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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Veröffentlicht in:Current controlled trials in cardiovascular medicine Jg. 23; H. 1; S. 414 - 9
Hauptverfasser: 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
Format: Journal Article
Sprache:Englisch
Veröffentlicht: London BioMed Central 18.05.2022
BioMed Central Ltd
Springer Nature B.V
BMC
Schlagworte:
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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Zusammenfassung: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.
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ISSN:1745-6215
1745-6215
DOI:10.1186/s13063-022-06259-z