Proof of Biological Activity of SAR100842 in Systemic Sclerosis

Sanofi

Status and phase: Completed; Phase 2

Conditions: Systemic Sclerosis

Treatments: Drug: SAR100842; Drug: Placebo (for SAR100842)

Study type: Interventional

Funder types: Industry

Identifiers

NCT01651143

2012-001369-34 (EudraCT Number)

ACT12339

U1111-1127-2854 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 32 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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32 participants in 2 patient groups, including a placebo group

SAR100842

Experimental group

Description:

Core part: SAR100842 300 mg, oral administration twice daily, for 8 weeks Extension part: SAR100842 300 mg, oral administration twice daily, for 16 additional weeks

Treatment:

Drug: SAR100842

Placebo

Placebo Comparator group

Description:

Core part: Placebo (for SAR100842), oral administration twice daily, for 8 weeks Extension part: SAR100842 300 mg, oral administration twice daily, for 16 additional weeks

Treatment:

Drug: Placebo (for SAR100842)

Drug: SAR100842