Lenalidomide in the Treatment of Mucosal Behçet's Syndrome
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Enrollment
- 42
- Locations
- 2
- Primary Endpoint
- The complete remission rate of oral ulcers in subjects after 12 weeks of treatment
Study Overview
Brief 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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 65 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Patients that can understand and voluntarily sign an informed consent document prior to the study;
- •Male and female subjects ≥ 18 years and ≤ 65 years of age at the time of signing the informed consent document.
- •Fulfilling the ICBD (International Conference on Behcet's Disease) criteria(2013);
- •Presented with active mucosal lesions: Subjects must have at least 1 oral ulcer within 4 weeks after the screening visit and at least 2 oral ulcers on the day of enrollment; subjects may be with or without genital ulcers and (or) skin lesions.
- •Refractory mucosal lesions: Subjects must experience at least 2 relapses of oral ulcers during 3 consecutive months of conventional treatment with corticosteroids and(or) immunosuppressants.
- •Without major organ involvement, including active gastrointestinal, ocular, nervous system, and major vessel involvement; previous major organ involvement is allowed if it occurred at least 1 year prior to the screening visit and is not active at the time of enrollment.; subjects with arthritis are permitted.
Exclusion Criteria
- •The presence of any of the following will exclude a subject from the study enrollment.
- •Exclusion Criteria:
- •Pregnant women or breastfeeding mothers, Male and female patients with recent fertility requirements.
- •Skin and mucosal lesions should exclude erythema multiforme, syphilis, Sweet disease, Stevens-Johnson syndrome, acne vulgaris, herpes simplex infection, periodic granulocytopenia, and acquired immunodeficiency.
- •Subjects with Behçet's syndrome-related active major organ involvement that requires aggressive immunosuppressive therapy, including active gastrointestinal, ocular, nervous system, and major vessel involvement.
- •Severe Concomitant disease: including heart failure(≥level Ⅲ, NYHA), respiratory failure, renal insufficiency (Serum creatinine ≥ 1.5 mg/dL ), hepatic insufficiency(Aspartate transaminase (AST) and alanine transaminase (ALT) ≥ 2 X ULN.), myelosuppression(WBC<3.0×109/L or N<1.5×109/L, HGB≤85g/L, PLT<100×109/L), peripheral neuropathy.
- •Acute severe infections such as sepsis and cellulitis, active hepatitis B or C virus infection, active tuberculosis, and history of a positive test for, or any clinical suspicion of, human immunodeficiency virus (HIV).
- •Patients with malignancy, or any history of malignancy within the 5 years prior to the screening phase, risk factors for myocardial infarction (including a history of thrombosis), or hypercoagulability.
- •History of use of lenalidomide or thalidomide within 1 month before enrollment.
- •Patients with allergies or contraindications to lenalidomide or thalidomide.
- •Having received concomitant immune-modulating therapy or small molecule drugs. At least 5 terminal half-lives for all biologics, including, but not limited to, those listed below; within:
- •Ten days prior to the day of enrollment for tofacitinib and baricitinib Four weeks prior to the day of enrollment for etanercept Eight weeks prior to the day of enrollment for infliximab Ten weeks prior to the day of enrollment for adalimumab, golimumab, certolizumab, abatacept, and tocilizumab Six months prior to the day of enrollment for secukinumab
Arms & Interventions
Intervention with lenalidomide
All subjects will be treated with lenalidomide 10mg/day.
Intervention: Lenalidomide 10 mg (Drug)
Outcomes
Primary Outcomes
The complete remission rate of oral ulcers in subjects after 12 weeks of treatment
Time Frame: 12 weeks
A complete remission at week 12 was defined as participants who were oral ulcer-free at week 12.
Secondary Outcomes
- Percentage of participants who had no response of genital ulcer at Week 12(12 weeks)
- Percentage of participants who had no response of oral ulcer at Week 12(12 weeks)
- Percentage of participants who had a complete response of genital ulcer at Week 12(12 weeks)
- Percentage of participants who had an partial response of genital ulcer at Week 12(12 weeks)
- Change from baseline on oral ulcer pain as measured by visual analogue scale (VAS) at week 12(Baseline to week 12)
- Change from baseline in disease activity as measured by Behçet's Disease Current Activity Form (BDCAF)(Baseline to week 12)
- Change from baseline in Behçet's Disease Quality of Life (BD Qol) Scores at week 12(Baseline to week 12)
- Percentage of participants who had an partial response of oral ulcer at Week 12(12 weeks)
- Change from baseline on genital ulcer pain as measured by visual analogue scale (VAS) at week 12(Baseline to week 12)
- Change from baseline in disease activity as measured by Behçet's Syndrome Activity Score (BSAS) at Week 12(Baseline to week 12)
- Change From baseline in Short Form-36 Health Status Questionnaire(SF-36)(Baseline to week 12)
- Corticosteroid-tapering effects.(Baseline to week 12)