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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.

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cam.issuedOnline2022-05-18
dc.contributor.authorDunger, David
dc.contributor.authorBruggraber, Sylvaine FA
dc.contributor.authorMander, Adrian P
dc.contributor.authorMarcovecchio, Loredana
dc.contributor.authorTree, Timothy
dc.contributor.authorChmura, Piotr Jaroslaw
dc.contributor.authorKnip, Mikael
dc.contributor.authorSchulte, Anke M
dc.contributor.authorMathieu, Chantal
dc.contributor.authorINNODIA consortium
dc.contributor.orcidDunger, David [0000-0002-2566-9304]
dc.contributor.orcidMarcovecchio, Loredana [0000-0002-4415-316X]
dc.date.accessioned2022-05-18T15:00:54Z
dc.date.available2022-05-18T15:00:54Z
dc.date.issued2022-05-18
dc.date.submitted2021-08-10
dc.date.updated2022-05-18T15:00:54Z
dc.description.abstractBACKGROUND: 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.
dc.identifier.doi10.17863/CAM.84689
dc.identifier.eissn1745-6215
dc.identifier.issn0012-186X
dc.identifier.others13063-022-06259-z
dc.identifier.other6259
dc.identifier.urihttps://www.repository.cam.ac.uk/handle/1810/337274
dc.languageen
dc.publisherSpringer Science and Business Media LLC
dc.subjectStudy Protocol
dc.subjectBeta-cell function
dc.subjectC-peptide
dc.subjectMaster protocol
dc.subjectPhase 2
dc.subjectPrevention
dc.subjectTrials
dc.subjectType 1 diabetes
dc.titleINNODIA Master Protocol for the evaluation of investigational medicinal products in children, adolescents and adults with newly diagnosed type 1 diabetes.
dc.typeArticle
dcterms.dateAccepted2022-03-30
prism.issueIdentifier1
prism.publicationNameTrials
prism.volume23
pubs.funder-project-idEuropean Commission Horizon 2020 (H2020) Societal Challenges (115797 INNODIA)
rioxxterms.licenseref.urihttp://creativecommons.org/licenses/by/4.0/
rioxxterms.versionVoR
rioxxterms.versionofrecord10.1186/s13063-022-06259-z

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