Proof of Biological Activity of SAR100842 in Systemic Sclerosis - Full Text View

Purpose

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.

Condition	Intervention	Phase
Systemic Sclerosis	Drug: SAR100842 Drug: Placebo (for SAR100842)	Phase 2


Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	Double-blind, Randomized, 8-week Placebo-controlled, and 16-week Open-label Extension Study Investigating the Safety, Pharmacokinetics and Pharmacodynamics of SAR100842 Given Orally to Patients With Diffuse Cutaneous Systemic Sclerosis

Resource links provided by NLM:

Genetics Home Reference related topics: systemic scleroderma

MedlinePlus related topics: Scleroderma

Genetic and Rare Diseases Information Center resources: Systemic Scleroderma Diffuse Cutaneous Systemic Sclerosis

U.S. FDA Resources

Further study details as provided by Sanofi:

Primary Outcome Measures:

Safety and tolerability during the 8 week treatment period (core part): Number of patients reporting adverse events [ Time Frame: Up to 8 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Change from baseline to Week 8 in biomarkers obtained from blood and skin [ Time Frame: Day 1 and Week 8 (core part) ] [ Designated as safety issue: No ]
Change from baseline to Week 8 in Modified Rodnan Skin Score (mRSS) [ Time Frame: Day 1 and Week 8 (core part) ] [ Designated as safety issue: No ]
Change from baseline to Week 8 in Scleroderma Health Assessment Questionnaire (SHAQ) score [ Time Frame: Day 1 and Week 8 (core part) ] [ Designated as safety issue: No ]


Enrollment:	32
Study Start Date:	January 2013
Study Completion Date:	April 2014
Primary Completion Date:	November 2013 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: SAR100842 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	Drug: SAR100842 Pharmaceutical form: tablets Route of administration: oral
Placebo Comparator: Placebo 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	Drug: SAR100842 Pharmaceutical form: tablets Route of administration: oral Drug: Placebo (for SAR100842) Pharmaceutical form: tablets Route of administration: oral

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.

Eligibility


Ages Eligible for Study:	18 Years and older (Adult, Senior)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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:

Patients with high dose or unstable low dose immunosuppressive drugs, cytotoxic, anti-fibrotic or glucocorticoids drugs at least 4 weeks prior to screening
Serum creatinine > 2.0 mg/dL
Gastrointestinal involvement preventing oral administration of study drug
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.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01651143

Locations


United States, Arizona
Investigational Site Number 840006
Scottsdale, Arizona, United States, 85259-5499
United States, District of Columbia
Investigational Site Number 840003
Washington, District of Columbia, United States, 20007
United States, Maryland
Investigational Site Number 840004
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Investigational Site Number 840001
Boston, Massachusetts, United States, 02118
United States, Michigan
Investigational Site Number 840002
Ann Arbor, Michigan, United States, 48106
United States, New Jersey
Investigational Site Number 840007
New Brunswick, New Jersey, United States, 08903
United States, Pennsylvania
Investigational Site Number 840008
Pittsburgh, Pennsylvania, United States, 15213
France
Investigational Site Number 250003
Lille, France, 59037
Investigational Site Number 250001
Paris Cedex 14, France, 75679
Italy
Investigational Site Number 380001
Milano, Italy, 20122
Switzerland
Investigational Site Number 756001
Zürich, Switzerland, 8091
United Kingdom
Investigational Site Number 826001
London, United Kingdom
Investigational Site Number 826002
Salford, United Kingdom, M6 8HD

Sponsors and Collaborators

Sanofi

Investigators


Study Director:	Clinical Sciences & Operations	Sanofi

More Information


Responsible Party:	Sanofi
ClinicalTrials.gov Identifier:	NCT01651143 History of Changes
Other Study ID Numbers:	ACT12339 2012-001369-34 U1111-1127-2854
Study First Received:	July 24, 2012
Last Updated:	February 26, 2016
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:


Sclerosis Scleroderma, Systemic Scleroderma, Diffuse	Pathologic Processes Connective Tissue Diseases Skin Diseases

ClinicalTrials.gov processed this record on September 27, 2016