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

Phase 1

• Conditions: Systemic Sclerosis (SSc) is a rare and orphan disease (DPCM 12 gennaio 2017-GU SGn°65-S.O. n°15 - 18/03/2017), characterized by immunological, vascular and fibroticabnormalities. The estimated incidence is 18 to 20 cases per million population yearand a prevalence of 100 to 300 cases per million population. In Europe the prevalencerate is estimated around 200 per million while in the Italian population around 20000persons suffer from this form of autoimmune disease.; MedDRA version: 20.0Level: SOCClassification code: 10040785Term: Skin and subcutaneous tissue disorders Class: 16; MedDRA version: 20.0Level: SOCClassification code: 10047065Term: Vascular disorders Class: 12; MedDRA version: 21.0Level: LLTClassification code: 10042953Term: Systemic sclerosis Class: 10028395; MedDRA version: 20.0Level: SOCClassification code: 10028395Term: Musculoskeletal and connective tissue disorders Class: 17; MedDRA version: 20.0Level: SOCClassification code: 10021428Term: Immune system disorders Class: 4; MedDRA version: 20.0Level: SOCClassification code: 10017947Term: Gastrointestinal disorders Class: 14; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: CTIS2024-516050-22-00
• Lead Sponsor: Azienda Ospedaliero-Universitaria Policlinico Umberto I

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-07-10
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 151

Inclusion Criteria

Diagnosis of SSc according 2013 ACR/EULAR criteria for classification of SSc (van den Hoogen et al, 2013)., Written Informed Consent (IC)(patient must be able and agree to sign an IC according ICH-GCP guidelines and local laws), Age >= 18 years, Disease duration <= 5 years from the first non-Raynaud’s symptom, Stable SSc standard treatment within 4 weeks prior to Screening visit, Subjects naïve to treatment with hydroxychloroquine or who have undergone a wash-out period of at least 16 weeks (approximately 3 half-lives of the drug)

Exclusion Criteria

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

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<br>oral dose of 6 mg/kg daily (up to 400 mg/day), in the treatment of early systemic<br>sclerosis (SSc) associated to SSc standard therapies (immunosuppressive and/or<br>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.