NEw Clinical End-points in patients with primary Sjögren’s Syndrome (pSS): an Interventional Trial based on stratifYing patients

NEw Clinical End-points in patients with primary Sjögren’s Syndrome (pSS): an Interventional Trial based on stratifYing patients - NECESSITY

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-002470-32-NL

Enrollment

300

Registered

2021-07-23

Start date

2022-12-12

Completion date

Unknown

Last updated

2024-04-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary Sjögren’s syndrome (pSS) MedDRA version: 21.0 Level: PT Classification code 10040767 Term: Sjogren's syndrome System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders

Interventions

Trade Name: PLAQUENIL Product Name: Hydroxychloroquine Pharmaceutical Form: Tablet INN or Proposed INN: Hydroxychloroquine CAS Number: 747-36-4 Other descriptive name: HYDROXYCHLOROQUINE SULFATE Conce

Eligibility

Sex/Gender

All

Inclusion criteria

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 (see addenda 4) - With a high level of symptoms (ESSPRI = 5) and low systemic disease activity (ESSDAI =65 years) yes F.1.3.1 Number of subjects for this age range 50

Exclusion criteria

Exclusion criteria: For both cohorts: - Age 2x ULN (re-testing is allowed, see section 5.10) - Prolonged ECG's corrected QT interval (>500 ms) - Known history of maculopathy - Patients not informed of the risk of alcohol consumption and the recommendations to avoid alcohol during the entire study - Not affiliated to a social security regime (specific for France)

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the efficacy of each active treatment combination (hydroxychloroquine + leflunomide and hydroxychloroquine + mycophenolate mofetil) based on proportion of responder patients according to preliminary STAR at week 24. ;Secondary Objective: Cohort 1 and 2 together: To evaluate efficacy of each active treatment combination based on proportion of responder patients according to preliminary STAR at w24 Cohorts 1 and 2 separately: To evaluate efficacy of each active treatment combination at w12 and 36 based on proportion of responder patients according to preliminary STAR To evaluate efficacy of each active treatment combination at w12, 24 and 36 based on change in ESSPRI. To evaluate efficacy of each active treatment combination at w12, 24 and 36 based on change in ESSDAI/clinESSDAI To assess discriminant capacity of STAR (preliminary STAR and alter opt) relative to ESSDAI (clinESSDAI)/ESSPRI to detect changes at w24 and 36 To evaluate effect of each active treatment combination on - glandular function at w12, 24, 36 - anxiety and depression, health-related QoL at w12, 24 and 36 To evaluate utility of the PEPSS WebApp in collecting symptoms on a daily basis over 24 weeks;Primary end point(s): Each cohort is analysed separately •Cohort 1: Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm. •Cohort 2: Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm. ;Timepoint(s) of evaluation of this end point: 24 weeks	—

Measure

Time frame

Main Objective: To evaluate the efficacy of each active treatment combination (hydroxychloroquine + leflunomide and hydroxychloroquine + mycophenolate mofetil) based on proportion of responder patients according to preliminary STAR at week 24. ;Secondary Objective: Cohort 1 and 2 together: To evaluate efficacy of each active treatment combination based on proportion of responder patients according to preliminary STAR at w24 Cohorts 1 and 2 separately: To evaluate efficacy of each active treatment combination at w12 and 36 based on proportion of responder patients according to preliminary STAR To evaluate efficacy of each active treatment combination at w12, 24 and 36 based on change in ESSPRI. To evaluate efficacy of each active treatment combination at w12, 24 and 36 based on change in ESSDAI/clinESSDAI To assess discriminant capacity of STAR (preliminary STAR and alter opt) relative to ESSDAI (clinESSDAI)/ESSPRI to detect changes at w24 and 36 To evaluate effect of each active treatment combination on - glandular function at w12, 24, 36 - anxiety and depression, health-related QoL at w12, 24 and 36 To evaluate utility of the PEPSS WebApp in collecting symptoms on a daily basis over 24 weeks;Primary end point(s): Each cohort is analysed separately •Cohort 1: Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm. •Cohort 2: Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm. ;Timepoint(s) of evaluation of this end point: 24 weeks

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Secondary

Measure	Time frame
Secondary end point(s): Cohorts 1 and 2 together • Cohort 1 and 2: Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm. Cohorts 1 and 2 (the two cohorts will be analysed separately): • Proportion of patients achieving a response according to preliminary STAR from baseline at week 12 and 36 between each active treatment arm and placebo arm. • Change in ESSPRI from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. The difference of ESSPRI score at week 12, 24 and 36 (adjusted for baseline value) will be also compared between each active treatment arm and placebo arm. • Proportion of patients achieving a response in ESSPRI (defined as a decrease of ESSPRI score of = 1 (or = 15%)) from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. • Change in ESSDAI/clinESSDAI from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. The difference of ESSDAI/clinESSDAI score at week 12, 24 and 36 (adjusted for baseline value) will be also compared between each active treatment arm and placebo arm. Cohort 1: This analysis will be performed only if the number of patients with ESSDAI/clinESSDAI > 0 is sufficient. • Proportion of patients achieving a response in ESSDAI/clinESSDAI (defined as improvement of ESSDAI/clinESSDAI = 3 points) from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. • Discriminant capacity of STAR (preliminary STAR and alternate options) relative to ESSDAI (clinESSDAI)/ESSPRI at week 24 and 36 to detect changes in the placebo arm versus in each active treatment arm in each cohort and globally • Change in unstimulated whole salivary flow from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. • Change in Schirmer’s score from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. • Change in Tear	—

Measure

Time frame

Secondary end point(s): Cohorts 1 and 2 together • Cohort 1 and 2: Proportion of patients achieving a response according to preliminary STAR at week 24 between each active treatment arm and placebo arm. Cohorts 1 and 2 (the two cohorts will be analysed separately): • Proportion of patients achieving a response according to preliminary STAR from baseline at week 12 and 36 between each active treatment arm and placebo arm. • Change in ESSPRI from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. The difference of ESSPRI score at week 12, 24 and 36 (adjusted for baseline value) will be also compared between each active treatment arm and placebo arm. • Proportion of patients achieving a response in ESSPRI (defined as a decrease of ESSPRI score of = 1 (or = 15%)) from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. • Change in ESSDAI/clinESSDAI from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. The difference of ESSDAI/clinESSDAI score at week 12, 24 and 36 (adjusted for baseline value) will be also compared between each active treatment arm and placebo arm. Cohort 1: This analysis will be performed only if the number of patients with ESSDAI/clinESSDAI > 0 is sufficient. • Proportion of patients achieving a response in ESSDAI/clinESSDAI (defined as improvement of ESSDAI/clinESSDAI = 3 points) from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. • Discriminant capacity of STAR (preliminary STAR and alternate options) relative to ESSDAI (clinESSDAI)/ESSPRI at week 24 and 36 to detect changes in the placebo arm versus in each active treatment arm in each cohort and globally • Change in unstimulated whole salivary flow from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. • Change in Schirmer’s score from baseline at week 12, 24 and 36 between each active treatment arm and placebo arm. • Change in Tear

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Countries

Italy, Netherlands, Norway

Contacts

Public ContactProject manager

ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS ( AP-HP)

melanie.rousseaux@aphp.fr+3301 40275724

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026