Projects

One of the key goals of the Conect4Children consortium (c4c) is the selection and funding of Proof-of-viability (non-commercial and Industry) studies. On April 29, 2019, the selection of the first portfolio of pan-European pediatric academic studies was published:

  • Paracetamol in Premature Babies: Will assess the effectiveness of paracetamol in premature babies with a patent ductus arteriosus, and aims to recruit around 600 babies as part of the study (Lead: Prof. Jean-Christophe Roze of INSERM, a public research organization in Paris, France entirely dedicated to human health).
     
  • Steroids to Treat Kawasaki Disease: 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 (Leads: Dr. Despina Eleftheriou and Prof Paul Brogan of the University College of London (UCL), an academic research institution in the UK).

  • Posaconazole in Children with Cystic Fibrosis: Will assess the dose of posaconazole in children and young people with Cystic Fibrosis and infection with Aspergillus and aims to recruit 130 children as part of the study (Lead: Prof. Adilia Warris of the MRC Centre for Medical Mycology, University of Aberdeen and Ospedale Pediatrico Bambino Gesù (OPBG), a children’s hospital in Rome, Italy).
     
  • Losartan to Treat Osteogenesis Imperfecta: Will assess losartan in children and young people with Osteogenesis Imperfecta and aims to recruit 30 children (Lead: Prof. Nick Bishop of the University of Sheffield, an academic research institution in the UK).

TREOCAPA

SponsorFrench National Institute of Health and Medical Research (INSERM)
Therapeutic AreaPaediatrics, Neonatology
Study TitleProphylactic treatment of the ductus arteriosus in preterm infants by acetaminophen
Study AcronymTREOCAPA
Study TypeInterventional
CT PhasePhase II/III
Nº EudraCT2019-004297-26
Number of Countries17
Number of Sites
(Portugal)
3
Portuguese sites and PIsCHULN, EPE – HSM, André Graça, MD, PhD
CHUP, EPE -CMIN, Elisa Proença, MD
2CA – Braga, Almerinda Pereira, MD
Target of Patients  (Global)Phase II: 30
Phase III: 794
Target of Patients (Portugal)Phase III: 27
% recruitment achivedUnder submission
National CoordinatorAndré Graça, MD, PhD
StatusStart-up

cASPerCF

SponsorOspedale Pediatrico Bambino Gesu (OPBG)
Therapeutic AreaPaediatrics, Pulmonology
Study TitleProspective validation and clinical evaluation of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection
Study AcronymcASPerCF
Study TypeInterventional
CT PhasePhase II/III
Nº EudraCT2019-004511-31
Number of Countries12
Number of Sites
(Portugal)
1
Portuguese sites and PIsCHULN, EPE -HSM, Celeste Barreto, MD
Target of Patients (Global)Large screening phase: 1500
Intervention phase: 135
Target of Patients  (Portugal)Large screening phase: 25
Intervention phase: 4
% recruitment
achieved
(Portugal)
Under submission
National
Coordinator
N/A
StatusStart-up

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 777389. The Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA