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. ORCID: https://orcid.org/0000-0002-0742-9040, Marcovecchio, M. Loredana, Tree, Timothy, Chmura, Piotr Jaroslaw, Knip, Mikael, Schulte, Anke M., Mathieu, Chantal, Mathieu, C., Gillard, P., Casteels, K., Overbergh, L., Dunger, D., Wallace, C., Evans, M., Thankamony, A., Hendriks, E., Bruggraber, S., Peakman, M., Tree, T., Morgan, N., Richardson, S., Todd, J., Wicker, L., Mander, A. ORCID: https://orcid.org/0000-0002-0742-9040, Dayan, C., Alhadj Ali, M., Pieber, T., Eizirik, D., Cnop, M., Brunak, S., Pociot, F., Johannesen, J., Rossing, P., Legido Quigley, C., Mallone, R., Scharfmann, R., Boitard, C., Knip, M., Otonkoski, T., Veijola, R., Lahesmaa, R., Oresic, M., Toppari, J., Danne, T., Ziegler, A. G., Achenbach, P., Rodriguez-Calvo, T., Solimena, M., Bonifacio, E., Speier, S., Holl, R., Dotta, F., Chiarelli, F., Marchetti, P., Bosi, E., Cianfarani, S., Ciampalini, P., de Beaufort, C., Dahl-Jørgensen, K., Skrivarhaug, T., Joner, G., Krogvold, L., Jarosz-Chobot, P., Battelino, T., Thorens, B., Gotthardt, M., Roep, B., Nikolic, T., Zaldumbide, A., Lernmark, A., Lundgren, M., Costecalde, G., Strube, T., Schulte, A., Nitsche, A., von Herrath, M., Wesley, J., Napolitano-Rosen, A., Thomas, M., Schloot, N., Goldfine, A., Waldron-Lynch, F., Kompa, J., Vedala, A., Hartmann, N., Nicolas, G., van Rampelbergh, J., Bovy, N., Dutta, S., Soderberg, J., Ahmed, S., Martin, F., Agiostratidou, G., Koralova, A., Willemsen, R., Smith, A., Anand, B., Puthi, V., Zac-Varghese, S., Datta, V., Dias, R., Sundaram, P., Vaidya, B., Patterson, C., Owen, K., Piel, B., Heller, S., Randell, T., Gazis, T., Bismuth Reismen, E., Carel, J-C, Riveline, J-P, Gautier, J-F, Andreelli, F., Travert, F., Cosson, E., Penfornis, A., Petit, C., Feve, B., Lucidarme, N., Cosson, E., Beressi, J-P, Ajzenman, C., Radu, A., Greteau-Hamoumou, S., Bibal, C., Meissner, T., Heidtmann, B., Toni, S., Rami-Merhar, B., Eeckhout, B., Peene, B., Vantongerloo, N., Maes, T., Gommers, L., Marcovecchio, M.L., Vela, J. and Latres, E. 2022. INNODIA Master Protocol for the evaluation of investigational medicinal products in children, adolescents and adults with newly diagnosed type 1 diabetes. Trials 23 (1) , 414. 10.1186/s13063-022-06259-z

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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.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Medicine Centre for Trials Research (CNTRR)
Additional Information:	This article is licensed under a Creative Commons Attribution 4.0 International License
Publisher:	BioMed Central
ISSN:	1745-6215
Date of First Compliant Deposit:	9 August 2022
Date of Acceptance:	30 March 2022
Last Modified:	05 May 2023 03:09
URI:	https://orca.cardiff.ac.uk/id/eprint/151830

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