Therapeutic approach in Colchicine-resistant Recurrent pEricarditis in children: an open-label randomized trial comparing Anakinra vs sTEroids

Phase 1

• Conditions: Recurrent pericarditis, idiopathic or secondary to invasive cardiac procedures, not responsive to colchicine and NSAIDs after the first relapse.; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]; MedDRA version: 20.0Level: PTClassification code: 10034484Term: Pericarditis Class: 100000004849; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: CTIS2023-504756-96-00
• Lead Sponsor: Giannina Gaslini Institute For Scientific Hospitalization And Care

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-03-31
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 48

Inclusion Criteria

Male and female patients, from 8 months to less than 18 years, Parent or legal guardian written informed consent and child assent, if appropriate, are required before any assessment is performed, Diagnosis of relapse of pericarditis in a patient with previous diagnosis of acute pericarditis (idiopathic or secondary to invasive cardiac procedures), Inadequate response or intolerance to non-steroidal anti-inflammatory drugs or colchicine

Exclusion Criteria

Pericarditis secondary to a known infection (viral, bacterial, mycobacterial), Any conditions or significant medical problems, which in the opinion of the investigator places the patient at unacceptable risk for immunomodulatory therapy, Main alteration in the blood count: Neutropenia (absolute neutrophil count <1000/mmc), Thrombocytopenia (platelets <100.000/mmc) or Severe anaemia (Hb < 7.5 mg/dl), Presence of Human Immunodeficiency Virus (HIV) infection, Hepatitis B or Hepatitis C infections, Evidence of active or latent tuberculosis (TB) determined by positive QuantiFERON (QFT-TB G In-Tube) test or positive Purified Protein Derivative (PPD) test (>5 mm induration) within 2 months prior to randomization, Presence of laboratory or echocardiographic findings of myopericarditis, Administration of any investigational drug or implantation of investigational device, or participation in another trial, within 30 days before screening, Use of steroids at the dosage of 1 mg/kg/day of prednisone or equivalent for at least 5 days in the 30 days before randomization, Live vaccinations within 1 months prior to the start of the trial and during the trial, Pregnancy, confirmed by a positive hCG laboratory test, Female adolescents (<18 years of age) of childbearing potential who do not agree to abstinence or, if sexually active, do not agree to the use of contraception, Pericarditis in a patient with a previous diagnosis of any neoplasm and without complete recovery from at least one year, Patients fulfilling diagnostic criteria for an autoimmune systemic disease, Patients with a previous diagnosis of a genetically confirmed autoinflammatory disease

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Main objective of this study is to demonstrate that anakinra is more effective than steroids to control the disease and prevent further flares in paediatric (from 8 months to < 18 years) patients with RP not responding to first line treatment with NSAIDS and colchicine at the proper dosage, or in case of colchicine intolerance.;Secondary Objective: In the study we aim to collect the RNA to perform trascriptomic study in the patients enrolled in the study at different timepoints (at baseline, at day 14, 28 and, in responder patient, at the time of the relapse or at last visit of the study). Through a correlation between the clinical, laboratory parameters and the response to treatment with the trascriptome analysis, we aim to identify transcripts associated to a higher probability of response to treatment and long-term remission off-therapy.;Primary end point(s): Complete response of disease flare at day 7, Lack of relapse at month 3

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):Time to achieve a complete control of the flare;Secondary end point(s):Time to flare after treatment tapering;Secondary end point(s):Number of flares at follow up;Secondary end point(s):Behavior of acute phase reactants during 6 months of observation;Secondary end point(s):Quality of life;Secondary end point(s):Safety (registration of side effects occurred during the whole period of the study);Secondary end point(s):Number of day of hospitalization;Secondary end point(s):Transcriptomic study