The GAPP Study is a multicentre, non-profit Phase III clinical trial sponsored by TEDDY Network to evaluate the efficacy, safety, and pharmacokinetics of a paediatric liquid formulation of gabapentin for chronic moderate to severe neuropathic, nociplastic, or mixed pain in children and adolescents.
As part of the TEDDY General Assembly and Scientific Meeting 2026, held in Bari from 18 – 20 September, the GAPP Study Round Table brought together members of the Study Steering Committee (SSC), Data Safety Monitoring Board (DSMB) and Patient Advisory Board (PAB) for an open, multidisciplinary discussion on three key areas of paediatric clinical research: methodology, participant safety and patient perspectives.
The session provided a shared forum to discuss how these complementary perspectives can contribute to the design and practical implementation of the GAPP clinical study, which investigates innovative approaches to evidence generation in a small and heterogeneous paediatric population affected by chronic neuropathic, nociplastic or mixed pain.
The round table brought together experts involved in different aspects of the GAPP Study and its governance bodies, including:
- Study steering Committee (SSC) Members, including Anna Szumowska (IPCZD Warsaw), Oscar Della Pasqua, a member of the TEDDY Board of Directors, legal representative of TEDDY Stichting and Chair in Clinical Pharmacology & Therapeutics at University College London, UK, Eleonora Di Campi (Clinical Project manager, TEDDY Network), Giorgio Reggiardo, Scientific Advisor of GAPP, Chair of the TEDDY Working Group on Paediatric Research Methodologies and a member of the TEDDY Board of Directors.
- Data Safety Monitoring Board (DSMB) Members, including Paola Baiardi (ICS Maugeri), and Evelyne Jacqz-Aigrain (Université Paris Cité).
- Patient Advisory Board (PAB) Members, including Aaisha-Seddika Muminah (TEDDY KIDS Network – Moorfields eye-YPAG, United Kingdom), Thuraya Layla Pearl Derby (TEDDY KIDS Network – Moorfields eye-YPAG, United Kingdom), Eleonora Passeri (Rare Special Powers (RSP APS) and TEDDY KIDS NETWORK – KIDS Umbria), Francesco Meloni (Italian Association of Bladder Exstrophy and Epispadias ODV), Aleksander Wiśniewski (Children’s Memorial Health Institute, Warsaw, Poland and TEDDY KIDS NETWORK – POLPEDNET KIDS Warsaw), Alessandra Monacelli (KIDS Bari)
Moderated by Oscar Della Pasqua and Eleonora Di Campi, the session was structured as an open and multidisciplinary exchange, organised around three interconnected areas: methodological innovation, participant safety and the patient perspective.
The methodological discussion focused on the GAPP Study design and the challenges of generating robust evidence in a small and heterogeneous paediatric population. Particular attention was given to the combination of a single-arm design, Bayesian statistical approach and adaptive methodology, together with an individualised dose-optimisation strategy. Participants discussed the challenges of interpreting evidence across heterogeneous paediatric populations, including the role of extrapolation, interim analyses and the identification of appropriate treatment signals and doses within small populations.
The safety discussion focused on the role of independent oversight and on how safety considerations can be integrated into the implementation of an innovative study design. The DSMB, together with the other governance bodies, contributed to the discussion on monitoring, interpretation of emerging evidence and the practical aspects of conducting research in a paediatric population.
The patient perspective discussion explored how the experience and priorities of children, young people, advocates and families can inform the practical implementation of the study. The members of the GAPP PAB, including representatives from the TEDDY KIDS Network, shared perspectives on participating in clinical research, including blood sampling, communication of risks and benefits, interactions with healthcare professionals and the hospital environment. They emphasised the importance of clear, age-appropriate communication, ensuring that children understand why procedures are carried out and how their participation contributes to research.
By bringing these perspectives into a shared discussion, the Round Table highlighted how methodological choices, safety considerations and the experiences of children and families are closely interconnected in paediatric clinical research. The session provided an opportunity to explore practical challenges in implementing the GAPP Study while fostering dialogue between researchers, clinicians, statisticians, patients, advocates and families.
The GAPP Round Table ultimately demonstrated the value of an open, multidisciplinary approach in which scientific and methodological innovation, participant safety and patient perspectives can inform one another throughout the development and implementation of a paediatric clinical study.