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RECRUITING
NCT06722586
PHASE4

Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia

Sponsor: Peking University First Hospital

View on ClinicalTrials.gov

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

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

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

84

Start Date

2025-01-07

Completion Date

2027-12

Last Updated

2026-06-30

Healthy Volunteers

No

Interventions

DRUG

Sirolimus

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.

DRUG

Placebo

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