Publications

30

Dec, 2019

Standardising neonatal and pediatric anitibiotic clinical trial design and conduct: the PENTA-ID network review

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Authors: Folgori L, Lutsar I, Standing JF, et al.

Published in: BMJ Open. 2019; 9:e032592

Abstract: Antimicrobial development for children remains challenging due to multiple barriers to conducting randomised clinical trials (CTs). There is currently considerable heterogeneity in the design and conduct of paediatric antibiotic studies, hampering comparison and meta-analytic approaches. The board of the European networks for paediatric research at the European Medicines Agency (EMA), in collaboration with the Paediatric European Network for Treatments of AIDS-Infectious Diseases network (www.penta-id.org), recently developed a Working Group on paediatric antibiotic CT design, involving academic, regulatory and industry representatives. The evidence base for any specific criteria for the design and conduct of efficacy and safety antibiotic trials for children is very limited and will evolve over time as further studies are conducted. The suggestions being put forward here are based on the adult EMA guidance, adapted for neonates and children. In particular, this document provides suggested guidance on the general principles of harmonisation between regulatory and strategic trials, including (1) standardised key inclusion/exclusion criteria and widely applicable outcome measures for specific clinical infectious syndromes (CIS) to be used in CTs on efficacy of antibiotic in children; (2) key components of safety that should be reported in paediatric antibiotic CTs; (3) standardised sample sizes for safety studies. Summarising views from a range of key stakeholders, specific criteria for the design and conduct of efficacy and safety antibiotic trials in specific CIS for children have been suggested. The recommended criteria are intended to be applicable to both regulatory and clinical investigator-led strategic trials and could be the basis for harmonisation in the design and conduct of CTs on antibiotics in children. The next step is further discussion internationally with investigators, paediatric CTs networks and regulators.
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11

Nov, 2019

c4c Call for patients – get involved!

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c4c (conect4children) is a large collaborative European network that aims to facilitate the development of new drugs and other therapies for the entire paediatric population.

The voices of children, young people and their families are a pivotal part of the c4c project. It places patients at the centre and will assign them an active role in the development of the different clinical trials that are going to be carried out during this project.

For this reason, and in order to guarantee patients’ involvement in all the activities of the project, c4c has setup a database to gather information on patients, caregivers, patient organisations and/or YPAGs (Young Person’s advisory boards) of rare/paediatric diseases.

If you wish (or know someone who may wish) to be part of the c4c pool of expert patients, you can find out more by following the instructions here!

4

Apr, 2019

SMILE Clinical Trial: 100th Patient Enrolled

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The Penta-sponsored clinical trial SMILE has achieved a significant milestone by recruiting its 100th patient yesterday. The patient was enrolled at the Perinatal HIV Research Unit (PHRU) in Soweto, South Africa. This is a wonderful result achieved through hard work and cooperation among the sites, the CTUs and the sponsor.

SMILE (also known as PENTA 17) stands for “Strategy for Maintenance of HIV suppression with once daiLy Integrase inhibitor + darunavir/ritonavir in childrEn” and is a multicentre randomised study evaluating safety and antiviral effect of a once daily integrase inhibitor administered with darunavir/ritonavir compared to standard of care among HIV-1 infected, virologically suppressed paediatric participants. 300 children are due to be enrolled in the trial from about 30 sites spread across 14 countries in Europe, Asia, Africa and South America.

With one third of the patients enrolled and several new sites starting recruitment this month, the SMILE clinical trial is successfully reaching its goals and expected enrollment targets. We extend our congratulations and thanks to all teams involved for your ongoing collaboration!

The trial is supported by Janssen Pharmaceutica and ViiV Healthcare.

5

Apr, 2018

SMILE: enrollment has resumed

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We are pleased to announce that enrollment in the SMILE study has recommenced. 3 new participants have recently been recruited in the clinical trial: 2 in Porto, Portugal and 1 in Zurich, Switzerland.

SMILE is a multicentre randomised study evaluating safety and antiviral effect of a once daily integrase inhibitor administered with Darunavir/Ritonavir compared to standard of care among HIV-1 infected, virologically suppressed paediatric participants.

PENTA is sponsor of this study, with 300 children due to be enrolled from about 30 sites from 10 countries.

2

Feb, 2018

PREPARE response to seasonal influenza in Europe – a press release from the PREPARE project

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The EU funded project PREPARE has released a communication brief updating stakeholders on the project activities related to this season’s influenza activity.

press release has also been published with more information about the exciting and innovative PREPARE’s ALIC4E clinical trial. This is the only study that is currently randomizing patients to receive antiviral treatment or not. This study will answer the question about whether Oseltamivir is clinically and/or cost effective or not when treating primary care patients with influenza-like illness.

PENTA is partner in PREPARE (Platform for European Preparedness Against (Re-)emerging Epidemics) project, an EU funded network for harmonised large-scale clinical research studies on infectious diseases, prepared to rapidly respond to any severe ID outbreak, providing real-time evidence for clinical management of patients and for informing public health responses.

To know more about the project, visit the PREPARE website.