Clinical Trial: AIMSPRO in Established Diffuse Cutaneous Systemic Sclerosis
Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional
Official Title: A Double-Blind Placebo-Controlled Pilot Study of Safety and Tolerability of AIMSPRO in Established Diffuse Cutaneous Systemic Sclerosis
Brief Summary: To study the safety and tolerability of a hyperimmune goat serum product (AIMSPRO) in the treatment of systemic sclerosis (SSc) through a period of 26 weeks of study participation. The secondary objective of the study is to assess the efficacy of AIMSPRO as a therapeutic agent for SSc using inter alia the SSc-HAQ questionnaire and the modified Rodnan skin score.
Detailed Summary:
Systemic sclerosis (scleroderma, SSc) is a multisystemic disease clinically characterized by fibrosis of the skin, joints, muscles and internal organs. Systemic sclerosis is a rare disease, with an estimated incidence of 19 individuals per million population per year.
The pathogenesis of SSc remains incompletely understood although it seems likely that there is an interplay between inflammatory, vascular and fibroblast dysfunction, leading ultimately to the sustained activation of a population of fibroblasts that deposit increased amounts of extracellular matrix in lesional tissues, including the skin and internal organs.
Impairment of the immune system is currently thought to play an important role. This is based on the observation that in the early phases of SSc, mononuclear cells migrate to the dermis and accumulate around small blood vessels, nerves and skin appendages. Furthermore, there is a direct relation between the extent of cutaneous inflammation and the extent and progression of fibrosis of the skin. Stimulated T-lymphocytes of patients with SSc produce more tumor necrosis factor-α, interleukin-1 and -2 compared with healthy controls and the serum concentrations of IL-2, IL-4, IL-6 and IL-8 and soluble IL-2 receptors are elevated. The occurrence of autoantibodies, predominantly antitopoisomerase-1 (ATA) and anticentromere antibodies (ACA), in approximately 90% of the patients, points to an alteration of the humoral immune system.
Systemic sclerosis can be subdivided into diffuse and limited forms. Diffuse cutaneous SSc (dcSSc) is characterised by skin involvement proximal to the elbows and knees, limited cutaneous SSc (lcSSc) by skin involvement distal to these joints. In approximately 50% of patients with dcSSc, ATA can be detected, and in approximately 50
Sponsor: Daval International Limited
Current Primary Outcome: Modified Rodnan Skin Score [ Time Frame: Baseline, Week 6 and Week 26 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Scleroderma Health Assessment Questionnaire [ Time Frame: Baseline, Week 6 and Week 26 ]
- Scleroderma UK Functional Score [ Time Frame: Baseline, Week 6 and Week 26 ]
- Patient and Physician Global Assessment (VAS) [ Time Frame: Baseline, Week 6 and Week 26 ]
- SF-36 (Short form 36) [ Time Frame: Baseline, Week 6 and Week 26 ]
- MRC Sum Score [ Time Frame: Week 0, Week 6 and Week 26 ]
Original Secondary Outcome: Same as current
Information By: Daval International Limited
Dates:
Date Received: October 7, 2008
Date Started: December 2008
Date Completion: September 2011
Last Updated: August 16, 2011
Last Verified: August 2011
