RecruitingPhase 2

Drug Rediscovery for Rare Immune Mediated Inflammatory Diseases

Netherlands60 participantsStarted 2024-03-12

Plain-language summary

Research into novel therapies for rare, immune-mediated inflammatory diseases (IMIDs) is limited due to small patient populations. Patients with Behçet's disease (BD), idiopathic inflammatory myopathy (IIM, also known as myositis) and IgG4-related disease (IgG4-RD) are treated with high-dosed glucocorticoids, methotrexate, azathioprine and mycophenolate mofetil, mostly for long periods of time with attendant risks of long-term toxicity, including infections. Therefore, there is an urgent need for new, more specific anti-inflammatory therapies such as targeted synthetic and biological disease-modifying antirheumatic drugs. Due to the role of type 1 interferon in both BD, IIM and IgG4-RD, JAK-STAT inhibition may be a promising treatment strategy in these conditions, because JAK1 is critical for the signal transduction of pro-inflammatory cytokine receptors. Previous research showed that JAK1 inhibition reduces activation of type 1 interferon-regulated proteins and key chemokines that control tissue inflammation.

Who can participate

Age range

18 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Age 18 years of older * One of the following rare IMIDs: * Diagnosis of Behçet's disease without refractory life, organ or sight-threatening symptoms with active disease, defined as a BDCAF \>2 (new BDCAF) or \>15 (old BDCAF) or with active disease, based on clinical grounds (e.g. the need to start new or additional medication * Diagnosis of idiopathic inflammatory myopathy, according to diagnostic criteria: Dermatomyositis: Dermatomyositis Classification Criteria according to the European Neuromuscular Centre guidelines 201852 or anti-synthetase syndrome: Anti- synthetase syndrome Classification Criteria according to the European Neuromuscular Centre guidelines 200353, both with active disease, defined as a CDASI score of ≥5 or abnormal levels of at least 1 of the following enzymes: creatine kinase (≥ 4× upper limit of normal \[ULN\]), aldolase (≥4 × ULN), lactate dehydrogenase (LDH ≥4 × ULN), aspartate transaminase (AST ≥4 × ULN), alanine aminotransferase (ALT ≥4 × ULN) or a MRI within the last 3 months indicative of active inflammation (e.g. edema signal pattern in affected proximal muscles) or active disease based on clinical grounds, e.g. the need to start new or additional medication * Diagnosis of IgG4-related disease, according to 2019 ACR/EULAR guidelines, with active disease, defined as: IgG4-related disease responder index \>10 or active disease based on clinical grounds, e.g. the need to start new or additional medication * Refract…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1This trial is testing existing drugs in new ways for rare diseases like Behcet's, myositis, or IgG4-related disease — could you help me understand which specific drug or drugs are being studied, and why they're being considered for my particular condition?
2Since this is a Phase 2 trial, what does that mean for how much is already known about the safety and effectiveness of the treatment being tested, and how does that weigh against my current standard-of-care options?
3The trial is measuring disease activity specifically for my condition as a primary outcome — can you explain what tools or scoring systems they'll use to track whether my disease is getting better or worse, and how that compares to how you currently monitor me?
4The trial also measures quality of life using the EQ-5D-5L questionnaire — do you think my day-to-day functional challenges would be captured well by that measure, and is quality of life improvement a realistic goal with this approach?
5Given that this trial is recruiting across three different rare diseases at once, would my care still be closely tailored to my specific diagnosis, and are there any visit schedules or monitoring requirements I should know about before considering whether this fits my life?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

EQ-5D-5L

Timeframe: 26 weeks

Disease activity in Behcet's patients

Timeframe: 26 weeks

Disease activity in myositis patients

Timeframe: 26 weeks

Disease activity in IgG4-RD patients

Timeframe: 26 weeks

Trial details

NCT IDNCT06285539

SponsorUMC Utrecht

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-12

Contact for this trial

Anne Karien Marijnissen, Dr

+887550459 a.c.a.marijnissen@umcutrecht.nl

View on ClinicalTrials.gov More Behcet's Disease trials

Plain-language summary

Who can participate

Age range

18 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.