Systemic Sclerosis
Conditions
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.
Interventions
DRUGSirolimus
Sirolimus or placebo were added to patients every day
Sponsors
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants whose modified Rodnan skin score （mRSS）decreasing | week 48 | mRSS was evaluated in 17 sites of skin. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The number of adverse event and severe adverse event occured | week 48 | SAE were recorded as life-threatening and others were AE. |
Contacts
Primary ContactWuri Liga, Master
Outcome results
None listed