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type1.science
Phase 2CompletedNCT01781975

Imatinib in recent-onset type 1 diabetes

What this study tests

Multicenter phase-2 trial of the oral tyrosine kinase inhibitor imatinib in adults within 100 days of diagnosis. It met the 12-month C-peptide endpoint, but the effect was not sustained at 24 months.

Editorial review: .

Registry checked: 2026-09-17. Registry’s own update: 2020-02-11.

Most recent recorded citation date: 2021-07-05. Only explicit date metadata is included; an undated citation may be newer. This does not mean every claim was reviewed on that date.

Trial status, labels and access can change between reviews. How we review the evidence · How to read the evidence

Full sources ↓Full discussion ↓Report an issue →

Evidence at a glance

Who can enter the study?
Adults aged 18-45, within 100 days of T1D diagnosis, autoantibody-positive, with stimulated C-peptide above 0.2 nmol/L. Registered eligibility ↗Enrollment criteria describe who may enter. They do not establish who was analyzed or the denominator for a reported result.
Reported benefit and results
Of 67 randomized adults (45 imatinib, 22 placebo), 64 contributed to the primary 12-month analysis. The adjusted C-peptide AUC difference was 0.095 (90% CI −0.003 to 0.191; one-tailed p=0.048), meeting the trial’s prespecified test but not sustained at 24 months after randomization. Treatment lasted 26 weeks. Grade 2 or worse adverse events occurred in 71% versus 59%; 38% of imatinib recipients needed a temporary dosing change and 13% permanently stopped treatment because of adverse events.Read the result sources and their limitations →
Important harms
Read the reported results, safety discussion and original sources. A separate harms summary is not available; this does not establish safety.
Research access and approval
Study regions: United States, Australia. A trial listing does not establish product approval or current recruitment at a particular site. Check the study’s current entry requirements.
What remains uncertain?
Read the full discussion and original sources for study limitations. Planned endpoints and completion dates are not results.

Research status alone does not establish approval, clinical benefit or local availability.

Australian sites in the registry

  • Walter and Eliza Hall Institute of Medical Research, Melbourne, Victoria — Site status not reported

Overall recruitment does not imply availability at every site. Confirm eligibility and local recruitment with the study team.

Primary endpoints

  • 2-hour mixed-meal stimulated C-peptide AUC at 12 months

The full picture

Imatinib is a useful "positive but not enough" trial: C-peptide moved at 12 months, but the signal faded by 24 months and tolerability limits enthusiasm for long-term prevention use.

The endpoint used a one-tailed test, with a 90% confidence interval that crossed zero. The modest 12-month signal, lack of persistence at month 24 and treatment interruptions all matter when interpreting efficacy. Neither time point is measured from the end of the 26-week course. Primary trial report.

Sources

  1. [1]
  2. [2]