Recruiting young pre-symptomatic children for a clinical trial in type 1 diabetes: Insights from the Fr1da insulin intervention study

Abstract

BackgroundAlthough detection of children at high risk of developing type 1 diabetes and diagnosis of early stages is possible, up to now there exists no approved therapy to delay or prevent type 1 diabetes. Thus it is vital to develop evidence-based interventions. For this a sufficient number of trial participants is crucial but difficult to obtain especially in asymptomatic children.AimIdentifying family characteristics that lead to or impede trial participation and analyze reasons stated by families for non-participation.MethodsParticipants for the Fr1da Insulin Intervention study are recruited from the Fr1da study, a population based screening for early stage type 1 diabetes in Bavaria. Families with eligible children were invited to enroll. We analyzed sex and age of the child, distance of the family to the study center in Munich and the existence of a first degree family member with type 1 as possible influential factors for study participation. We also analyzed reasons stated by families who declined study participation in a phone interview.ResultsOf 146 eligible children 77 (53%) were enrolled into the trial. None of the tested family characteristics differed significantly between the enrolling and the families not participating, but in general enrolling families lived closer to the study site than families not participating. This is also reflected in the reasons given by non-participating families. The most frequent reason stated were time restrictions. The second most frequent reason was the venous blood draw.ConclusionThe factors for non-participation identified in this project need be taken into account for the design of future trials in young children to ensure proper recruitment and thus to generate valid results for medical treatment of children. More research on the reason of participation and non-participation in clinical trials is needed.

Prevention of Type 1 Diabetes: Past Experiences and Future Opportunities

“…In particular, a population-wide screening program for T1D is not yet standard practice, because large-scale programs are incredibly expensive. However, there are some intervention trials, for instance, the Fr1da Insulin Intervention or the DIPP study, among high-risk for T1D participants enrolled from the general population [36,37]. Those individuals might more benefit from preventive therapies at the earliest stages since the destruction of beta-cell is not yet advanced.…”

“…This trial aims to test whether daily administration of up to 67.5 mg oral insulin for 12 months to children with at least two islet autoantibodies will have the potential to control the autoimmune responses by diverting the immune system to a protective rather than destructive response. The study is currently recruiting participants [36].…”

Typ-1-Diabetes bei Kindern früh erkennen und präventiv handeln

“…Kinder mit Stadium 1 wurden eingeladen, an einer randomisierten klinischen Studie (Fr1da Insulin Intervention; ClinicalTrials.gov: NCT02620072) teilzunehmen. Insgesamt 220 Kinder im Alter von 2-12 Jahren wurden in die Studie eingeschlossen [38]. Die Rekrutierung ist abgeschlossen, und Ergebnisse werden für 2024 erwartet.…”

Typ-1-Diabetes: Früherkennung und Ansätze zur Prävention

“…Das bestehende Interesse aller Familien an experimentellen Behandlungsmöglichkeiten zeigt sich auch bei Fr1da: Die Hälfte der Familien nahm für ihre Kinder im Stadium 1 das Angebot der Teilnahme an der Fr1da-Insulin-Interventionsstudie an [ 17 ].…”