaw

Contact name

Agnieszka Szypowska

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 7 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications