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

NCT06757426 | Not Yet Recruiting

• 2 other identifiers: ARIES2024-A02472-45
• Study Type: observational
• Target: 80
• Locations: 1 country
• Sites: 17

Brief Summary

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.

Conditions

Autoimmune Diseases; Inflammatory Diseases

Keywords

autoimmune; inflamatory disease; Refraction

Outcome Measures

Primary Outcomes (1)

• proportion of complete remission from the disease at week 24

  The primary outcome will be the proportion of complete remission from the disease at week 24 : 1. absence of all clinical signs and symptoms ; 2. normalization of fibrinogen and CRP values; 3. absence of radiological signs of active disease (stable or improved imaging).

  week 24

Secondary Outcomes (7)

• Evaluate the proportion of patients in clinical, biological or radiological remission

  week 12 and week 48

• Cumulative incidence of relapse (i.e., recurrence of clinical symptoms associated with biological and/or radiological inflammatory activity following complete remission)

  weeks 12, 24, 36 and 52

• Cumulative incidence of remission according to primary endpoint definitions

  weeks 12, 36 and 52

• Evaluate changes in median disease-specific activity scores

  weeks 12, 24, 36 and 52

• Cumulative prednisone dose

  weeks 12, 24, 36 and 52

Study Arms (1)

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with refractory inflammatory and autoimmune diseases

You may qualify if:

• Patients over 18 years
• Enrolled in the French national social security system
• Diagnosis of an inflammatory/autoimmune disease meeting internationally accepted classification criteria;
• 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.
• No formal contraindication to the new therapeutic class.

You may not qualify if:

• Pregnancy or breast-feeding (for women of childbearing potential, a negative serum pregnancy test will be required);
• History of severe immunosuppression, HIV or HBsAg positive.
• Positive QuantiFERON test result (QFT-TBGIn-Tube) for active tuberculosis (latent tuberculosis under treatment may be included).
• Have received live vaccines in the 3 months preceding the start of treatment.
• History of malignant tumor within the last 5 years.
• Severe renal insufficiency (creatinine clearance \<30mL/min/1.73m²)
• Liver dysfunction defined by aspartate transaminase (AST) or alanine transaminase (ALT) levels ≥ 5 times the upper limit of normal.
• Blood count abnormality:
• Platelets \< 50 x 103/mm3
• Neutropenia \< 1000/mm3
• Hemoglobin \< 8 g/dL

Sponsors & Collaborators

• Groupe français d'étude des Maladies Inflammatoires de loeil: lead

Study Sites (17)

Clinique Axium - Aix-en-provence

Aix-en-Provence, 13100, France

Location

CH Simone Veil de BLOIS

Blois, 41000, France

Location

CHU Caen Normandie

Caen, 14000, France

Location

CH du Mans

Le Mans, 72037, France

Location

APHM_Hôpital La Conception

Marseille, 13005, France

Location

APHM_Hôpital Nord

Marseille, 13915, France

Location

GHSIF Melun

Melun, 77000, France

Location

GHI Le Raincy Montfermeil

Montfermeil, 93370, France

Location

CHU Nantes - Hotel-Dieu

Nantes, 44000, France

Location

CHU Rouen_Hôpital Charles Nicolle

Rouen, 76000, France

Location

Clinique Saint-Exupéry

Saint-Exupéry, 31400, France

Location

CHRU de Tours_Hôpital Bretenneau

Tours, 37000, France

Location

APHP_Hopital Lariboisière

Paris, Île-de-France Region, 75010, France

Location

APHP_Hôpital St Antoine

Paris, Île-de-France Region, 75012, France

Location

APHP_ Hôpital Pitié-Salpêtrière

Paris, Île-de-France Region, 75013, France

Location

APHP_Hôpital Bichat

Paris, Île-de-France Region, 75018, France

Location

APHP_Hopital Ambroise Paré

Paris, Île-de-France Region, 92100, France

Location

Related Publications (4)

• Venhoff N, Schmidt WA, Bergner R, Rech J, Unger L, Tony HP, Finzel S, Andreica I, Kofler DM, Weiner SM, Lamprecht P, Schulze-Koops H, App C, Pournara E, Mendelson MH, Sieder C, Maricos M, Thiel J. Safety and efficacy of secukinumab in patients with giant cell arteritis (TitAIN): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Rheumatol. 2023 Jun;5(6):e341-e350. doi: 10.1016/S2665-9913(23)00101-7.

  PMID: 38251601 BACKGROUND

• Klein C, Brinkmann U, Reichert JM, Kontermann RE. The present and future of bispecific antibodies for cancer therapy. Nat Rev Drug Discov. 2024 Apr;23(4):301-319. doi: 10.1038/s41573-024-00896-6. Epub 2024 Mar 6.

  PMID: 38448606 BACKGROUND

• Hayter SM, Cook MC. Updated assessment of the prevalence, spectrum and case definition of autoimmune disease. Autoimmun Rev. 2012 Aug;11(10):754-65. doi: 10.1016/j.autrev.2012.02.001. Epub 2012 Feb 23.

  PMID: 22387972 BACKGROUND

• Watts RA, Hatemi G, Burns JC, Mohammad AJ. Global epidemiology of vasculitis. Nat Rev Rheumatol. 2022 Jan;18(1):22-34. doi: 10.1038/s41584-021-00718-8. Epub 2021 Dec 1.

  PMID: 34853411 BACKGROUND

Related Links

• LBA0001 EFFICACY AND SAFETY OF UPADACITINIB IN PATIENTS WITH GIANT CELL ARTERITIS (SELECT-GCA): A DOUBLE-BLIND, RANDOMIZED CONTROLLED PHASE 3 TRIAL

MeSH Terms

Conditions

Autoimmune Diseases

Condition Hierarchy (Ancestors)

Immune System Diseases

Central Study Contacts

DAVID SAADOUN, Professor

CONTACT

+33142178042; david.saadoun@aphp.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2024
• First Posted: January 3, 2025
• Study Start: January 15, 2025
• Primary Completion: July 15, 2025
• Study Completion (Estimated): July 15, 2028
• Last Updated: January 3, 2025

Record last verified: 2024-12

Locations

FR (17)