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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

, ; Ziegler, Anette Gabriele; Achenbach, Peter; Berner, Reinhard; Casteels, Kristina; Danne, Thomas; Gündert, Melanie; Hasford, Joerg; Hoffmann, Verena Sophia and Kordonouri, Olga, et al. (2019) In BMJ Open 9(6).
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... (More)

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.

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published
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keywords
clinical trials, general diabetes, paediatric endocrinology
in
BMJ Open
volume
9
issue
6
pages
9 pages
publisher
British Medical Journal Publishing Group
external identifiers
  • scopus:85068461302
ISSN
2044-6055
DOI
10.1136/bmjopen-2018-028578
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English
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yes
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c01c483d-8f47-46cf-8e59-7a4016ffc23d
date added to LUP
2019-07-17 11:51:02
date last changed
2019-08-06 03:24:48
@article{c01c483d-8f47-46cf-8e59-7a4016ffc23d,
  abstract     = {<p>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 &gt;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.</p>},
  articleno    = {e028578},
  author       = {,  and Ziegler, Anette Gabriele and Achenbach, Peter and Berner, Reinhard and Casteels, Kristina and Danne, Thomas and Gündert, Melanie and Hasford, Joerg and Hoffmann, Verena Sophia and Kordonouri, Olga and Lange, Karin and Elding Larsson, Helena and Lundgren, Markus and Snape, Matthew D. and Szypowska, Agnieszka and Todd, John A. and Bonifacio, Ezio},
  issn         = {2044-6055},
  keyword      = {clinical trials,general diabetes,paediatric endocrinology},
  language     = {eng},
  month        = {06},
  number       = {6},
  pages        = {9},
  publisher    = {British Medical Journal Publishing Group},
  series       = {BMJ Open},
  title        = {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},
  url          = {http://dx.doi.org/10.1136/bmjopen-2018-028578},
  volume       = {9},
  year         = {2019},
}