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Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia

NCT06722586 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 84

Last updated 2026-06-30

No results posted yet for this study

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

Conditions

  • Antiphospholipid (aPL)-Positive
  • Thrombocytopaenia

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.

Sponsors & Collaborators

  • North China Pharmaceutical Co.,Ltd

    collaborator UNKNOWN

  • Peking University First Hospital

    lead OTHER

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-01-07
Primary Completion
2027-12-31
Completion
2027-12-31

Countries

  • China

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06722586 on ClinicalTrials.gov