Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune Thrombocytopenia

A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Clinical Trial and Open-Label Extension Study of Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune Thrombocytopenia

The goal of this clinical trial is to learn if sirolimus, added to standard background therapy (corticosteroids and hydroxychloroquine), works to treat systemic lupus erythematosus-associated immune thrombocytopenia (SLE-ITP) in adults aged 18 to 65 years. The study also aims to evaluate the long-term effectiveness and safety of sirolimus over 48 weeks.

The main questions it aims to answer are:

1. Does sirolimus increase the overall response rate (complete or partial remission of thrombocytopenia) at 24 weeks compared with placebo?
2. The safety issues when taking sirolimus over 24 weeks (Phase 1) and up to 48 weeks (Phase 2).
3. How long does the treatment effect last, and what is the relapse rate during open-label extension? In Phase 1 (first 24 weeks), researchers will compare sirolimus to a placebo (an identical-looking capsule containing no active drug) to see if sirolimus works to treat SLE-ITP when both groups also receive standardized prednisone and hydroxychloroquine. In Phase 2 (weeks 24 to 48), all participants will receive open-label sirolimus to assess long-term efficacy and safety.

Participants will:

1. Phase 1: Take sirolimus (1.5 mg once daily) or a placebo for 24 weeks, plus standardized prednisone (tapered according to protocol) and hydroxychloroquine (0.4 g daily)
2. Phase 2: After completing Phase 1, take open-label sirolimus (1.5 mg once daily) for an additional 24 weeks, continuing stable or tapering doses of prednisone and hydroxychloroquine
3. Visit the clinic at screening, baseline, weeks 4, 12, 24 (end of Phase 1), and then at weeks 28, 36, and 48 (end of Phase 2) for checkups, blood tests, and disease activity assessments
4. Receive telephone follow-ups at weeks 8, 16, 20, 32, 40, and 44 to report platelet counts, medication adherence, and adverse events

Study Overview

• Status: Recruiting
• Conditions: Systemic Lupus Erythematosus; Immune Thrombocytopenia
• Intervention / Treatment: Drug: Sirolimus; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 164
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Liying Peng; Phone Number: 86 010 69159958; Email: liying_peng@qq.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100730
      • Recruiting
      • Peking Union Medical College Hospital
      • Contact: Liying Peng; Email: liying_peng@qq.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Age ≥ 18 and < 65 years

1. Meet 2012 SLICC or 2019 EULAR/ACR criteria for systemic lupus erythematosus
2. Meet ITP diagnostic criteria: platelet count ≤ 30×10^9/L, or ≤ 50×10^9/L with bleeding tendency
3. At least two CBC tests showing thrombocytopenia
4. Bone marrow smear showing megakaryocyte maturation disorder
5. Willing to sign informed consent form

Exclusion Criteria:

1. Other causes of thrombocytopenia
2. Refractory SLE-ITP (no partial response after 3 months of high-dose steroids or immunosuppressants)
3. Use of rituximab within 6 months
4. Severe organ involvement (e.g., proteinuria > 1g/24h, CNS involvement, cardiac/GI involvement)
5. Other autoimmune diseases (except Sjögren's syndrome)
6. Malignancy
7. Active infections (HIV, HBV, HCV, syphilis)
8. Severe liver or kidney dysfunction
9. Splenomegaly
10. Pregnancy or breastfeeding
11. Contraindications to study drugs
12. Unhealed major wounds or incisions
13. Judged unsuitable by investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo + prednisone + hydroxychloroquine
Experimental: Sirolimus	Drug: Sirolimus; Sirolimus 1.5mg/day + prednisone + hydroxychloroquine

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate (complete response + partial response) of ITP at Week 24	Complete response: platelet count ≥ 100×10^9/L with no overt bleeding Partial response: platelet count ≥ 30×10^9/L and doubled from baseline, with no overt bleeding	Week 24

Collaborators and Investigators

• Sponsor: Chinese SLE Treatment And Research Group

Study record dates

Study Major Dates

• Study Start (Actual): June 7, 2026
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): September 30, 2029

Study Registration Dates

• First Submitted: June 15, 2026
• First Submitted That Met QC Criteria: June 15, 2026
• First Posted (Actual): June 18, 2026

Study Record Updates

• Last Update Posted (Actual): June 18, 2026
• Last Update Submitted That Met QC Criteria: June 15, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Skin and Connective Tissue Diseases; Signs and Symptoms; Hemic and Lymphatic Diseases; Lupus Erythematosus, Systemic; Purpura, Thrombocytopenic, Idiopathic; Organic Chemicals; Macrolides; Lactones; Sirolimus
• Other Study ID Numbers: SiroinSLEITP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No