Table 1.
Primary or secondary prevention trials prior to clinical diagnosis of diabetes
| Study (ref.) | Drug/phase | Sponsor/contact | Age | Eligibility | Dosing | Placebo | Follow-up duration/primary end point | Status/target size |
|---|---|---|---|---|---|---|---|---|
| TRIGR (23) | Cow's milk hydrolyzate/phase III | NIH, NICHD, et al./www.trigr.org | 0–7 days | FDRs, high-risk HLA | Supplementation of breast-feeding up to the age of 8 months | Yes | 10 years/type 1 diabetes | Enrollment closed/n = 2,160 |
| BABY DIET (24) | Gluten-free diet/phase II pilot | German Research Foundation/anziegler@lrz.uni-munchen.de | <3 months | Relatives, high-risk HLA DR, DQ | Gluten-free diet until age 12 months | No | 3 years/islet autoantibodies | Enrollment closed/n = 50 |
| TrialNet NIP (27) | DHA/phase II pilot | NIH, NIDDK/www.diabetestrialnet.org | >24 weeks gestation/newborn | Relatives, HLA-DR3 or DR4 | Oral DHA once daily | Yes | 2 years/20% higher plasma levels of DHA | Enrollment closed/n = 119 |
| Vitamin D (31,82) | Vitamin D3/phase I pilot | Canadian Diabetes Association/dcatte@mich.ca | 0–4 weeks | High-risk HLA DR, DQ | Oral vitamin D 2,000 IU once daily | No | 1 year/25(OH) vitamin D levels, serum/urine Ca, islet autoantibodies | Enrolling/n = 20 |
| TrialNet Oral Insulin (33) | Human insulin/phase III | NIH, NIDDK/www.diabetestrialnet.org | 1–45 years | Relatives, 2+ islet antibodies including to insulin | Oral insulin 7.5 mg once daily | Yes | 7–8 years/type 1 diabetes | Enrolling/n ≈ 400 |
| INIT II (34) | Human insulin/phase II | Melbourne Health/harrison@wehi.edu.au | 4–30 years | Relatives, 2+ islet antibodies HLA not DR2, DQ6 | Intranasal 1.6 mg and 16 mg/day | Yes | 5 years/type 1 diabetes | Enrolling/n = 262 |
| Pre-POINT (26) | Human insulin/phase I/II | JDRF/prevent.diabetes@crt-dresden.de | 1.5–7 years | FDRs/>50% risk of type 1 diabetes | Insulin daily for the first 10 days, after that twice a week. Increasing dose: oral 2.5–67.5 mg/day, intranasal 0.28–7.5 mg/day* | Yes | 3–18 months/islet autoantibodies | Enrolling/n = 40 |
| FINDIA | Insulin-free whey-based formula/phase I/II | National Public Health Institute, Helsinki, Finland/outi.vaarala@ktl.fi | Infants | General population, high-risk HLA DQ | Yes | 2 years/islet autoantibodies, type 1 diabetes | Enrollment closed/n = 982 |
*Staggered enrollment. DHA, docosahexaenoic acid; FDR, first-degree relative; JDRF, Juvenile Diabetes Research Foundation; NICHD, National Institute of Child Health and Human Development; NIDDK, National Institute of Diabetes and Digestive and Kidney Diseases; NIH, National Institutes of Health; NIP, Nutritional Intervention to Prevent type 1 diabetes.