The c4c project aims to develop a Pan European Paediatric Clinical Trial Network. As part of the development of the c4c network, a selection of Proof of Viability (PoV) studies (clinical trials) will be carried out within the c4c network to test its functionalities, and to build a more effective and sustainable network for the future.

The portfolio of Proof of Viability studies consists of three non-industry sponsored studies (due to open in 2020) and four industry sponsored studies (due to open in 2021).

 

Non-industry studies

The non-industry Proof of Viability studies were selected through an open call for studies that would address an unmet need identified by children, young people and clinicians. The criteria asked that studies encouraged innovative methodologies and designs and spanned multiple countries and were investigator-initiated paediatric clinical trials of medicines or investigational products. A total of 57 expressions of interest were received, resulting in 27 full applications being submitted, and following a robust selection process,11 applications were shortlisted.

 

The final selected c4c non-industry Proof of Viability studies includes three studies covering different diseases and age groups. These are:

TREOCAPA 

 

Paracetamol in
Premature Babies

This study will assess the effectiveness of paracetamol on the closure of the ductus arteriosus and the increase in surviving without severe morbidity in extremely premature infants. The study aims to recruit around 800 babies in 17 European countries

KD – CAAP 

 

Kawasaki Disease Coronary Artery
Aneurysm Prevention trial

This study will assess the effectiveness of adding steroids to standard treatment in children with Kawasaki Disease, and aims to recruit 262 children as part of the study.

cASPerCF

Prospective validation & clinical evaluation of
a new posaconazole dosing regimen for children
& adolescents with cystic fibrosis and Aspergillus

This study has been discontinued.

Industry studies

c4c has four industry Proof of Viability studies currently running or shortly running through its network.

Operetta 2

Operetta 2 is a clinical research study for children and teenagers living with Relapsing-Remitting Multiple Sclerosis (RRMS). The study will look at the effects, good or bad, of Ocrelizumab to understand if it can help treat RRMS in children and teenagers.
Operetta 2 is sponsored by Hoffmann-La Roche. The study is utilizing the c4c network to support the running of the study.
Interested to find out more? Please visit the Operetta 2 website or clinicaltrials.gov

FIONA

A 6-month multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety and pharmacokinetics and -dynamics of an age-and body weight-adjusted oral finerenone regimen, in addition to an ACEI or ARB, for the treatment of children, 6 months to <18 years of age, with chronic kidney disease and proteinuria (EudraCT Number: 2021-002071-19; NCT05196035;)

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Janssen IBD Study

This study is a Phase 3 study in paediatric subjects aged 2 to 17 years with moderately to severely active ulcerative colitis.

Novartis Asthma Study

This is a Phase 3 study in children aged 6 to <12 years with moderate-severe asthma.