Oral UlcerNCT05449548
Lenalidomide in the Treatment of Mucosal Behçet's Syndrome
A study testing Lenalidomide 10 mg in people with Oral Ulcer, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Oral Ulcer
- What is being tested?
- Lenalidomide 10 mg
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteChina
- Study dates
- 1 Apr 2023to 31 Dec 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Wenjie Zheng, M.D. contact
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Jinjing Liu
Wenjie Zheng, M.D.
About this study
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.
Read the 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
- Being tested
- Lenalidomide 10 mg
- Medical subject headings
- Oral Ulcer
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lenalidomide 10 mgDrug
Also called Lenalidomide
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.
| Study group | Role | What they receive |
|---|---|---|
| Intervention with lenalidomide | Experimental | All subjects will be treated with lenalidomide 10mg/day. Receives: Drug: Lenalidomide 10 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
The complete remission rate of oral ulcers in subjects after 12 weeks of treatment
A complete remission at week 12 was defined as participants who were oral ulcer-free at week 12.
Measured: 12 weeks
Secondary outcomes (12)
Percentage of participants who had an partial response of oral ulcer at Week 12
A partial response at week 12 was defined as participants with a 50% or more reduction in the number of oral ulcers at week 12
Measured: 12 weeks
Percentage of participants who had no response of oral ulcer at Week 12
No response at week 12 was defined as all other participants at week 12
Measured: 12 weeks
Percentage of participants who had a complete response of genital ulcer at Week 12
A complete remission at week 12 was defined as participants who were genital ulcer-free at week 12.
Measured: 12 weeks
Percentage of participants who had an partial response of genital ulcer at Week 12
A partial response at week 12 was defined as participants with a 50% or more reduction in the number of genital ulcers at week 12
Measured: 12 weeks
Percentage of participants who had no response of genital ulcer at Week 12
No response at week 12 was defined as all other participants at week 12
Measured: 12 weeks
Change from baseline on oral ulcer pain as measured by visual analogue scale (VAS) at week 12
Pain of oral ulcers was measured using a 100 mm VAS scale. The participant was asked to draw a single line perpendicular to the VAS line at the point that represented the severity of their pain during the previous week, with 0 mm (the left-hand end of the scale) representing no pain and 100 mm (the right-hand end of the scale) representing the worst pain imaginable. The distance of the perpendicular line from the left-hand end of the scale was recorded.
A negative change from baseline indicates improvement.
Measured: Baseline to week 12
Change from baseline on genital ulcer pain as measured by visual analogue scale (VAS) at week 12
Pain of genital ulcers was measured using a 100 mm VAS scale. The participant was asked to draw a single line perpendicular to the VAS line at the point that represented the severity of their pain during the previous week, with 0 mm (the left-hand end of the scale) representing no pain and 100 mm (the right-hand end of the scale) representing the worst pain imaginable. The distance of the perpendicular line from the left-hand end of the scale was recorded.
A negative change from baseline indicates improvement.
Measured: Baseline to week 12
Change from baseline in disease activity as measured by Behçet's Disease Current Activity Form (BDCAF)
The Behçet's Disease Current Activity Form (BDCAF) consists of 3 component scores: the Behçet's Disease Current Activity Index (BDCAI) score, the Patient's Perception of Disease Activity, and the Clinician's Overall Perception of Disease Activity.
The BDCAI consists of 12 questions regarding disease manifestations over the previous 4 weeks, The score is the sum score of 12 items and ranges from 0 to 12. A higher score indicates a higher level of disease activity, and a negative change from baseline indicates improvement.
The Patient's Perception of Disease Activity was assessed on a scale from 1 to 7, and the Clinician's Overall Perception of Disease Activity was assessed on a scale from 1 to 7. A higher score indicates a higher level of disease activity and a negative change from baseline indicates improvement.
Measured: Baseline to week 12
Change from baseline in disease activity as measured by Behçet's Syndrome Activity Score (BSAS) at Week 12
The Behçet's Syndrome Activity Score (BSAS) contains 10 questions that assess the number of new oral and genital ulcers and skin lesions, GI, CNS, vascular, and ocular involvement, and the participant's current level of discomfort. The Behçet's Syndrome Activity Score ranges from 0 to 100, with a higher score indicating a higher level of disease activity. A negative change from baseline indicates improvement.
Measured: Baseline to week 12
Change from baseline in Behçet's Disease Quality of Life (BD Qol) Scores at week 12
The Behçet's Disease Quality of Life questionnaire was developed to measure the influence of BD on a particpant's life. It consists of 30 self-completed itemized questions that measure disease- related restrictions on the participant's activities and their emotional response to these restrictions. The total score is the sum of all 30 items (each yes scores 1 and each no scores 0), with 0 representing no influence of Behçet's disease on a participant's quality of life and 30 representing the most severe influence. A negative change from baseline indicates improvement.
Measured: Baseline to week 12
Change From baseline in Short Form-36 Health Status Questionnaire(SF-36)
Short Form-36 Health Status Questionnaire(SF-36) is a 36-item, patient-reported survey of patient health. The SF-36 is a measure of health status. The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability i.e., a score of zero is equivalent to maximum disability and a score of 100 is equivalent to no disability.
Measured: Baseline to week 12
Corticosteroid-tapering effects.
The dosage of glucocorticoids will be tapered tailored to the disease activity of each participant, and the change of dosage from baseline will be recorded.
Measured: Baseline to week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2022
- Study start1 Apr 2023
- Primary completion30 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Oral UlcerStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Wenjie Zheng, M.D., Principal investigator, Department of Rheumatology, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05449548
- Sponsor's protocol ID
- lenalidomide in BS
- Status last verified
- Jun 2026
- First submitted
- 5 Jul 2022
- Last updated
- 22 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Lenalidomide in the Treatment of Mucosal Behçet's Syndrome
- ClinicalTrials.gov ID
- NCT05449548
- Acronym / sponsor ID
- lenalidomide in BS
- Conditions
- Oral Ulcer
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 1 Apr 2023 (actual)
- Primary completion
- 30 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 8 Jul 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intervention with lenalidomide | Experimental | Drug: Lenalidomide 10 mgAll subjects will be treated with lenalidomide 10mg/day. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (10 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lenalidomide 10 mgAlso: LenalidomideAll 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. | Drug | Intervention with lenalidomide |
Primary outcome
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.
12 secondary outcomes
Percentage of participants who had an partial response of oral ulcer at Week 12
Time frame: 12 weeks
Percentage of participants who had no response of oral ulcer at Week 12
Time frame: 12 weeks
Percentage of participants who had a complete response of genital ulcer at Week 12
Time frame: 12 weeks
Percentage of participants who had an partial response of genital ulcer at Week 12
Time frame: 12 weeks
Percentage of participants who had no response of genital ulcer at Week 12
Time frame: 12 weeks
Change from baseline on oral ulcer pain as measured by visual analogue scale (VAS) at week 12
Time frame: Baseline to week 12
Change from baseline on genital ulcer pain as measured by visual analogue scale (VAS) at week 12
Time frame: Baseline to week 12
Change from baseline in disease activity as measured by Behçet's Disease Current Activity Form (BDCAF)
Time frame: Baseline to week 12
Change from baseline in disease activity as measured by Behçet's Syndrome Activity Score (BSAS) at Week 12
Time frame: Baseline to week 12
Change from baseline in Behçet's Disease Quality of Life (BD Qol) Scores at week 12
Time frame: Baseline to week 12
Change From baseline in Short Form-36 Health Status Questionnaire(SF-36)
Time frame: Baseline to week 12
Corticosteroid-tapering effects.
Time frame: Baseline to week 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Wenjie Zheng, M.D. contact
Wenjie Zheng, M.D. principal investigator
Department of Rheumatology, Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Lenalidomide 10 mg in people with Oral Ulcer, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jinjing Liu, M.D. (8617810391494, questionmark003@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The complete remission rate of oral ulcers in subjects after 12 weeks of treatment (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2026.