NCT06227221

Efficacy and Safety of Sorafenib in New-Onset Type 1 Diabetes Mellitus

A Phase 2, Randomized, Placebo Controlled Study Investigating the Efficacy and Safety of Sorafenib in New-Onset Type 1 Diabetes Mellitus

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Second Xiangya Hospital of Central South University · Academic / Other
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to investigate the therapeutic effect and safety of Sorafenib in T1DM patients.

Detailed description

Type 1 Diabetes Mellitus (T1DM) is caused by autoimmune destruction of beta cells in the islet. Insulin has been used as a routine therapy for T1DM to alleviate the hyperglycemic status, yet cannot effectively prevent the progressing destruction of beta cells or preserve its function. Our preliminary data identified sorafenib as an inhibitor of Th1 differentiation and an indirect inhibitor of JAK2 and found that sorafenib prevented and reversed T1DM in NOD mice by decreasing the accumulation of Th1 cells and the expression of inflammatory cytokines in pancreas. Sorafenib is already in clinical use for renal cell carcinoma, hepatocellular carcinoma and differentiated thyroid cancer. It is hypothesized that sorafenib treatment for 26 weeks will preserve beta cell function in adults with new-onset T1DM. The aim of this study is to investigate the potential of sorafenib on preserving beta cell functions in human T1DM.

Conditions

• Type 1 Diabetes

Interventions

Type	Name	Description
DRUG	Sorafenib	Subjects receive sorafenib 400mg once daily. Insulin therapy will be continued as a routine therapy.
DRUG	Placebo	Subjects receive placebo 400mg once daily. Insulin therapy will be continued as a routine therapy.

Timeline

• Start date: 2026-02-01
• Primary completion: 2028-10-01
• Completion: 2029-06-01
• First posted: 2024-01-26
• Last updated: 2026-02-12