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

Peking University People's Hospital72 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Systemic Sclerosis

Interventions

SirolimusDRUG

Sirolimus or placebo were added to patients every day

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Number of participants whose modified Rodnan skin score （mRSS）decreasing

mRSS was evaluated in 17 sites of skin.

Time frame: week 48

Secondary Outcomes

The number of adverse event and severe adverse event occured

SAE were recorded as life-threatening and others were AE.

Time frame: week 48

Central Contacts

Wuri Liga, Master

CONTACT

+8618801231167 murongster@163.com

Data from ClinicalTrials.gov

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