ACT14604
Research type
Research Study
Full title
Efficacy and safety of SAR156597 in the treatment of diffuse cutaneous Systemic Sclerosis (dcSSc): A randomized, double-blind, placebo-controlled, 24-week, proof of concept study.
IRAS ID
208969
Contact name
Christopher Denton
Contact email
Sponsor organisation
Sanofi
Eudract number
2016-001028-80
Duration of Study in the UK
2 years, 3 months, 12 days
Research summary
The ACT14604 study will evaluate the effectiveness and safety of SAR156597 administered subcutaneously over a 24-week period for the treatment of patients with diffuse cutaneous Systemic Sclerosis (dcSSc).
dcSSc is a rare autoimmune disease characterised by excessive connective tissue in the skin (causing skin thickening called ‘scleroderma’), muscle, joints and internal organs. People with diffuse scleroderma are often chronically tired, have a diminished appetite, show weight loss, and suffer from joint swelling and pain. Changes in the skin can include swelling, tightening and chronic itchiness. Problems of the digestive tract can make eating, nutrient absorption and elimination difficult. The most serious complications of diffuse scleroderma involve the kidneys, lungs and heart and may include renal failure, pulmonary arterial hypertension (PAH) and the potential for heart failure. PAH is the leading cause of death among scleroderma patients.
Currently, there is no approved therapy for dcSSc. The general therapeutic strategy is to address specific symptoms, while controlling any underling inflammatory process of the skin or internal organs with use of potent immunosuppressive therapies. Because these immunosuppressive therapies do not target specific pathways related to the thickening of connective tissue, they are not particularly effective and are often fraught with significant side effects.
SAR156597 is a monoclonal antibody designed to specifically disrupt signalling pathways of the immune system that could result in Systemic Sclerosis.
13 countries shall participate in the study with the aim to enrol 94 patients with dcSSc of which 47 will receive SAR156597 and 45 will receive a matching placebo.
Patients in a blinded manner will randomly be assigned to receive either 200mg SAR156597 or matching placebo per week for 24 weeks. This shall be followed by an 8 week follow up period off study treatment. In total, there shall be 8 on site and 4 telephone visits.
REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
16/EE/0335
Date of REC Opinion
6 Sep 2016
REC opinion
Further Information Favourable Opinion