Drug Rediscovery for Rare Immune Mediated Inflammatory Diseases
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorUMC Utrecht
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age 18 years of older
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 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
Refractory disease, defined as symptomatic disease that persists despite a 12-week trial of glucocorticoid therapy as well as lack of response to at least one other immunosuppressive agent such as methotrexate (MTX), mycophenolate mofetil (MMF), azathioprine (AZA) or rituximab or intolerance to standard-of-care treatment, as defined by the treating physician.
Disqualifiers
Age <18 years
Age ≥65 years
Life expectancy less than 6 months
Juvenile DM, myositis overlapping with other autoimmune diseases, immune mediated necrotizing myopathy (IMNM), inclusion-body myositis or cancer-associated myositis
Trial design
Design model
Single group
Treatments tested in this trial
Filgotinib
DrugFilgotinib
Treatment groups
60 Participants
are divided into 1 treatment groupGroup A: InterventionExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
EQ-5D-5L
Change from baseline in EuroQoL-5D-5L
Time frame
26 weeks2
Disease activity in Behcet's patients
Change from baseline in Behcet's Disease Current Activity Form (BDCAF)
Time frame
26 weeks3
Disease activity in myositis patients
Change from baseline in Total Improvement Score (TIS) of the International Myositis Assessment Clinical Studies (IMACS) group
Time frame
26 weeks4
Disease activity in IgG4-RD patients
Change from baseline in IgG4-RD responder index
Time frame
26 weeksSecondary outcomes
1
Corticosteroid toxicity
Change from baseline in Glucocorticoid Toxicity Index (GTI)
Time frame
26 weeks2
Corticosteroid dosage
Change from baseline in glucocorticoid dose
Time frame
26 weeks3
VAS score of pain
Change from baseline in pain scores on the Visual Analog Scale (0-100mm). A higher score is a worse outcome
Time frame
26 weeks4
Fatigue
Change from baseline in fatigue scores on the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F). A higher score is a worse outcome
Time frame
26 weeksSponsors and contacts
Click on the lead sponsor to view all of their trials.
UMC Utrecht
Lead sponsor
Alfasigma S.p.A.
Collaborator
ReumaNederland
Collaborator
Autoimmune Research and Collaboration Hub
Collaborator
Source ClinicalTrials.gov