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

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ClinicalTrials.gov Identifier: NCT03365869
Recruitment Status : Not yet recruiting
First Posted : December 7, 2017
Last Update Posted : December 7, 2017
Sponsor:
Information provided by (Responsible Party):
Peking University People's Hospital

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)).


Condition or disease Intervention/treatment Phase
Systemic Sclerosis Drug: Sirolimus Phase 2

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  (Clinical Trial)
Estimated Enrollment : 72 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Official Title: A Phase Ⅱ Pilot-Study With Sirolimus for the Treatment of Systemic Sclerosis
Estimated Study Start Date : June 1, 2018
Estimated Primary Completion Date : December 31, 2018
Estimated Study Completion Date : June 30, 2019


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 Name: placebo

Placebo Comparator: placebo
sirolimus placebo: 2mg po. QD
Drug: Sirolimus
Sirolimus or placebo were added to patients every day
Other Name: placebo




Primary Outcome Measures :
  1. Number of participants whose modified Rodnan skin score (mRSS)decreasing [ Time Frame: week 48 ]
    mRSS was evaluated in 17 sites of skin.


Secondary Outcome Measures :
  1. The number of adverse event and severe adverse event occured [ Time Frame: week 48 ]
    SAE were recorded as life-threatening and others were AE.



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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03365869


Contacts
Contact: Wuri Liga, Master +8618801231167 murongster@163.com

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

Responsible Party: Peking University People's Hospital
ClinicalTrials.gov Identifier: NCT03365869     History of Changes
Other Study ID Numbers: PKUPH-R-SRL
First Posted: December 7, 2017    Key Record Dates
Last Update Posted: December 7, 2017
Last Verified: December 2017

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

Additional relevant MeSH terms:
Scleroderma, Systemic
Scleroderma, Diffuse
Sclerosis
Pathologic Processes
Connective Tissue Diseases
Skin Diseases
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs