A Pilot-Study of Sirolimus for the Treatment of Systemic Sclerosis
- Registration Number
- NCT03365869
- Lead Sponsor
- Peking University People's Hospital
- Brief Summary
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
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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 72
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description placebo Sirolimus sirolimus placebo: 2mg po. QD Sirolimus Sirolimus Add sirolimus according to the protocol. Sirolimus active: 2mg po. QD
- Primary Outcome Measures
Name Time Method Number of participants whose modified Rodnan skin score （mRSS）decreasing week 48 mRSS was evaluated in 17 sites of skin.
- Secondary Outcome Measures
Name Time Method The number of adverse event and severe adverse event occured week 48 SAE were recorded as life-threatening and others were AE.