Systemic SclerosisPhase 2NCT03365869
A Pilot-Study of Sirolimus for the Treatment of Systemic Sclerosis
A Phase 2 study testing Sirolimus in people with Systemic Sclerosis, run by Peking University People's Hospital.
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… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Systemic Sclerosis
- What is being tested?
- Sirolimus
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 72 participants
- Where
- No sites listed yet
- Study dates
- 1 Jun 2018to 30 Jun 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
No sites are listed yet.
About this study
Official title: 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)).
Read the 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.
- Conditions
- Systemic Sclerosis
- Being tested
- Sirolimus
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called placebo
Sirolimus or placebo were added to patients every day
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus | Active comparator | Add sirolimus according to the protocol. Sirolimus active: 2mg po. QD Receives: Drug: Sirolimus |
| placebo | Placebo comparator | sirolimus placebo: 2mg po. QD Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants whose modified Rodnan skin score （mRSS）decreasing
mRSS was evaluated in 17 sites of skin.
Measured: week 48
Secondary outcomes (1)
The number of adverse event and severe adverse event occured
SAE were recorded as life-threatening and others were AE.
Measured: week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2017
- Study start1 Jun 2018 (estimated)
- Primary completion31 Dec 2018 (estimated)
- Study completion30 Jun 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mu Rong, Poster, Principal investigator, Peking University Institute of Rheumatology and Immunology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03365869
- Sponsor's protocol ID
- PKUPH-R-SRL
- Status last verified
- Dec 2017
- First submitted
- 4 Dec 2017
- Last updated
- 7 Dec 2017
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ⅱ Pilot-Study With Sirolimus for the Treatment of Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT03365869
- Acronym / sponsor ID
- PKUPH-R-SRL
- Conditions
- Systemic Sclerosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 1 Jun 2018 (estimated)
- Primary completion
- 31 Dec 2018 (estimated)
- Study completion
- 30 Jun 2019 (estimated)
- First posted
- 7 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sirolimus | Active comparator | Drug: SirolimusAdd sirolimus according to the protocol. Sirolimus active: 2mg po. QD |
| placebo | Placebo comparator | Drug: Sirolimussirolimus placebo: 2mg po. QD |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (11 listed)
- 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) )
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SirolimusAlso: placeboSirolimus or placebo were added to patients every day | Drug | Sirolimus, placebo |
Primary outcome
Number of participants whose modified Rodnan skin score （mRSS）decreasing
Time frame: week 48
mRSS was evaluated in 17 sites of skin.
1 secondary outcome
The number of adverse event and severe adverse event occured
Time frame: week 48
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Dec 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mu Rong, Poster principal investigator
Peking University Institute of Rheumatology and Immunology
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sirolimus in people with Systemic Sclerosis, run by Peking University People's Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Wuri Liga, Master (+8618801231167, murongster@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants whose modified Rodnan skin score （mRSS）decreasing (time frame: week 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Dec 2017.