Investigating the effectiveness and safety of existing medications in patients with rare immune diseases

Investigating the effectiveness and safety of existing medications in patients with rare immune diseases

This month the first study within the DRIMID consortium (DRIMID stands forRediscovering drugs for rare immune-mediated inflammatory diseases) has begun. This study examines the effectiveness and safety of the drug filgotinib (approved for the treatment of rheumatoid arthritis and ulcerative colitis) in three rare immune diseases (Behçet's disease, idiopathic inflammatory myositis, IgG4-related disease). DRIMID wants to investigate whether this drug – despite the lack of formal drug approval – can also be used to treat these rare immune diseases.

New medicines are typically developed for diseases that involve large patient populations (and are therefore more quickly accessible). However, developing drugs for rare diseases is more difficult. With the establishment of the DRIMID partnership, important steps have now been taken to make new medications accessible to these patient groups. The project is a collaboration between the ARCH Foundation, ReumaNederland, the pharmaceutical company AlfaSigma and a number of Dutch hospitals with the aim of (re)developing drugs for rare diseases.

Rare immune diseases

Rare immune-mediated inflammatory diseases usually have an unknown cause and are often accompanied by the formation of autoantibodies (the immune system attacks its own body). Examples of such diseases include granulomatosis with polyangiitis, inflammatory myositis, large vessel vasculitis, IgG4-related diseases, Behçet's disease, Sjögren's disease and systemic sclerosis. A major problem in clinical practice is that many patients do not respond adequately to common anti-inflammatory medications over time. Therefore, there is an unmet medical need for appropriate treatment options for this group.

The study

The aim of the study is to investigate whether filgotinib is effective in patients with rare immune diseases and whether the drug is well tolerated. Now that the first DRIMID study has been approved by the medical ethics committee, patients with one of the following rare immune diseases are being sought:

• Behçet-Krankheit
• idiopathische entzündliche Myositis
• IgG4-bedingte Erkrankung

It is important that participants have active disease symptoms at the start of the study. In addition, it is important that they have first tried regular treatment methods, such as prednisone and at least one other anti-inflammatory drug. If the disease did not respond or responded inadequately to these medications, or if the patient was hypersensitive to these medications, the patient may be eligible for this new study.

As part of the study, patients will be followed up for 26 weeks. At several points during the study, the drug's effect on symptoms will be measured by a doctor through physical examination, blood tests and questionnaires. If the drug is found to work well for the patient at the end of the study, the patient may continue taking it. Participation in the study is free of charge.

Participating hospitals

The rheumatologist Prof. Dr. Jaap van Laar (Department of Rheumatology and Clinical Immunology, UMC Utrecht) coordinated study will start in six Dutch hospitals. Patients can be approached by their doctor to participate, but they can also inquire about eligibility themselves. You can do this by sending an email to [email protected]. The research nurse at UMC Utrecht will then contact the most suitable hospital for the patient. The study will be conducted at the following locations:

– UMC Utrecht

– Amsterdam UMC

– Erasmus MC (Rotterdam)

– Radboudumc (Nijmegen)

– Haga Hospital (The Hague)

– Zuyderland Medical Center (Heerlen)

DRIMID Consortium

DRIMID is an acronym that stands for “Drug Rediscovery for Rare Immune Mediated Inflammatory Diseases.” ARCH (Arthritis Research and Collaboration Hub, a Dutch medical expert platform for rare autoimmune diseases), ReumaNederland and UMC Utrecht (the first academic partner within DRIMID) founded the public-private DRIMID consortium in 2021. The consortium is funded by ReumaNederland and Health Holland. Over time, researchers within the consortium intend to expand the project to other immune diseases, as well as to other pharmaceutical companies and drugs.

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