Research Initiatives

Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicenter study to evaluate safety, tolerability and efficacy of inclisiran in children (2 to less than 12 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-19)

Two-part randomized multicenter study (double-blind inclisiran versus placebo wpcodeself followed by open-label inclis[Anno 1]iran wpcodeself).[Anno 2]

Two-part randomized multicenter study (double-blind inclisiran versus placebo [Year 1] followed by open-label study with inclisiran [Year 2]) for[Anno 2][Anno 1]

evaluate the safety, tolerability and efficacy of inclisiran in children (2 to less than 12 years) to less than 12 years) with homozygous familial hypercholesterolemia and

with heterozygous familial hypercholesterolemia with elevated LDL cholesterol (ORION 19-20)

It is a multicenter, phase III study designed to evaluate the safety, tolerability and efficacy of inclisiran in children (aged 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) and heterozygous familial hypercholesterolemia (HeFH) with low lipoprotein cholesterol. density (LDL) high.< The use of inclisiran for the treatment of HoFH in children will be studied in order to obtain the necessary pediatric information on inclisiran.

Double-blind, randomized, active-controlled, two-way cross-over study, with 12-week treatment duration per period, to evaluate the efficacy and safety of QMF149 (indacaterol acetate / mometasone furoate) compared to budesonide in children from 6 to less than 12 years of age with asthma?

Double-blind, randomized, active-controlled, two-way cross-over study, with 12-week treatment duration per period, to evaluate the efficacy and safety of QMF149 (indacaterol acetate / mometasone furoate) compared to budesonide in children from 6 to less than 12 years of age with asthma

The purpose of this study is to evaluate the efficacy and safety of QMF149 (indacaterol acetate/mometasone furoate) compared to budesonide in children aged 6 to less than 12 years with mild to moderate asthma.

The primary objective of this study is to demonstrate the superiority of indacaterol (acetate)/mometasone (furoate) (QMF149) over budesonide in terms of FEV1 at week 12 of each treatment period.

Approximately 200 pediatric patients will be randomized to obtain 182 complete pediatric asthma patients.

FInerenone for the treatment of children with chrOnic kidNey disease and proteinuriA

A 6-month, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and PK/PD of an age- and bodyweight-adjusted oral finerenone regimen, as an adjunct to an ACEI or ARB, for the treatment of children aged 6 months <to <18 years.

ACEI or ARB, for the treatment of children aged 6 months to <18 years with chronic kidney disease and proteinuria.<

Finerenone has been shown to be safe and effective in reducing proteinuria and decreasing the risk of renal events, such as renal failure, and cardiovascular events in high-risk populations with chronic kidney disease (CKD) and type 2 diabetes mellitus (T2D).

The objective of the study is to demonstrate that Finerenone, in addition to an ACEI or an ARB, is safe and effective in reducing urinary protein excretion which represents a modifiable risk factor for worsening renal function in children with CKD and severely increased proteinuria.

The extension study, FIONA OLE, will be an 18-month, single-arm, open-label, multicenter study to evaluate the long-term safety and efficacy of an age- and body weight-adjusted oral finerenone regimen, as an adjunct to an ACEI or an ARB, for the treatment of pediatric and young adult patients, aged 1 <to <19 years, with CKD stages 1-3. Only patients who have completed the double-blind FIONA study will be eligible for inclusion in the FIONA OLE study.

The main aim of the study is to provide data on the long-term safety of Finerenone in the treatment of pediatric and young adult patients with CKD.

A phase III multicenter, randomized, double-blind, double-dummy study to evaluate safety and efficacy of ocrelizumab in comparison with fingolimod in children and adolescents with relapsing-remitting multiple sclerosis

Studio di fase III multicentrico, randomizzato, in doppio cieco e con doppia falsa testimonianza per valutare la sicurezza e l'efficacia di ocrelizumab rispetto a fingolimod in bambini e adolescenti con sclerosi multipla recidivante-remittente

It is a double-blind phase III study, conducted in countries around the world and sponsored by Roche.

The aim of this study is to compare the effects, positive or negative, of the study drug compared to the comparator drug in the treatment of relapsing-remitting multiple sclerosis (RRMS) in children and adolescents.

Multiple sclerosis (MS) is an unpredictable, sometimes disabling autoimmune disease that affects the nervous system. Most children and adolescents with MS have a type of MS called relapsing-remitting MS (RRMS). RRMS is the most common type of MS and is known to have periods in which new or existing symptoms worsen (relapse), followed by a recovery period in which there are few or no symptoms (remission). Durante le recidive della SMRR, il sistema immunitario attacca le fibre nervose e During RRMS relapses, the immune system attacks nerve fibers and causes inflammation (or swelling), causing MS symptoms. In people with MS the body's immune system attacks and damages a fatty substance that surrounds and protects the nerves, called myelin. As a result, the nerve impulses (or messages) going to and from the brain are unclear or interrupted, like a frayed electrical cord that isn't working properly. 233 children and adolescents from all over the world affected by RRMS aged between 10-17 years will participate in the study.

The study drug and the comparator are medicines that temporarily reduce inflammation caused by the immune system and are intended to reduce the frequency of relapses.

Prospective validation and clinical evaluation of a new dosage of posaconazole for children and adolescents with cystic fibrosis and Aspergillus infection.

Cystic fibrosis (CF) is the most common hereditary disabling disease in the Northern European population, affecting 90,000 people worldwide and with approximately 45,000 registered in the European Cystic Fibrosis Society (ECFS) Patient Registry. Progressive lung damage caused by recurrent infection and persistent inflammation is the major determinant of survival, with a median age of death of 29 years. Approximately 60% of CF patients are infected with A. fumigatus, a ubiquitous environmental fungus, and its presence is associated with an accelerated decline in lung function. Half of patients with Aspergillus infection are under 18 years of age. No specific evidence is available to guide the clinical management of CF-related Aspergillus disease. A recent survey showed considerable variability in clinical practice among CF consultants.

The primary objective of the study is to define the dose of posaconazole for children and adolescents with CF and Aspergillus infection in terms of clearance of Aspergillus from the airways after three months.

Approximately a year into the study, the C4C CPS reviewed the cASPerCF study and decided to withdraw funding because there was no prospect of completing the study, as initially agreed Since the study was funded, the standard of care for the target population has changed significantly. This change led to a marked improvement in the quality of life of potential study participants and reduced the sites' ability to recruit participants.

At the time of study completion, 29 patients were included in the large initial screening phase and 1 patient progressed to the intervention phase (the latter was assigned to the control arm).

• Link to official website: https://www.clinicaltrialsregister.eu/ctr-search/search?query=treocapa

• Link to Trial website: https://treocapa.inserm.fr/

• Link to official website: https://www.clinicaltrialsregister.eu/ctr-search/trial/2019-004433-17/NL#A

• Link to Trial website: http://kdcaap.mrcctu.ucl.ac.uk/