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

Study Overview
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Brief Summary
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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)).

Detailed Description
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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.

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

Conditions
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Systemic Sclerosis

Intervention / Treatment
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* Drug: Sirolimus


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Sirolimus<br>Add sirolimus according to the protocol. Sirolimus active: 2mg po. QD | Drug: Sirolimus<br>* Sirolimus or placebo were added to patients every day<br>* Other names: placebo;|
| Placebo Comparator: placebo<br>sirolimus placebo: 2mg po. QD | Drug: Sirolimus<br>* Sirolimus or placebo were added to patients every day<br>* Other names: placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants whose modified Rodnan skin score （mRSS）decreasing | mRSS was evaluated in 17 sites of skin. | week 48 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The number of adverse event and severe adverse event occured | SAE were recorded as life-threatening and others were AE. | week 48 |