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

Systemic Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT03365869
• Other study ID #: PKUPH-R-SRL
• Status: Not yet recruiting
• Phase: Phase 2
• First received: December 4, 2017
• Last updated: December 4, 2017
• Start date: June 1, 2018
• Est. completion date: June 30, 2019

Study information

• Verified date: December 2017
• Source: Peking University People's Hospital
• Contact: Wuri Liga, Master
• Phone: +8618801231167
• Email: murongster[@]163.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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)).

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 information / eligibility

• Status: Not yet recruiting
• Enrollment: 72
• Est. completion date: June 30, 2019
• Est. primary completion date: December 31, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Scleroderma, Diffuse
• Scleroderma, Systemic
• Sclerosis
• Systemic Sclerosis

Intervention

Drug:
• Sirolimus
Sirolimus or placebo were added to patients every day

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Peking University People's Hospital

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants whose modified Rodnan skin score (mRSS)decreasing	mRSS was evaluated in 17 sites of skin.	week 48
Secondary	The number of adverse event and severe adverse event occured	SAE were recorded as life-threatening and others were AE.	week 48