Clinical Trial: Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis: A Phase 2a, Single-centered, Randomized, Placebo-controlled, Double-blind, Proof-of-concept Pilot Stud
Brief Summary: This is a 48 week, phase IIa, single center, randomized, double-blind, placebo-controlled, proof-of-concept pilot study. All participants will first be treated with mycophenolate mofetil (MMF, Cellcept) and titrated up to a dose of 2 grams/day. Following this period, half will be given either a belimumab (Benlysta®) or placebo intravenous infusion to treat early diffuse cutaneous systemic sclerosis. Belimumab/MMF is expected to improve disease activity measured by an improvement in skin thickening and stability of pulmonary function test measurements when compared to patients treated with placebo/MMF.
Detailed Summary:
The specific objectives of this study are to:
- Determine whether belimumab used in combination with MMF is safe and tolerable in the treatment of patients with early diffuse cutaneous systemic sclerosis (Disease duration < 3 years).
- Determine whether belimumab used in combination with MMF is more effective in the treatment of diffuse cutaneous systemic sclerosis than MMF alone, as measured by change in modified Rodnan Skin Score (mRSS), forced vital capacity (FVC), hemoglobin corrected diffusion capacity (DLCO), Medsger Severity Scale (MSS), and by other physician and patient derived outcome measures.
- Determine the biological activity of Belimumab/MMF as assessed by effect on histology of skin, change in B-Cell profiles, effect on BLyS levels, and effect on serological and cutaneous biomarkers of disease activity.
Sponsor: Hospital for Special Surgery, New York
Current Primary Outcome:
- Change in Modified Rodnan Skin Score [ Time Frame: At 48 weeks ]The efficacy of the drug will be measured as the change in the Modified Rodnan Skin Score (MRSS) at 48 weeks. MRSS is a measurement of the degree of skin disease in systemic sclerosis where 17 areas of skin are rated by the examiner.
- Number of Participants with Adverse and Serious Adverse Events [ Time Frame: At 52 weeks ]The safety and tolerability of belimumab in patients with systemic sclerosis will be as assessed by comparing the rates of adverse and serious adverse events between treatment and placebo groups.
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Change in Pulmonary Function Test Measures [ Time Frame: At 24 and 48 weeks ]This endpoint would measure the change in forced vital capacity and diffusion capacity of the pulmonary function test (the breathing test)
- Change in the Medsger Severity Scale [ Time Frame: At 24 and 48 weeks ]This Medsger Severity Scale(MSS)is an objective method of assessing disease severity in patients with systemic sclerosis.
- Change in the Scleroderma Health Assessment Questionnaire, Short Form-36, and Raynaud's Condition Score [ Time Frame: At each visit ]The Scleroderma Health Assessment Questionnaire (SHAQ) is a patient survey which indexes the participants degree of disability. The Short Form-36 is a questionnaire that addresses the patient's health-related quality of life. The Raynaud's Condition Score is a measure of the degree to which the patient's Raynaud's affects his or her daily functioning.
Original Secondary Outcome: Same as current
Information By: Hospital for Special Surgery, New York
Dates:
Date Received: August 15, 2012
Date Started: August 2012
Date Completion:
Last Updated: March 15, 2017
Last Verified: March 2017
