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INNODIA Master Protocol for the evaluation of investigational medicinal products in children, adolescents and adults with newly diagnosed type 1 diabetes.
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.
  • Dunger DB; Department of Paediatrics, University of Cambridge, Cambridge, UK.
  • Bruggraber SFA; Wellcome Trust-MRC Institute of Metabolic Sciences, University of Cambridge, Cambridge, UK.
  • Mander AP; Department of Paediatrics, University of Cambridge, Cambridge, UK.
  • Marcovecchio ML; Centre for Trials Research, Cardiff University, Cardiff, UK.
  • Tree T; NIHR Biomedical Research Centre, Guy's and St Thomas' NHS Foundation Trust and King's College London, London, UK.
  • Chmura PJ; Department of Paediatrics, University of Cambridge, Cambridge, UK. mlm45@medschl.cam.ac.uk.
  • Knip M; Centre for Trials Research, Cardiff University, Cardiff, UK.
  • Schulte AM; NIHR Biomedical Research Centre, Guy's and St Thomas' NHS Foundation Trust and King's College London, London, UK.
  • Mathieu C; Novo Nordisk Foundation Center for Protein Research, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark.
Trials ; 23(1): 414, 2022 May 18.
Article in English | MEDLINE | ID: covidwho-1862143
ABSTRACT

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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Full text: Available Collection: International databases Database: MEDLINE Main subject: Diabetes Mellitus, Type 1 / COVID-19 Type of study: Diagnostic study / Experimental Studies / Prognostic study / Randomized controlled trials Limits: Adolescent / Adult / Child / Humans Language: English Journal: Trials Journal subject: Medicine / Therapeutics Year: 2022 Document Type: Article Affiliation country: S13063-022-06259-z

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Full text: Available Collection: International databases Database: MEDLINE Main subject: Diabetes Mellitus, Type 1 / COVID-19 Type of study: Diagnostic study / Experimental Studies / Prognostic study / Randomized controlled trials Limits: Adolescent / Adult / Child / Humans Language: English Journal: Trials Journal subject: Medicine / Therapeutics Year: 2022 Document Type: Article Affiliation country: S13063-022-06259-z