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

NCT ID: NCT03365869

Last Updated: 2017-12-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

72 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-06-01

Study Completion Date

2019-06-30

Brief Summary

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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

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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.

Conditions

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Systemic Sclerosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Participants

Study Groups

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Sirolimus

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

Group Type ACTIVE_COMPARATOR

Sirolimus

Intervention Type DRUG

Sirolimus or placebo were added to patients every day

placebo

sirolimus placebo: 2mg po. QD

Group Type PLACEBO_COMPARATOR

Sirolimus

Intervention Type DRUG

Sirolimus or placebo were added to patients every day

Interventions

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Sirolimus

Sirolimus or placebo were added to patients every day

Intervention Type DRUG

Other Intervention Names

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placebo

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Peking University People's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Mu Rong, Poster

Role: PRINCIPAL_INVESTIGATOR

Peking University Institute of Rheumatology and Immunology

Central Contacts

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Wuri Liga, Master

Role: CONTACT

Phone: +8618801231167

Email: [email protected]

Other Identifiers

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PKUPH-R-SRL

Identifier Type: -

Identifier Source: org_study_id