Ongoing Clinical Trials for Behçet’s Syndrome
Behçet’s syndrome is a rare chronic condition that causes inflammation of blood vessels throughout the body, leading to symptoms such as mouth sores, eye inflammation, skin rashes, and genital sores. Currently, there are 5 ongoing clinical trials investigating new treatment options for patients with this condition. These trials are testing medications such as apremilast, filgotinib, tocilizumab, adalimumab, and rivaroxaban, focusing on managing various manifestations of the disease including oral ulcers, severe uveitis, and deep vein thrombosis. (Also known as: Behçet’s disease)
Clinical trial locations
- France
- Greece
- Italy
- Netherlands
- Spain
Study on Apremilast for Treating Oral Ulcers in Children with Behçet’s Disease
This trial is evaluating the effectiveness and safety of apremilast in treating oral ulcers in children aged 2 to less than 18 years with Behçet’s disease. The study focuses on a particularly challenging symptom of the condition: recurrent painful mouth sores that can significantly impact quality of life.
Who can participate: Children between 2 and 18 years old who have been diagnosed with Behçet’s disease and currently have at least 2 oral ulcers are eligible. Participants must have experienced oral ulcers at least 3 times in the past year and have tried at least one previous treatment such as topical corticosteroids or other systemic medications that is not a biologic therapy.
Who cannot participate: Children outside the specified age range, those without active oral ulcers, or those unable to follow study procedures are excluded. Other exclusion criteria include recent infections, pregnancy or breastfeeding in adolescents, allergic reactions to the study medication, and recent participation in another clinical trial.
Study focus: The main goal is to determine whether apremilast can reduce the number of oral ulcers and improve pain levels over a 12-week treatment period. The study uses a double-blind design, meaning neither participants nor researchers initially know who receives the actual medication or placebo. After this phase, all participants receive apremilast in an open-label extension lasting up to 52 weeks. Researchers will monitor disease activity, side effects, and overall growth and development throughout the study.
Investigational drug: Apremilast is a phosphodiesterase 4 inhibitor taken orally as a film-coated tablet or oral solution. It works by reducing inflammation in the body through inhibiting an enzyme involved in producing inflammatory molecules.
Study on Filgotinib for Patients with Refractory Behcet’s Disease, Idiopathic Inflammatory Myopathies, and IgG4-Related Disease
This trial investigates the effects of filgotinib on quality of life, disease activity, and safety in patients with rare immune-mediated inflammatory diseases who have not responded well to standard treatments. The study includes patients with Behçet’s disease alongside those with idiopathic inflammatory myopathies and IgG4-related disease.
Who can participate: Adults aged 18 years or older with a diagnosis of refractory disease are eligible. For Behçet’s disease specifically, participants must have active symptoms with a BDCAF score greater than 2 or requiring new medication. Patients must have tried corticoid therapy for 12 weeks and not responded to prednisone plus at least one other medication like methotrexate, mycophenolate mofetil, azathioprine, or rituximab. Participants must test negative for active or latent tuberculosis.
Who cannot participate: Patients with active infections, severe heart, liver, or kidney problems, uncontrolled high blood pressure, pregnancy or breastfeeding, recent cancer history (within 5 years), or unstable mental health conditions are excluded. Those who participated in another clinical trial within the last 30 days cannot join.
Study focus: The study evaluates how filgotinib affects various aspects of the diseases, including the number of oral ulcers, skin disease severity, and overall disease activity. Participants complete regular assessments using standardized forms such as the EuroQol 5D-5L to track quality of life changes throughout the treatment period.
Investigational drug: Filgotinib is a JAK inhibitor taken orally in tablet form (200 mg or 100 mg). It works by blocking Janus kinases, proteins that play a key role in inflammatory signaling pathways, thereby reducing inflammation.
Study on the Effectiveness and Safety of Tocilizumab and Adalimumab for Patients with Severe Uveitis in Behçet’s Disease
This comparative trial evaluates two different treatments for sight-threatening uveitis (eye inflammation) in patients with Behçet’s disease. The study compares tocilizumab and adalimumab to determine which provides better outcomes for this serious complication.
Who can participate: Adults aged 18 years or older with a diagnosis of Behçet’s disease or a history of recurrent mouth sores according to international criteria are eligible. Participants must have non-infectious uveitis in at least one eye with sight-threatening features, such as significant vision loss or retinal inflammation. A recent chest X-ray or CT scan showing no active tuberculosis is required, along with a negative pregnancy test for women of childbearing potential. Participants must agree to use birth control during the study.
Who cannot participate: Patients with other serious eye conditions unrelated to Behçet’s disease, recent or planned eye surgery, current use of interfering medications, history of severe allergic reactions to similar drugs, certain infections, pregnancy or breastfeeding, poorly controlled chronic diseases, or recent participation in another trial are excluded.
Study focus: The primary goal is to achieve complete remission of eye inflammation with a low dose of prednisone by week 16. The study also assesses corticosteroid-sparing effects, changes in disease activity, quality of life, and the time to treatment response. Follow-up visits occur at weeks 4, 8, 12, 16, 24, 36, and 48, with the study expected to conclude by April 2028.
Investigational drugs: Tocilizumab is an interleukin-6 receptor inhibitor given via subcutaneous injection (162 mg pre-filled syringe). Adalimumab is a tumor necrosis factor inhibitor also administered by subcutaneous injection (80 mg pre-filled pen). Both medications work by blocking specific proteins involved in the inflammatory process.
Study on Steroid and Rivaroxaban Treatment for Deep Vein Thrombosis in Behçet’s Syndrome Patients
This trial compares the effectiveness of combining steroid therapy with anticoagulants versus steroid therapy alone for treating deep venous thrombosis (blood clots in deep veins) related to Behçet’s syndrome. The study aims to find the best approach for managing this serious complication.
Who can participate: Adults aged 18 years or older with a diagnosis of Behçet’s syndrome according to international criteria are eligible. Participants must have a first or recurrent deep venous thrombosis confirmed through imaging tests such as venous ultrasound, CT scan, or MRI. Written informed consent is required, and participants must be affiliated with a social security system.
Who cannot participate: Patients without newly diagnosed or relapsing deep venous thrombosis related to Behçet’s syndrome, those outside the specified age range, or members of vulnerable populations requiring special protection are excluded.
Study focus: The study’s primary objective is to assess the absence of deep venous thrombosis relapse and major bleeding events over 6 months. Secondary evaluations include imaging to assess venous repermeabilization, monitoring of corticosteroid dose reduction at 3, 6, and 12 months, and tracking quality of life and disease activity scores. The 12-month follow-up period allows researchers to measure cumulative incidence of thrombosis relapse, bleeding events, and overall survival.
Investigational drugs: Prednisone is a corticosteroid administered orally in tablet form to reduce inflammation and suppress the immune system. Rivaroxaban is an anticoagulant taken orally to prevent blood clot formation. The study compares the combination of both medications against prednisone alone.
Study on the Safety and Effectiveness of Filgotinib for Patients with Refractory Behcet’s Disease, Idiopathic Inflammatory Myopathies, and IgG4-Related Disease
This trial examines the safety and effectiveness of filgotinib in patients with rare inflammatory diseases whose symptoms have not improved with standard treatments. The study includes Behçet’s disease along with idiopathic inflammatory myopathies and IgG4-related disease.
Who can participate: Adults aged 18 years or older with refractory disease are eligible. For Behçet’s disease, participants must have active but not life-threatening symptoms with appropriate BDCAF scores or require new medication. Patients must have tried a 12-week corticoid treatment and not responded to prednisone plus at least one other medication such as methotrexate, mycophenolate mofetil, azathioprine, or rituximab. Negative tuberculosis testing within 3 months before screening is required.
Who cannot participate: Patients with active infections, serious allergic reactions to similar medications, severe liver, heart, or kidney disease, uncontrolled high blood pressure, active cancer or recent cancer treatment (within 5 years), pregnancy or breastfeeding, or participation in another trial within the last 30 days are excluded.
Study focus: The trial evaluates filgotinib’s safety and effectiveness over a period of up to 26 weeks. Some participants receive a placebo for comparison. Regular follow-up visits include physical examinations, blood tests, and questionnaires to evaluate symptoms and overall well-being. The study monitors how well the medication reduces inflammation-related symptoms and any side effects that occur.
Investigational drug: Filgotinib is available as Jyseleca 100 mg or Jyseleca 200 mg film-coated tablets taken orally. It is a JAK inhibitor that works by blocking specific proteins involved in causing inflammation, helping to reduce symptoms like pain and swelling.
Summary
The five ongoing clinical trials for Behçet’s syndrome reflect a diverse approach to managing different manifestations of this complex disease. A notable concentration of trials is taking place in France, which hosts three studies, while the Netherlands hosts two studies focusing on the same investigational drug, filgotinib, for refractory cases. The trials span multiple European countries including Spain, Italy, and Greece.
The research covers a broad spectrum of disease complications, from oral ulcers in children to sight-threatening uveitis and deep vein thrombosis in adults. Several different therapeutic approaches are being investigated, including phosphodiesterase inhibitors, JAK inhibitors, interleukin inhibitors, TNF inhibitors, and combinations of steroids with anticoagulants. This variety reflects the challenging nature of the disease and the need for multiple treatment options tailored to specific manifestations and patient populations.
Particularly noteworthy is the inclusion of a pediatric trial for oral ulcers, addressing an important gap in treatment options for younger patients. The studies examining filgotinib represent innovative approaches for patients who have not responded to conventional therapies, potentially offering hope for those with refractory disease. Overall, these trials demonstrate ongoing efforts to improve outcomes and quality of life for people living with Behçet’s syndrome across all age groups and disease presentations.
