Primary Antiphospholipid SyndromePhase 2NCT06504420
The Safety and Efficiency of Sirolimus in Primary Antiphospholipid Syndrome: a Randomized Control Study
A Phase 2 study testing Sirolimus in people with Primary Antiphospholipid Syndrome, run by Peking University People's Hospital.
Trial summary
This study is an Investigator initiated, randomized, multicenter, double-blind, placebo-control study. The aim of this study is to evaluate the safety and efficiency of Sirolimus for primaty antiphospholipid syndrome patients at week 24 and week 48. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Primary Antiphospholipid Syndrome
- What is being tested?
- Sirolimus
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- No sites listed yet
- Study dates
- 15 Oct 2024to 30 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Understand and sign the informed consent form
- Male or Female
- aged 18-70 at the time of screening visit
- Met 2006 Sapporo classification criteria of APS or 2023 ACR/EULAR classification criteria of APS
- With the stable combination therapy
Who cannot join
- history of serious adverse events or contraindication to Sirolimus
- Catastrophic APS within 90 days
- Acute thrombosis within 30 days
- ≥4/11 American College of Rheumatology Classification Criteria for SLE or other systemic autoimmune diseases
- Historically positive HIV test or test positive at screening for HIV
- currently on any suppressive therapy for a chronic infection (such as tuberculosis, hepatitis B infection, hepatitis C infection, CMV, EBV, Syphilis, etc)
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- Surgery treatment within one month
- History of malignant neoplasm within the last 5 years
- White blood cell counts<3×10*9/L
- Abnormal Liver function tests: ALT or AST ≥ 1.5 times the upper limit of the normal value, and total bilirubin and blood lipids ≥ 2 times the upper limit of the normal value
- Pregnant or pregnancy preparation or breastfeed
- Any circumstances that may cause the subjects to be unable to complete the study or pose significant risks to the subjects
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Randomized, Multicenter, Double-blind, Placeobo-control Study of Sirolimus for Primary Antiphospholipid Syndrome Patients
This study is an Investigator initiated, randomized, multicenter, double-blind, placebo-control study. The aim of this study is to evaluate the safety and efficiency of Sirolimus for primaty antiphospholipid syndrome patients at week 24 and week 48.
- Conditions
- Primary Antiphospholipid Syndrome
- Being tested
- Sirolimus, Placebo
- Keywords
- APS
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Subjects will receive Sirolimus 1.5mg/d for 48 weeks in addition to their ongoing APS treatment regimen
PlaceboDrug
Subjects will receive Placebo 1.5mg/d for first 24 weeks and Sirolimus 1.5mg/d for next 24 weeks in addition to their ongoing APS treatment regimen
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus | Experimental | Sirolimus 1.5mg po. QD Receives: Drug: Sirolimus |
| placeobo | Placebo comparator | Sirolimus placebo 1.5mg po. QD Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete response(CR) rate at week 24
CR will be defined as follows: 1.No thrombosis events; 2.PLT ≥100 and with nobleeding; 3. No persistent autoimmnune haemolytic anaemia; 4. No skin ulcer; 5.No renal thrombotic microangiopathy; 5.Normal cognitive function (MoCA score ≥26）.
Measured: week 24
Partial response (PR) rate at week 24
PR will be defined as one of follows: 1.superficial thrombotic events;2. PLT ≥30 or increased at least 2 times of baseline and with no bleeding;3.haemoglobulin concentration normal or increased 20g/L compared to baseline; 4.50% improvement; 5. a serum creatinine level 15% abov baseline, RBCs per high-power field 50% above baseline with no casts, 50% improvement in the urinary prt:cr; 6. milde cognitive diysfunction (MoCA 9~25).
Measured: week 24
Secondary outcomes (3)
Complete response(CR) rate at week 48
CR will be defined as follows: 1.No thrombosis events; 2.PLT ≥100 and with nobleeding; 3. No persistent autoimmnune haemolytic anaemia; 4. No skin ulcer; 5.No renal thrombotic microangiopathy; 5.Normal cognitive function (MoCA score ≥26）.
Measured: week 48
Partial response (PR) rate at week 48
PR will be defined as one of follows: 1.superficial thrombotic events;2. PLT ≥30 or increased at least 2 times of baseline and with no bleeding;3.haemoglobulin concentration normal or increased 20g/L compared to baseline; 4.50% improvement; 5. a serum creatinine level 15% abov baseline, RBCs per high-power field 50% above baseline with no casts, 50% improvement in the urinary prt:cr; 6. milde cognitive diysfunction (MoCA 9~25).
Measured: week 48
Rate of Participants with adverse effects and serious adverse effects during treatment
Adverse effects include fever, rash, abnormal liver function, rate of new-onset infections and any abnormal measures after recivede study drug.
Measured: baseline to week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2024
- Study start15 Oct 2024 (estimated)
- Primary completion30 Dec 2026 (estimated)
- Study completion30 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Antiphospholipid SyndromeStudies 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
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhanguo Li, Peking University People's Hospital
- Investigators
- Zhanguo Li, Study director, Peking university peoples hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06504420
- Sponsor's protocol ID
- 20240125
- Status last verified
- Jul 2024
- First submitted
- 11 Jul 2024
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Multicenter, Double-blind, Placeobo-control Study of Sirolimus for Primary Antiphospholipid Syndrome Patients
- ClinicalTrials.gov ID
- NCT06504420
- Acronym / sponsor ID
- 20240125
- Conditions
- Primary Antiphospholipid Syndrome
- Keywords
- APS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 15 Oct 2024 (estimated)
- Primary completion
- 30 Dec 2026 (estimated)
- Study completion
- 30 Dec 2027 (estimated)
- First posted
- 16 Jul 2024 (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.
This study is an Investigator initiated, randomized, multicenter, double-blind, placebo-control study. The aim of this study is to evaluate the safety and efficiency of Sirolimus for primaty antiphospholipid syndrome patients at week 24 and week 48.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sirolimus | Experimental | Drug: SirolimusSirolimus 1.5mg po. QD |
| placeobo | Placebo comparator | Drug: PlaceboSirolimus placebo 1.5mg po. QD |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Understand and sign the informed consent form
- Male or Female
- aged 18-70 at the time of screening visit
- Met 2006 Sapporo classification criteria of APS or 2023 ACR/EULAR classification criteria of APS
- With the stable combination therapy
Key exclusion criteria (12 listed)
- history of serious adverse events or contraindication to Sirolimus
- Catastrophic APS within 90 days
- Acute thrombosis within 30 days
- ≥4/11 American College of Rheumatology Classification Criteria for SLE or other systemic autoimmune diseases
- Historically positive HIV test or test positive at screening for HIV
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 |
|---|---|---|
| SirolimusSubjects will receive Sirolimus 1.5mg/d for 48 weeks in addition to their ongoing APS treatment regimen | Drug | Sirolimus |
| PlaceboSubjects will receive Placebo 1.5mg/d for first 24 weeks and Sirolimus 1.5mg/d for next 24 weeks in addition to their ongoing APS treatment regimen | Drug | placeobo |
Primary outcome
Complete response(CR) rate at week 24
Time frame: week 24
CR will be defined as follows: 1.No thrombosis events; 2.PLT ≥100 and with nobleeding; 3. No persistent autoimmnune haemolytic anaemia; 4. No skin ulcer; 5.No renal thrombotic microangiopathy; 5.Normal cognitive function (MoCA score ≥26）.
Partial response (PR) rate at week 24
Time frame: week 24
PR will be defined as one of follows: 1.superficial thrombotic events;2. PLT ≥30 or increased at least 2 times of baseline and with no bleeding;3.haemoglobulin concentration normal or increased 20g/L compared to baseline; 4.50% improvement; 5. a serum creatinine level 15% abov baseline, RBCs per high-power field 50% above baseline with no casts, 50% improvement in the urinary prt:cr; 6. milde cognitive diysfunction (MoCA 9~25).
3 secondary outcomes
Complete response(CR) rate at week 48
Time frame: week 48
Partial response (PR) rate at week 48
Time frame: week 48
Rate of Participants with adverse effects and serious adverse effects during treatment
Time frame: baseline to week 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Individual participant dataSharing planned: No
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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.
Zhanguo Li study director
Peking university peoples hospital
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sirolimus in people with Primary Antiphospholipid Syndrome, 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 12 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: opening soon, as of the sponsor's last update on 19 Sep 2024. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Liling Xu, Ph.D (+86 010 88324173, xuliling1079@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete response(CR) rate at week 24 (time frame: week 24). The study lists 2 primary outcomes in all. 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 19 Sep 2024.