INNODIA Master Protocol for the evaluation of investigational medicinal products in children, adolescents and adults with newly diagnosed type 1 diabetes

Background The INNODIA consortium has established a pan-European infrastructure using validated centres to prospectively evaluate clinical data from individuals with newly diagnosed type 1 diabetes combined with centralised collection of clinical samples to determine rates of decline in beta-cell fu...

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Vydané v:	Current controlled trials in cardiovascular medicine Ročník 23; číslo 1; s. 414 - 9
Hlavní autori:	Dunger, David B., Bruggraber, Sylvaine F. A., Mander, Adrian P., Marcovecchio, M. Loredana, Tree, Timothy, Chmura, Piotr Jaroslaw, Knip, Mikael, Schulte, Anke M., Mathieu, Chantal
Médium:	Journal Article
Jazyk:	English
Vydavateľské údaje:	London BioMed Central 18.05.2022 BioMed Central Ltd Springer Nature B.V BMC
Predmet:	Adolescent Adult Beneficiaries Beta-cell function Biomarkers Biomedicine C-peptide Care and treatment Charitable foundations Child Complications and side effects COVID-19 Data integrity Data warehouses Decision making Demographic aspects Diabetes Diabetes Mellitus, Type 1 - diagnosis Diabetes Mellitus, Type 1 - drug therapy Drug development Health Sciences Humans Immunology Laboratories Life Sciences Master protocol Medicine Medicine & Public Health Medicine, Botanic Medicine, Herbal Natural history Patient outcomes Peptides Phase 2 Prevention SARS-CoV-2 Statistics for Life Sciences Study Protocol Teenagers Treatment Outcome Trials Type 1 diabetes France Europe Prevention Trials Type 1 diabetes Beta-cell function C-peptide Phase 2 Master protocol
ISSN:	1745-6215, 1745-6215
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Popis
Shrnutí:	Background The INNODIA consortium has established a pan-European infrastructure using validated centres to prospectively evaluate clinical data from individuals with newly diagnosed type 1 diabetes combined with centralised collection of clinical samples to determine rates of decline in beta-cell function and identify novel biomarkers, which could be used for future stratification of phase 2 clinical trials. Methods In this context, we have developed a Master Protocol, based on the “backbone” of the INNODIA natural history study, which we believe could improve the delivery of phase 2 studies exploring the use of single or combinations of Investigational Medicinal Products (IMPs), designed to prevent or reverse declines in beta-cell function in individuals with newly diagnosed type 1 diabetes. Although many IMPs have demonstrated potential efficacy in phase 2 studies, few subsequent phase 3 studies have confirmed these benefits. Currently, phase 2 drug development for this indication is limited by poor evaluation of drug dosage and lack of mechanistic data to understand variable responses to the IMPs. Identification of biomarkers which might permit more robust stratification of participants at baseline has been slow. Discussion The Master Protocol provides (1) standardised assessment of efficacy and safety, (2) comparable collection of mechanistic data, (3) the opportunity to include adaptive designs and the use of shared control groups in the evaluation of combination therapies, and (4) benefits of greater understanding of endpoint variation to ensure more robust sample size calculations and future baseline stratification using existing and novel biomarkers.
Bibliografia:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 14 content type line 23
ISSN:	1745-6215 1745-6215
DOI:	10.1186/s13063-022-06259-z