NCT05449548

Lenalidomide in the Treatment of Mucosal Behçet's Syndrome

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 42 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The study is to evaluate the efficacy and safety of lenalidomide in the treatment of oral ulcers in adult patients with refractory mucosal Behcet's syndrome.

Detailed description

Behçet's Syndrome (BS) is a systemic vasculitis involving blood vessels of all sizes. It is characterised by recurrent oral and genital ulcers, skin lesions, musculoskeletal, ophthalmic, large vessel and intestinal involvements. Mucosal BS is the most common phenotype of BS, commonly treated with thalidomide and colchicine, yet some patients respond poorly or had limited use due to side effects. Lenalidomide, a second-generation derivative of thalidomide, has a role as an angiogenesis inhibitor, an antineoplastic agent and an immunomodulator. Its neurotoxicity and reproductive toxicity are significantly reduced, and the ability of TNF-alpha inhibition is significantly increased. Reports on lenalidomide for refractory mucosal BS have been mostly case reports and preliminary studies, clinical trials are lacking. This is a single-centre, prospective, open-label, single-arm study to evaluate the efficacy and safety of lenalidomide in the treatment of refractory mucosal BS; with the rate of complete remission of oral ulcers in subjects at 12 weeks as the primary endpoint; partial remission of oral and genital ulcers, non-response rate and BS disease activity as secondary endpoints; and adverse events and newly-developed BS-related symptoms as safety endpoints. This study aims to enroll adult patients with refractory mucosal BS with a stable dosage of low-dose glucocorticoids and/or other conventional immunomodulators. All subjects will be treated with lenalidomide 10mg/day with regular follow-up, those having adverse effects will be evaluated by investigators and adjusted to 5mg/day if necessary, with glucocorticoids and immunosuppressive agents adjusted as needed. Each subject will complete a 12-week treatment period, followed by 4 weeks of observation after cessation of lenalidomide.

Conditions

• Oral Ulcer

Interventions

Type	Name	Description
DRUG	Lenalidomide 10 mg	All subjects will be treated with lenalidomide 10mg/day with a regular follow-up of 12 weeks, followed by a 4-week observation after cessation of lenalidomide.

Timeline

• Start date: 2023-04-01
• Primary completion: 2025-10-30
• Completion: 2025-12-31
• First posted: 2022-07-08
• Last updated: 2025-09-05

Locations

1 site across 1 country: China