Evaluation of Filgotinib on Quality of Life, Disease Activity, and Safety in Refractory Behcet's Disease, Idiopathic Inflammatory Myopathies, and IgG4-Related Disease
- Trial ID
- 2022-502968-20-02
- Protocol
- 2022-502968-20
Trial statistics
Diseases & Conditions
Objectives
The primary objective of the study titled "Drug Rediscovery for rare Immune Mediated Inflammatory Diseases (DRIMID)" is to evaluate the effects of **filgotinib**, an approved JAK-inhibitor, on changes in quality of life, disease activity, and safety in patients with refractory Behcet's disease, Idiopathic Inflammatory Myopathies, and IgG4-related disease. This objective is clinically relevant as it aims to assess the potential of filgotinib to improve patient outcomes in these rare and challenging conditions, where current treatment options may be limited or ineffective.
Participants
The clinical trial involves a study population comprising both **male** and **female** participants aged 18 years and older. The trial focuses on individuals diagnosed with **Idiopathic Inflammatory Myopathies**, **Behcet's disease**, or **IgG4-related disease**. Participants are required to have refractory disease, characterized by symptoms persisting despite a 12-week trial of corticoid therapy and lack of response to at least prednisone and one other immunosuppressive agent. The trial does not include a vulnerable population. The sponsor has not provided information regarding the total number of participants. Selection criteria emphasize the absence of active or latent tuberculosis infection, as confirmed by specific diagnostic tests and evaluations. The study does not specify any particular lifestyle considerations such as diet or physical activity. Key inclusion criteria include the presence of active disease as defined by specific clinical and diagnostic parameters for each condition. The trial aims to assess the effects of filgotinib on quality of life, disease activity, and safety in this patient population.
Plans and Procedures
The clinical trial is designed to evaluate the effects of **filgotinib**, a JAK-inhibitor, on quality of life, disease activity, and safety in patients with refractory Behcet's disease, idiopathic inflammatory myopathies, and IgG4-related disease. This is a Phase IV, randomized, double-blind, controlled trial. The trial is expected to commence recruitment on March 18, 2024, and conclude by December 31, 2026. Participants will be involved for a maximum treatment period of 26 weeks, during which they will receive either 100 mg or 200 mg of Jyseleca film-coated tablets orally, depending on the assigned group.
The study will include several key visits: an initial screening visit to confirm eligibility based on inclusion criteria such as age (18 years or older) and refractory disease status, followed by regular follow-up visits to monitor treatment efficacy and safety. The primary endpoints include assessments using the EuroQol 5D-5L form, Behcet’s Disease Current Activity Form, Total Improvement Score of the International Myositis Assessment Clinical Studies group, and the IgG4-RD responder index. Secondary endpoints will evaluate various disease-specific measures, including the number of oral ulcers, cutaneous dermatomyositis disease area and severity index, and glucocorticoid toxicity index, among others.
Participants will be monitored for treatment-emergent adverse events, and any significant safety concerns may lead to early termination from the study. Conditions for early withdrawal include the development of active or latent tuberculosis, as determined by negative QuantiFERON-TB Gold and Mantoux tests, or any other serious adverse events as assessed by the clinical team. The trial aims to provide comprehensive data on the impact of filgotinib on these rare immune-mediated inflammatory diseases, contributing to the understanding of its therapeutic potential and safety profile.
Treatment
The clinical trial involves the administration of **Jyseleca 200 mg film-coated tablets**, which contain the active substance **filgotinib**. Filgotinib is a chemical compound classified under the ATC code L04AA45. The pharmaceutical form of the medication is a film-coated tablet, designed for oral administration. The maximum daily dose for this formulation is 200 mg, with a total maximum dose of 36,400 mg over a treatment period of 26 weeks. The tablets are manufactured by Galapagos and are not formulated for pediatric use. Participants are required to take the medication orally, and compliance with the dosing schedule is monitored throughout the study.
Additionally, the trial includes the use of **Jyseleca 100 mg film-coated tablets**, also containing the active substance **filgotinib**. This formulation is similarly classified under the ATC code L04AA45 and is provided in a film-coated tablet form for oral administration. The maximum daily dose for the 100 mg formulation is 100 mg, with a total maximum dose of 6,700 mg over the same 26-week treatment period. Like the 200 mg tablets, these are produced by Galapagos and are not intended for pediatric use. Participants are instructed to adhere to the oral administration route, and their compliance is systematically monitored to ensure adherence to the prescribed dosing regimen.
No non-experimental treatments, such as standard-of-care therapy, placebo, or comparator treatments, are specified in the trial protocol. The primary objective of the study is to evaluate the effects of filgotinib on quality of life, disease activity, and safety in patients with refractory Behçet's Disease (BD), Idiopathic Inflammatory Myopathies (IIM), and Immunoglobulin G4-Related Disease (IgG4-RD). The trial is designed to ensure rigorous monitoring of participant compliance and safety throughout the study duration.
Efficacy
Efficacy in the clinical trial titled "Drug Rediscovery for rare Immune Mediated Inflammatory Diseases (DRIMID)" will be assessed using a combination of primary and secondary endpoints. The primary endpoints include the EuroQol 5D-5L (EQ-5D-5L) form, Behçet’s Disease Current Activity Form (BDCAF), Total Improvement Score (TIS) of the International Myositis Assessment Clinical Studies (IMACS) group, and the IgG4-RD responder index. These endpoints are designed to evaluate changes in quality of life, disease activity, and response to treatment in patients with refractory Behçet’s disease, idiopathic inflammatory myopathy, and IgG4-related disease.
Secondary endpoints will further assess efficacy through various measures, including the area under the curve (AUC) for the number of oral ulcers, Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), and the total number of flares in each disease, as indicated by a ≥1 point increase in Physician Global Assessment (PGA) on a scale from 0-3. Additional secondary measures include the Visual Analogue Scale (VAS) of disease activity, Glucocorticoid Toxicity Index (GTI), changes in glucocorticoid dose, VAS score of pain from the patient's perspective, Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F), Patient Acceptable Symptom State (PASS), and exposure-adjusted incidence rates for treatment-emergent adverse events.
The trial will utilize validated scales and indices to collect and analyze data at specified timepoints throughout the study duration. These assessments will provide comprehensive insights into the efficacy of **filgotinib**, the active substance in Jyseleca film-coated tablets, in improving patient outcomes in the context of rare immune-mediated inflammatory diseases.
Inclusion and Exclusion Criteria
Inclusion Criteria
- Age 18 years or older
- Refractory disease, defined as symptomatic disease that persists despite a 12-week trial of corticoid therapy as well as lack of response to at least prednisone and 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.
- No evidence of active or latent or inadequately treated infection with mycobacterium tuberculosis (TB) as defined by all of the following: both a negative QuantiFERON-TB Gold (QFT-G) In-Tube test and a Mantoux tuberculin skin test performed at or within 3 months prior to screening and no signs suggestive of active TB infection as determined (and documented) by a qualified radiologist or pulmonologist as per local standard of care on a chest radiograph and no history of either untreated or inadequately treated latent or active TB infection.
- One of the following: (1) Diagnosis of Behçet’s disease without refractory life, organ or sight-threatening symtoms with active disease, defined as a BDCAF >2 or with active disease, based on clinical grounds (e.g. the need to start new or additional medication) or (2) Diagnosis of idiopathic inflammatory myopathy, according to diagnostic criteria: Dermatomyositis Classification Criteria according to the European Neuromuscular Centre guidelines 2018 or Anti-synthetase syndrome Classification Criteria according to the European Neuromuscular Centre guidelines 2003 or overlap/non-specific myositis, including polymyositis with active disease, defined as: dermatomyositis with 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 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
Exclusion Criteria
- Age <18 years
- History of VTE
- Concomitant malignancies or previous malignancies within the last five years (with exception of adequately treated basal or squamous cell carcinoma of the skin)
- Kidney injury with estimated glomerular filtration rate <15mL/min/1.73m2
- Liver failure Child Pugh C
- Absolute neutrophil count <1*109
- Absolute leukocyte count <0.5*109
- Hemoglobin <5mmol/L
- Inability to comply with study and/or follow-up procedures
- Known recent substance abuse (drugs or alcohol).
- Poor tolerability of venipuncture or lack of adequate venous access for required blood sampling during the study period.
- Life expectancy less than 6 months
- Previous non-adherence to immunosuppressants
- Hypersensitivity to the active substance or to any of the excipients
- Rare hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption
- Myositis overlapping with other autoimmune diseases, immune mediated necrotizing myopathy (IMNM) or cancer-associated myositis
- End-stage IIM wherein muscle weakness is most likely due to muscle damage, rather than myositis disease activity
- Pregnancy or lactation
- Previous use of other JAK-inhibitors
- Use of any investigational drug within one month prior to screening or within five half-lives of the investigational agent, whichever is longer.
- History of HIV
- Presence of an active infection or hepatitis
- Age ≥65 years
- Increased risk of major cardiovascular problems
- Current smoker or smoked for a long time in the past
- Increased risk of cancer
Trial Status by Country
| Country | Status | Start of Recruitment | Planned Patients |
|---|---|---|---|
 The Netherlands | Recruiting | 18 Mar 2024 | — |
| Netherlands | — | — | 40 |
Sites & Investigators
Investigational Products
Details about the medicinal products being studied in this clinical trial.
| Product Name | Role in Trial | Formulation | Administration | Max Daily Dose | Treatment Duration | EU MP Number |
|---|---|---|---|---|---|---|
Jyseleca 200 mg film-coated tablets | Test | FILM-COATED TABLETS | ORAL | 200 | 26 | PRD9422638 |
Jyseleca 100 mg film-coated tablets | Test | FILM-COATED TABLETS | ORAL | 100 | 26 | PRD9422607 |