The Safety and Efficiency of Sirolimus in Primary Antiphospholipid Syndrome: a Randomized Control Study

NCT06504420 | Not Yet Recruiting Phase 2

• 1 other identifier: 20240125
• Study Type: interventional
• Target: 70
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Primary Antiphospholipid Syndrome

Keywords

APS

Outcome Measures

Primary Outcomes (2)

• 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）.

  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).

  week 24

Secondary Outcomes (3)

• Complete response(CR) rate at week 48

  week 48

• Partial response (PR) rate at week 48

  week 48

• Rate of Participants with adverse effects and serious adverse effects during treatment

  baseline to week 48

Study Arms (2)

Sirolimus

EXPERIMENTAL

Sirolimus 1.5mg po. QD

Drug: Sirolimus

placeobo

PLACEBO COMPARATOR

Sirolimus placebo 1.5mg po. QD

Drug: Placebo

Interventions

Sirolimus DRUG

Subjects will receive Sirolimus 1.5mg/d for 48 weeks in addition to their ongoing APS treatment regimen

Sirolimus

Placebo DRUG

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

placeobo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)
• 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

Sponsors & Collaborators

• Peking University People's Hospital: lead

MeSH Terms

Interventions

Sirolimus

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Zhanguo Li

  Peking university peoples hospital

  STUDY DIRECTOR

Central Study Contacts

Liling Xu, Ph.D

CONTACT

+86 010 88324173; xuliling1079@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Department of Rheumatology and immunology

Study Record Dates

• First Submitted: July 11, 2024
• First Posted: July 16, 2024
• Study Start: October 15, 2024
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): December 30, 2027
• Last Updated: September 19, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share