Systemic Sclerosis
Conditions
Brief summary
Primary Objective: \- To evaluate safety and tolerability of 8-week oral administration of SAR100842 in patients with diffuse cutaneous systemic sclerosis. Secondary Objectives: * To evaluate the pharmacodynamic effect of SAR100842 in patients with systemic sclerosis as measured by disease related biomarkers and Lysophosphatidic acid (LPA) receptor signaling markers in blood and skin; * To explore the effect of SAR100842 on skin thickness in patients with systemic sclerosis as measured by the modified Rodnan Skin Score (mRSS); * To explore the effect of SAR100842 on quality of life as measured by the Scleroderma Modified Health Assessment Questionnaire (SHAQ); * To document long term safety of SAR100842 during the extension part.
Detailed description
Core part: randomized, double-blind, placebo-controlled study - 8-week treatment Extension part for participants completing the core part: Open label non-controlled study - 16-week treatment Each patient's participation in the study will be approximately 13 or 33 weeks depending on their participation in the extension part: up to 2 weeks of screening, 8 weeks of treatment in the core part, 1 to 30 days wash-out between core part and extension part , 16 weeks of treatment in the extension part and 3 weeks of follow up.
Interventions
DRUGSAR100842
Pharmaceutical form: tablets Route of administration: oral
DRUGPlacebo (for SAR100842)
Pharmaceutical form: tablets Route of administration: oral
Sponsors
Sanofi
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
: \- Patients who meet the American College of Rheumatology (ACR) criteria for systemic sclerosis with diffuse cutaneous involvement and \<36 months since the onset of the first systemic sclerosis manifestation other than Raynaud's phenomenon and have a Modified Rodnan Skin Score (mRSS) ≥ 15 and an area of definite involvement of the dorsal forearm that is considered amenable to repeated 4mm skin biopsies.
Exclusion criteria
1. Patients with high dose or unstable low dose immunosuppressive drugs, cytotoxic, anti-fibrotic or glucocorticoids drugs at least 4 weeks prior to screening 2. Serum creatinine \> 2.0 mg/dL 3. Gastrointestinal involvement preventing oral administration of study drug 4. Severe cardiac and/or pulmonary disease The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability during the 8 week treatment period (core part): Number of patients reporting adverse events | Up to 8 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline to Week 8 in biomarkers obtained from blood and skin | Day 1 and Week 8 (core part) |
| Change from baseline to Week 8 in Modified Rodnan Skin Score (mRSS) | Day 1 and Week 8 (core part) |
| Change from baseline to Week 8 in Scleroderma Health Assessment Questionnaire (SHAQ) score | Day 1 and Week 8 (core part) |
Countries
France, Italy, Switzerland, United Kingdom, United States
Outcome results
None listed