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

Phase 1

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: 21.0Level: LLTClassification code 10042953Term: Systemic sclerosisSystem Organ Class: 100000004859
Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Registration Number: EUCTR2021-000230-33-IT

Lead Sponsor: MBERTO I - POLICLINICO DI ROMA

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 151

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 <= 5 years from the first non-Raynaud’s symptom
5. Stable SSc standard treatment within 4 weeks prior to Screening visit.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 100
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 51

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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.<br>Primary Safety endpoint:;Timepoint(s) of evaluation of this end point: 52 weeks

Secondary Outcome Measures

Name	Time	Method

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