A Pilot-Study of Sirolimus for the Treatment of Systemic Sclerosis

A Phase Ⅱ Pilot-Study With Sirolimus for the Treatment of Systemic Sclerosis

The purpose of the study is to examine the safety and effectiveness of sirolimus treatment for people with systemic sclerosis.

The investigators perform a multi-centre, double-blind pilot trial with sirolimus in SSc.The investigators evaluate the effectiveness and safeness of sirolimus for Systemic Sclerosis by randomized controlled study (sirolimus 2mg/d (N = 36) versus placebo group (N = 36)).

Each SSc patients (n=72) received sirolimus or placebo (active group: placebo group =1:1, 2mg/day， oral administration, (SIR 2mg, po., Qd) .

The end points were the changement of modified RSS and the adverse events or severe adverse events onset.

Inclusion Criteria:

• Meet the American College of Rheumatology criteria for the diagnosis of SSc, 2013.
• Disease duration less than 5 years.
• mRSS was under stable level (>1 month) at the time inclusion.
• Negative urine pregnancy test
• Written informed consent form

Exclusion Criteria:

• Diagnosed with localized scleroderma .
• Added with immunosuppressor in one month such as MTX, AZA, CYC.
• Added with anti-fibosis drug in one month.
• Prednisone >10mg QD before inclusion.
• Sever chronic liver, kidney, lung or heart dysfunction; (heart failure (≥ grade III NYHA), hepatic insufficiency (transaminases> 3N) )
• Serious infection such as bacteremia, sepsis
• Mental disorder or any other chronic illness or drug-abuse that could interfere with the ability to comply with the protocol or to give information
• Cancer or history of cancer cured for less than five years (except in situ carcinoma of the cervix or Basocellular carcinoma)
• Positive HIV test
• Positive urine pregnancy test
• Combined with the other connective tissue diseases