Oral insulin therapy for primary prevention of type 1 diabetes in infants with high genetic risk: The GPPAD-POInT (global platform for the prevention of autoimmune diabetes primary oral insulin trial) study protocol

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Abstract

Introduction The POInT study, an investigator initiated, randomised, placebo-controlled, double-blind, multicentre primary prevention trial is conducted to determine whether daily administration of oral insulin, from age 4.0 months to 7.0 months until age 36.0 months to children with elevated genetic risk for type 1 diabetes, reduces the incidence of beta-cell autoantibodies and diabetes. Methods and analysis Infants aged 4.0 to 7.0 months from Germany, Poland, Belgium, UK and Sweden are eligible if they have a >10.0% expected risk for developing multiple beta-cell autoantibodies as determined by genetic risk score or family history and human leucocyte antigen genotype. Infants are randomised 1:1 to daily oral insulin (7.5 mg for 2 months, 22.5 mg for 2 months, 67.5 mg until age 36.0 months) or placebo, and followed for a maximum of 7 years. Treatment and follow-up is stopped if a child develops diabetes. The primary outcome is the development of persistent confirmed multiple beta-cell autoantibodies or diabetes. Other outcomes are: (1) Any persistent confirmed beta-cell autoantibody (glutamic acid decarboxylase (GADA), IA-2A, autoantibodies to insulin (IAA) and zinc transporter 8 or tetraspanin 7), or diabetes, (2) Persistent confirmed IAA, (3) Persistent confirmed GADA and (4) Abnormal glucose tolerance or diabetes. Ethics and dissemination The study is approved by the ethical committees of all participating clinical sites. The results will be disseminated through peer-reviewed journals and conference presentations and will be openly shared after completion of the trial. Trial registration number NCT03364868.

Detaljer

Författare
  • the GPPAD Study Group
  • Anette Gabriele Ziegler
  • Peter Achenbach
  • Reinhard Berner
  • Kristina Casteels
  • Thomas Danne
  • Melanie Gündert
  • Joerg Hasford
  • Verena Sophia Hoffmann
  • Olga Kordonouri
  • Karin Lange
  • Helena Elding Larsson
  • Markus Lundgren
  • Matthew D. Snape
  • Agnieszka Szypowska
  • John A. Todd
  • Ezio Bonifacio
Enheter & grupper
Externa organisationer
  • Helmholtz Zentrum München
  • Klinikum rechts der Isar
  • University Hospitals Leuven
  • Catholic University of Leuven
  • Ludwig-Maximilian University of Munich
  • Hannover Medical School
  • Skåne University Hospital
  • University of Oxford
  • Medical University of Warsaw
  • Dresden University of Technology
  • Technical University of Munich
  • University Hospital Carl Gustav Carus
  • Children's Hospital Auf der Bult
  • Oxford University Hospital
Forskningsområden

Ämnesklassifikation (UKÄ) – OBLIGATORISK

  • Endokrinologi och diabetes
  • Pediatrik

Nyckelord

Originalspråkengelska
Artikelnummere028578
Antal sidor9
TidskriftBMJ Open
Volym9
Utgivningsnummer6
StatusPublished - 2019 jun 28
PublikationskategoriForskning
Peer review utfördJa