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

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

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)).

Study Overview

• Status: Unknown
• Conditions: Systemic Sclerosis
• Intervention / Treatment: Drug: Sirolimus

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.

• Study Type: Interventional
• Enrollment (Anticipated): 72
• Phase: Phase 2

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Sirolimus; Add sirolimus according to the protocol. Sirolimus active: 2mg po. QD	Drug: Sirolimus; Sirolimus or placebo were added to patients every day; Other Names: placebo
Placebo Comparator: placebo; sirolimus placebo: 2mg po. QD	Drug: Sirolimus; Sirolimus or placebo were added to patients every day; Other Names: placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants whose modified Rodnan skin score （mRSS）decreasing	mRSS was evaluated in 17 sites of skin.	week 48

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of adverse event and severe adverse event occured	SAE were recorded as life-threatening and others were AE.	week 48

Collaborators and Investigators

• Sponsor: Peking University People's Hospital
• Investigators: Principal Investigator: Mu Rong, Poster, Peking University Institute of Rheumatology and Immunology

Study record dates

Study Major Dates

• Study Start (Anticipated): June 1, 2018
• Primary Completion (Anticipated): December 31, 2018
• Study Completion (Anticipated): June 30, 2019

Study Registration Dates

• First Submitted: December 4, 2017
• First Submitted That Met QC Criteria: December 4, 2017
• First Posted (Actual): December 7, 2017

Study Record Updates

• Last Update Posted (Actual): December 7, 2017
• Last Update Submitted That Met QC Criteria: December 4, 2017
• Last Verified: December 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Skin Diseases; Connective Tissue Diseases; Sclerosis; Scleroderma, Systemic; Scleroderma, Diffuse; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antifungal Agents; Sirolimus
• Other Study ID Numbers: PKUPH-R-SRL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No