Clinical Studies

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EPTRI is offering public and private stakeholders qualified basic, preclinical and translational research services covering paediatric medicines discovery and development and is providing expert assistance in the following paediatric clinical studies, based on the requests received by CVBF (Consortium for Biological and Pharmacological Evaluations).
Safety and Efficacy of MSC-EVs in the Prevention of BPD in Extremely Preterm Infants (EVENEW)
The phase 1/2 trial aims to evaluate the safety and efficacy of EXOB-001 consisting of extracellular vesicles derived from umbilical cord mesenchymal stromal cells in the prevention of bronchopulmonary dysplasia (BPD) in extremely premature neonates. The study population includes babies born between 23 and 28 (27 + 6 days) weeks of gestational age and body weight between 500g and 1,500 g. Thirty-six subjects will receive one or three administrations of the three doses of EXOB-001 via the endotracheal route in phase 1. In phase 2, two dosages based on the results of phase 1 will be selected and a total of 203 subjects will be randomised to receive either EXOB-001 or placebo (saline solution). Infants will be followed up to 2 years of corrected age (end of study).
Biogenera’s multi-center Phase 1 clinical trial of the anti-cancer drug BGA002 for aggressive pediatric cancers
BIOGENERA has officially signed an agreement with CVBF (Consortium for Biological and Pharmacological Evaluations) for the monitoring and execution of the Phase I clinical trial for the new drug BGA002, intended for the treatment of aggressive pediatric tumors associated with alterations in the MYCN tumor gene.

CVBF is part of various European research networks focused on knowledge transfer and scientific excellence, including EPTRI-AISBL which is dedicated to advancing the development of safe and effective new drugs for children.

The primary goal of the collaboration between BIOGENERA and CVBF is to initiate the Phase I clinical trial of the anti-tumor drug BGA002 in early 2025, leveraging the benefits of its Orphan Drug designation from both the European Medicines Agency and the U.S. FDA.

Gabapentin in Paediatric Chronic Pain study (GAPP Study)
The GAPP project, sponsored by CVBF, aims to evaluate the efficacy, pharmacokinetics, and safety of gabapentin in children aged 3 months to under 18 years who suffer from moderate to severe chronic neuropathic or mixed pain. Currently, Gabapentin is used off-label in these clinical conditions due to the lack of robust paediatric-specific evidence.

This represents a significant unmet need for adequate pharmacological treatments for neuropathic chronic and mixed pain in children, highlighting the necessity for therapies with a favourable benefit/risk profile (and age-appropriate formulation) to be considered as first-line options.