TRIAL EVALUATING EFFICACY AND SAFETY OF HYDROXYCHLOROQUINE VERSUS PLACEBO IN EARLY SYSTEMIC SCLEROSIS (SSc)

A DOUBLE BLIND, RANDOMIZED, PLACEBO-CONTROLLED, ADD-ON TRIAL EVALUATING EFFICACY AND SAFETY OF HYDROXYCHLOROQUINE IN EARLY SYSTEMIC SCLEROSIS (SSc)- HYDROXYSSc - HYDROXYSSc

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-000230-33-IT

Enrollment

151

Registered

2021-08-17

Start date

2022-01-26

Completion date

Unknown

Last updated

2024-12-03

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

Conditions

Systemic Sclerosis (SSc) is a rare and orphan disease (DPCM 12 gennaio 2017-GU SG n°65-S.O. n°15 - 18/03/2017), characterized by immunological, vascular and fibrotic abnormalities. The estimated incidence is 18 to 20 cases per million population year and a prevalence of 100 to 300 cases per million population. In Europe the prevalence rate is estimated around 200 per million while in the Italian population around 20000 persons suffer from this form of autoimmune disease. MedDRA version: 21.0 Le

Interventions

Product Name: Idrossiclorochina solfato Product Code: [N/A] Pharmaceutical Form: Tablet INN or Proposed INN: idrossiclorochina solfato CAS Number: 118-42-3 Current Sponsor code: idrossiclorochina solf

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Diagnosis of SSc according 2013 ACR/EULAR criteria for classification of SSc (van den Hoogen et al, 2013). 2. Written Informed Consent (IC)(patient must be able and agree to sign an IC according ICH-GCP guidelines and local laws) 3. Age >= 18 years 4. Disease duration =65 years) yes F.1.3.1 Number of subjects for this age range 51

Exclusion criteria

Exclusion criteria: 1. Known hypersensytivity to the study drug (active substance or excipients) or derivatives 4-aminoquinolines 2. Age < 18 years 3. Body weight < 45 kg 4. Anticoagulant and/or antiplatelet therapy 5. History of retynopathy and/or maculopathy 6. History of periferic neuropathy 7. History of severe miopathy (other than SSc related) 8. History of hypoglycemia 9. History of bradycardia (HR<50) or ventricular arrhythmias 10. Deficiency of glucose-6-phosphate dehydrogenase 11. Unstable SSc or SSc with end-stage organ involvement at Screening or Visit 1 12. Pregnant or breast feeding women 13. Other contraindicated clinical and / or laboratory conditions

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the efficacy and safety of hydroxychloroquine compared to placebo, at the oral dose of 6 mg/kg daily (up to 400 mg/day), in the treatment of early systemic sclerosis (SSc) associated to SSc standard therapies (immunosuppressive and/or vasoactive).;Secondary Objective: To evaluate the effectiveness of adding hydroxychloroquine to standard therapy for SSc on ESScGAI, capillaroscopic parameters and CSURI Index, VAS pain, Morning Stiffness (MS), FACIT Fatigue Index and Raynaud Condition Score (RCS); Assess the tolerability and variation of clinical, laboratory and biomarker parameters from baseline.;Primary end point(s): To evaluate efficacy of HCQ add-on compared to placebo by assessing changes from baseline of American College of Rheumatology Combined Response Index for Systemic Sclerosis (CRISS)(Khanna D 2009) at week 52. Primary Safety endpoint:;Timepoint(s) of evaluation of this end point: 52 weeks	—

Measure

Time frame

Main Objective: To evaluate the efficacy and safety of hydroxychloroquine compared to placebo, at the oral dose of 6 mg/kg daily (up to 400 mg/day), in the treatment of early systemic sclerosis (SSc) associated to SSc standard therapies (immunosuppressive and/or vasoactive).;Secondary Objective: To evaluate the effectiveness of adding hydroxychloroquine to standard therapy for SSc on ESScGAI, capillaroscopic parameters and CSURI Index, VAS pain, Morning Stiffness (MS), FACIT Fatigue Index and Raynaud Condition Score (RCS); Assess the tolerability and variation of clinical, laboratory and biomarker parameters from baseline.;Primary end point(s): To evaluate efficacy of HCQ add-on compared to placebo by assessing changes from baseline of American College of Rheumatology Combined Response Index for Systemic Sclerosis (CRISS)(Khanna D 2009) at week 52. Primary Safety endpoint:;Timepoint(s) of evaluation of this end point: 52 weeks

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Secondary

Measure	Time frame
Secondary end point(s): Secondary efficacy endpoints: To evaluate efficacy of HCQ add-on by assessing change from baseline of: 1. European Systemic Sclerosis Global Disease Activity Index (ESScGDAI) at week 52 2. Visual Analogue Scale (VAS) for pain at weeks 26 and 52 3. Morning stiffness (MS) duration (in minutes) at weeks 26 and 52 4. The FACIT fatigue Index at week 26 and 52 5. Raynaud Condition Score (RCS) scale at week 26 and 52 6. Naifold Capillaroscopy (NC) main parameters and CSURI (Capillaroscopic Skin Ulcer Risk Index) at week 52; Safety of treatment in terms of treatment-emergent adverse events (TEAEs) associated with treatment with HCQ in combination with standard therapy for SSc and changes in vital signs, weight, body mass index, physical examinations, laboratory safety tests, electrocardiograms (ECGs); Exploratory biological endpoints: to evaluate the effects of HCQ add-on compared to placebo by assessing changes from baseline in CXCL4 the levels or CXCL4-DNA complexes and in IFN-I concentrations in peripheral blood of SSc patients.;Timepoint(s) of evaluation of this end point: 26 e 52 weeks; 52 weeks; 52 weeks	—

Measure

Time frame

Secondary end point(s): Secondary efficacy endpoints: To evaluate efficacy of HCQ add-on by assessing change from baseline of: 1. European Systemic Sclerosis Global Disease Activity Index (ESScGDAI) at week 52 2. Visual Analogue Scale (VAS) for pain at weeks 26 and 52 3. Morning stiffness (MS) duration (in minutes) at weeks 26 and 52 4. The FACIT fatigue Index at week 26 and 52 5. Raynaud Condition Score (RCS) scale at week 26 and 52 6. Naifold Capillaroscopy (NC) main parameters and CSURI (Capillaroscopic Skin Ulcer Risk Index) at week 52; Safety of treatment in terms of treatment-emergent adverse events (TEAEs) associated with treatment with HCQ in combination with standard therapy for SSc and changes in vital signs, weight, body mass index, physical examinations, laboratory safety tests, electrocardiograms (ECGs); Exploratory biological endpoints: to evaluate the effects of HCQ add-on compared to placebo by assessing changes from baseline in CXCL4 the levels or CXCL4-DNA complexes and in IFN-I concentrations in peripheral blood of SSc patients.;Timepoint(s) of evaluation of this end point: 26 e 52 weeks; 52 weeks; 52 weeks

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Countries

Italy

Contacts

Public ContactScleroderma Clinic

Reumatologia-Dipartimento di Scienze Cliniche Internistiche, Anestesiologiche e Cardiovascolari- Policlinico Umberto I-

sclerodermaclinic@gmail.com064463534

Outcome results

None listed

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