RecruitingPhase 4NCT06722586

Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia

Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia: a Multicenter Randomized, Double Blind, Placebo Controlled, Clinical Trial


Sponsor

Peking University First Hospital

Enrollment

84 participants

Start Date

Jan 7, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

The goal of this clinical trial is to evaluate the efficacy and safety of sirolimus in patients with anti-phospholipid antibody-associated thrombocytopenia. In the 6-month randomized, double-blind, placebo-controlled phase, participants will receive either sirolimus 1 mg once daily or matching placebo. Participants will be followed at 2 weeks, 1 month, 3 months, and 6 months after enrollment. Participants who do not achieve the primary endpoint at 6 months may enter a 3-month open-label extension and receive sirolimus 1.5 mg once daily, with follow-up through month 9. The main question is whether sirolimus improves the overall response rate at 6 months compared with placebo. Primary outcome: \- Overall response rate at 6 months Secondary outcomes: * Overall response rate at 1 month and 3 months * Complete response rate at 6 months * Partial response rate at 6 months * Change in anti-phospholipid antibody titers at 6 months * Change in oral glucocorticoid dosage at 6 months Exploratory outcomes: * Proportion and reasons for early discontinuation during the double-blind phase * Among participants who do not achieve the primary endpoint at 6 months and enter the open-label extension, overall response rate, complete response rate, partial response rate, and safety outcomes after 3 months of open-label treatment


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Placebo and a drug called Sirolimus for people with antiphospholipid (apl)-positive and thrombocytopaenia. The study is currently recruiting participants at 10 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGSirolimus

Sirolimus 1 mg (two pills) once daily during the 6-month double-blind phase; sirolimus 1.5 mg once daily during the optional 3-month open-label extension for eligible participants.

DRUGPlacebo

Matching placebo two pills once daily during the 6-month double-blind phase; sirolimus 1.5 mg once daily during the optional 3-month open-label extension for eligible participants.


Locations(10)

Xiangya Hospital of Central South University

Changsha, Hunan, China

Qilu Hospital of Shandong University

Jinan, Shangdong, China

West China Hospital, Sichuan University

Chengdu, Sichuan, China

People's Hospital of Xinjiang Uygur Autonomous Region

Ürümqi, Xinjiang, China

The 1st Affiliated Hospital of Wenzhou Medical University

Wenzhou, Zhejiang, China

Beijing Chao-Yang Hospital

Beijing, China

Peking University First Hospital

Beijing, China

Peking University Third Hospital

Beijing, China

Beijing Shijitan Hospital

Beijing, China

Shanghai Renji Hospital

Shanghai, China

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT06722586


Related Trials