Ireland
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Recruiting Phase 2

New Clinical End-points in Patients With Primary Sjögren's Syndrome

NCT05113004 · tracked via the Priya Life Science France tracker
Phase
Phase 2
Started
2022-01-20
Last updated
2024-11-01

Condition(s) studied

Primary Sjögren's Syndrome (pSS)

Investigational drug(s) / intervention(s)

Hydroxychloroquine 400mg/d →Leflunomide 20mg/d →Mycophenolate mofetil 2000mg/d →Placebo of Hydroxychloroquine 400mg/dPlacebo of Leflunomide 20mg/dPlacebo of Mycophenolate mofetil 2000mg/d

Hydroxychloroquine 400mg/d: Hydroxychloroquine (HCQ) is a 4-aminoquinoline belonging to the group of antimalarial agents. Its immunomodulatory activity on B-cells has mainly been attributed to its inhibition of antigen presentation, cytokine production, and recently on Toll-like receptor signaling and IFN secretion that drives B cell activation.

Leflunomide 20mg/d: Leflunomide (LEF) is a derivative of isoxazole and is converted into an active metabolite which blocks de novo synthesis of pyrimidines in activated T lymphocytes, thereby inhibiting T cell proliferation and consequently T cell-dependent B cell formation of autoantibodies.

Mycophenolate mofetil 2000mg/d: Mycophenolate mofetil (MMF) is a morpholinoethyl ester of mycophenolic acid which blocks proliferation of lymphocytes by inhibiting the de novo pathway of purine biosynthesis (Allison, 2000).

Placebo of Hydroxychloroquine 400mg/d: Placebo of Hydroxychloroquine (HCQ) is a 4-aminoquinoline belonging to the group of antimalarial agents. Its immunomodulatory activity on B-cells has mainly been attributed to its inhibition of antigen presentation, cytokine production, and recently on Toll-like receptor signaling and IFN secretion that drives B cell activation.

Placebo of Leflunomide 20mg/d: Placebo of Leflunomide (LEF) is a derivative of isoxazole and is converted into an active metabolite which blocks de novo synthesis of pyrimidines in activated T lymphocytes, thereby inhibiting T cell proliferation

Placebo of Mycophenolate mofetil 2000mg/d: Placebo of Mycophenolate mofetil (MMF) is a morpholinoethyl ester of mycophenolic acid which blocks proliferation of lymphocytes by inhibiting the de novo pathway of purine biosynthesis (Allison, 2000).

Study summary

There are no approved treatments for pSS and the clinical endpoints currently used in clinical trials are inadequate to capture all aspects of the disease that should be evaluated in clinical trials. The newly developed composite endpoint: Sjögren's Tool for Assessing Response to treatment (STAR) will allow a more specific and meaningful assessment of treatment efficacy in pSS.

Because of the heterogeneity of the disease and of the central role of the interplay between B- and T-cells in the pathogenesis, it is worth to evaluate combination of conventional synthetic immunomodulatory drugs targeting both B- and T-cells.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * Cohort 1 * Having given written informed consent prior to undertaking any study-related procedures. * Patients with pSS according to ACR/EULAR 2016 criteria or AECG 2002 criteria * With a high level of symptoms (ESSPRI ≥ 5) and low systemic disease activity (ESSDAI \< 5). * Negative pregnancy test (serum at screening) * Use highly reliable contraception during research treatment from the screening and for two years after stopping treatment. * Cohort 2 * Having given written informed consent prior to undertaking any study-related procedures. * Patients with pSS according to ACR/EULAR 2016 criteria or AECG 2002 criteria * With moderate/high systemic disease activity, as defined by ESSDAI ≥ 5. * Negative pregnancy test (serum at screening) * Use highly reliable contraception during research treatment from the screening and for two years after stopping treatment Exclusion Criteria: * For both cohorts: * Age \< 18 years * Pregnant or breastfeeding women or women wanted to conceive either during or within two years after the end of the treatment period * Women of childbearing potential not using highly effective methods of contraception (as defined in section 6.3) * Participation in another interventional trial * Contra-indication to HCQ: pre-existing retinopathy, hypersensitivity to HCQ or to any of the excipients of the specialty used * Contra-indication to MMF: hypersensitivity to mycophenolate mofetil, acid mycophenolic, mycophenolate sodium or to any of the excipients of the specialty used * Contra-indication tor LEF: hypersensitivity to the active substance, the main active metabolite teriflunomide or to any excipients of the specialty used. * Concomitant treatment with corticosteroids more than 10 mg/day of prednisone equivalent at screening or inclusion (randomisation) * Concomitant treatment with other immunomodulators including methotrexate, azathioprine, cyclophosphamide, cyclosporine and tacrolimus * Previous treatment with HCQ, LEF, MMF in the last 3 months * Previous treatment with rituximab, other B-cell targeted biologic therapy or cyclophosphamide in the last 6 months * Previous treatment with anti-TNF, abatacept, tocilizumab or belimumab or any other biologic in the setting of a past clinical trial in the last 3 months * Severe life-threatening systemic involvement requiring cyclophosphamide or high dose corticosteroids, or any drug considered as an exclusion criteria * Impairment of other severe immunodeficiency states * Patients with active malignancy or history of malignancy within the last 5 years except non-melanoma skin cancer * Patients with history of gastrointestinal tract ulceration, hemorrhage and perforation * Patients with history of cardiomyopathy * Patients with known hereditary deficiency of hypoxanthine-guanine phosphoribosyl-transferase (HGPRT) such as Lesch-Nyhan and Kelley-Seegmiller syndrome * Serious infection in the past month * Evidence of active tuberculosis infection * Active HCV (positive PCR) * Active HBV infection (positivity for HBS antigen, or positivity for anti-HBC antibody without any HBS antigen) * HIV infection (positive serology) * Positive SARS-Cov2 PCR (if vaccinated for COVID-19, no PCR is required; if history of COVID-19 infection, positive serology is sufficient) * Cytopenia defined as neutrophils \< 1.0 G/L, lymphocytes \< 0.5 G/L, Hb \< 10 g/dl or platelets \< 100 G/L * Moderate to severe renal insufficiency (GFR \< 30 ml/min) * Severe hypogammaglobulinemia defined as gamma globulins or IgG \< 5 g/l Reduced hepatic function: AST or ALT \> 2x ULN (re-testing is allowed, see section 5.10) * Prolonged ECG's corrected QT interval (\>500 ms) * Known history of maculopathy * Patients will be informed of the risk of alcohol consumption and will be recommended to avoid alcohol during the entire study * Not affiliated to a social security regime (specific for France)

Primary outcome measure(s)

  • Cohort 1. Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm. — During the 24 weeks of the trials
  • Cohort 2. Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm. — During the 24 weeks of the trials

Trial sites (7)

FacilityCityRegionStatus
Valérie Devauchelle Brest France Recruiting
Eric Hachulla Lille France Recruiting
Jacques Morel Montpellier France Recruiting
Véronique Le Guern Paris France Recruiting
Jacques-Eric Gottenberg Strasbourg France Recruiting
Christophe Richez Talence France Recruiting
Raphaele Seror Le Kremlin-Bicêtre Île-de-France Region Recruiting

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05113004 on ClinicalTrials.gov ↗ ← All trials in France