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

Primary Purpose

Systemic Sclerosis

Status
Unknown status
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Sirolimus
Sponsored by
Peking University People's Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Systemic Sclerosis

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Active Comparator

    Placebo Comparator

    Arm Label

    Sirolimus

    placebo

    Arm Description

    Add sirolimus according to the protocol. Sirolimus active: 2mg po. QD

    sirolimus placebo: 2mg po. QD

    Outcomes

    Primary Outcome Measures

    Number of participants whose modified Rodnan skin score （mRSS）decreasing
    mRSS was evaluated in 17 sites of skin.

    Secondary Outcome Measures

    The number of adverse event and severe adverse event occured
    SAE were recorded as life-threatening and others were AE.

    Full Information

    First Posted
    December 4, 2017
    Last Updated
    December 4, 2017
    Sponsor
    Peking University People's Hospital
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03365869
    Brief Title
    A Pilot-Study of Sirolimus for the Treatment of Systemic Sclerosis
    Official Title
    A Phase Ⅱ Pilot-Study With Sirolimus for the Treatment of Systemic Sclerosis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    December 2017
    Overall Recruitment Status
    Unknown status
    Study Start Date
    June 1, 2018 (Anticipated)
    Primary Completion Date
    December 31, 2018 (Anticipated)
    Study Completion Date
    June 30, 2019 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Peking University People's Hospital

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Systemic Sclerosis

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Parallel Assignment
    Masking
    Participant
    Allocation
    Randomized
    Enrollment
    72 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Sirolimus
    Arm Type
    Active Comparator
    Arm Description
    Add sirolimus according to the protocol. Sirolimus active: 2mg po. QD
    Arm Title
    placebo
    Arm Type
    Placebo Comparator
    Arm Description
    sirolimus placebo: 2mg po. QD
    Intervention Type
    Drug
    Intervention Name(s)
    Sirolimus
    Other Intervention Name(s)
    placebo
    Intervention Description
    Sirolimus or placebo were added to patients every day
    Primary Outcome Measure Information:
    Title
    Number of participants whose modified Rodnan skin score （mRSS）decreasing
    Description
    mRSS was evaluated in 17 sites of skin.
    Time Frame
    week 48
    Secondary Outcome Measure Information:
    Title
    The number of adverse event and severe adverse event occured
    Description
    SAE were recorded as life-threatening and others were AE.
    Time Frame
    week 48

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    80 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Wuri Liga, Master
    Phone
    +8618801231167
    Email
    murongster@163.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Mu Rong, Poster
    Organizational Affiliation
    Peking University Institute of Rheumatology and Immunology
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Learn more about this trial

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

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