Prospective Observational Study Evaluating the Safety and Efficacy of Immunomodulatory Therapies in Refractory Inflammatory and Autoimmune Diseases

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Total Enrollment

80 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-01-15

Study Completion Date

2028-07-15

Brief Summary

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Inflammatory and/or autoimmune diseases represent a very broad group of diseases with highly variable clinical features, including - but not limited to - systemic connectivites and vasculitides. As these diseases are rare and heterogeneous, it is difficult to conduct randomized clinical trials in this setting. Refractory cases are therefore treated with drugs that are already available on the market for other indications in more frequent and clinically homogeneous diseases, such as inflammatory rheumatism and haematological malignancies.

Prescribing treatments from other specialties (e.g. rheumatology and oncohaematology) is a reality in the clinical practice of internists and immunologists, often representing an excellent solution for difficult-to-treat inflammatory and/or autoimmune diseases. As these molecules are prescribed without the availability of standardized data, it is essential to collect them prospectively to better characterize the efficacy and tolerability of these new therapeutic options in severe inflammatory and/or autoimmune diseases.

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Detailed Description

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Inflammatory and/or autoimmune diseases represent a very broad group of diseases with highly variable clinical features, including - but not limited to - systemic connectivites and vasculitis. Individually, they are very rare diseases, generally estimated in terms of the number of cases per 100,000 or 1,000,000 inhabitants. However, if we consider all immune-mediated inflammatory diseases, it is estimated that they affect 4.5% of the world's population .

Despite their extreme heterogeneity in terms of clinical presentation, these diseases share several key pathogenic mechanisms. For example, over the past two decades, adaptive immunity has been successfully targeted with several monoclonal antibodies directed against B lymphocytes (e.g. rituximab, an anti-CD20) and co-stimulation between B and T lymphocytes (e.g. abatacept, a CTLA-4 agonist). Targeting cytokines common to several of these diseases has also proved effective in their management, in particular anti-TNF alpha and anti-IL-6, produced by cells of the innate and adaptive immune systems.

Although these molecules have ushered in a new era in the management of inflammatory and/or autoimmune diseases, not all patients are yet fully controlled, representing a major challenge in clinical practice.

Refractory cases are then treated with drugs that are already available on the market for other indications in more frequent and clinically homogeneous diseases, such as inflammatory rheumatism and haematological malignancies.

Certain therapeutic targets stand out. The first is the cytokine IL-17, which plays a crucial role in the polarization of T helper 17 (Th17) lymphocytes and orchestrates the adaptive response detectable in the blood and target tissues of various inflammatory and/or autoimmune diseases.

Another interesting modern strategy is the inhibition of the Janus kinase and signal transducer/activator of transcription (JAK/STAT) pathways, which act on several downstream cytokine receptors. The development of targeted oral therapies based on small molecules such as JAK inhibitors (JAKi) represents an effective means of simultaneously attenuating several downstream inflammatory pathways, and has enabled a paradigm shift in the treatment of various autoimmune and inflammatory conditions refractory to conventional therapy, with cortisone sparing as well.

Bispecific antibodies (BsAb) are a new class of drugs and one of the most promising immunotherapies for solid tumors and hematological malignancies. BsAbs combine the specificities of two therapeutic antibodies and simultaneously target different antigens or epitopes. They have attracted a great deal of interest over the past decade, thanks to their unique and versatile modes of action.

In summary, the prescription of commercially available treatments from other specialties (e.g. rheumatology and oncohaematology) is a reality in the clinical practice of internists and immunologists, often representing an excellent solution for difficult-to-treat inflammatory and/or autoimmune diseases. As these molecules are prescribed without the availability of standardized data, it is essential to collect them prospectively to better characterize the efficacy and tolerability of these new therapeutic options in inflammatory and/or autoimmune diseases.

Conditions

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Autoimmune Diseases Inflammatory Diseases

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Adult patients with immune-mediated inflammatory disease refractory to conventional lines of therapy

Adult patients with immune-mediated inflammatory disease refractory to conventional lines of therapy.

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

1. Patients over 18 years
2. Enrolled in the French national social security system
3. Diagnosis of an inflammatory/autoimmune disease meeting internationally accepted classification criteria;
4. Clinical activity of their disease with biological and/or radiological signs, refractory to conventional therapeutic lines, requiring a new treatment as an addition or replacement according to clinical judgment.
5. No formal contraindication to the new therapeutic class.

Exclusion Criteria

1. Pregnancy or breast-feeding (for women of childbearing potential, a negative serum pregnancy test will be required);
2. History of severe immunosuppression, HIV or HBsAg positive.
3. Positive QuantiFERON test result (QFT-TBGIn-Tube) for active tuberculosis (latent tuberculosis under treatment may be included).
4. Have received live vaccines in the 3 months preceding the start of treatment.
5. History of malignant tumor within the last 5 years.
6. Severe renal insufficiency (creatinine clearance \<30mL/min/1.73m²)
7. Liver dysfunction defined by aspartate transaminase (AST) or alanine transaminase (ALT) levels ≥ 5 times the upper limit of normal.
8. Blood count abnormality:

* Platelets \< 50 x 103/mm3
* Neutropenia \< 1000/mm3
* Hemoglobin \< 8 g/dL

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Locations

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Clinique Axium - Aix-en-provence

Aix-en-Provence, , France

Site Status

CH Simone Veil de BLOIS

Blois, , France

Site Status

CHU Caen Normandie

Caen, , France

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CH du Mans

Le Mans, , France

Site Status

APHM_Hôpital La Conception

Marseille, , France

Site Status

APHM_Hôpital Nord

Marseille, , France

Site Status

GHSIF Melun

Melun, , France

Site Status

GHI Le Raincy Montfermeil

Montfermeil, , France

Site Status

CHU Nantes - Hotel-Dieu

Nantes, , France

Site Status

CHU Rouen_Hôpital Charles Nicolle

Rouen, , France

Site Status

Clinique Saint-Exupéry

Saint-Exupéry, , France

Site Status