A Phase Ⅱ Pilot-Study With Sirolimus for the Treatment of Systemic Sclerosis
Overview
- Phase
- Phase 2
- Intervention
- Sirolimus
- Conditions
- Systemic Sclerosis
- Sponsor
- Peking University People's Hospital
- Enrollment
- 72
- Primary Endpoint
- Number of participants whose modified Rodnan skin score （mRSS）decreasing
- Last Updated
- 8 years ago
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Meet the American College of Rheumatology criteria for the diagnosis of SSc,
- •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
Arms & Interventions
Sirolimus
Add sirolimus according to the protocol. Sirolimus active: 2mg po. QD
Intervention: Sirolimus
placebo
sirolimus placebo: 2mg po. QD
Intervention: Sirolimus
Outcomes
Primary Outcomes
Number of participants whose modified Rodnan skin score （mRSS）decreasing
Time Frame: week 48
mRSS was evaluated in 17 sites of skin.
Secondary Outcomes
- The number of adverse event and severe adverse event occured(week 48)