Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune Thrombocytopenia

NCT07657793 | Recruiting Phase 2 DMC

• 1 other identifier: SiroinSLEITP
• Study Type: interventional
• Target: 164
• Locations: 1 country
• Sites: 1

Brief Summary

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. 1.Does sirolimus increase the overall response rate (complete or partial remission of thrombocytopenia) at 24 weeks compared with placebo?
2. 2.The safety issues when taking sirolimus over 24 weeks (Phase 1) and up to 48 weeks (Phase 2).
3. 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.
4. 4.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)
5. 5.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
6. 6.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
7. 7.Receive telephone follow-ups at weeks 8, 16, 20, 32, 40, and 44 to report platelet counts, medication adherence, and adverse events

Conditions

Systemic Lupus Erythematosus; Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

Sirolimus

EXPERIMENTAL

Drug: Sirolimus

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Sirolimus DRUG

Sirolimus 1.5mg/day + prednisone + hydroxychloroquine

Sirolimus

Placebo DRUG

Placebo + prednisone + hydroxychloroquine

Placebo

Eligibility Criteria

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

You may qualify if:

• Age ≥ 18 and \< 65 years
• Meet 2012 SLICC or 2019 EULAR/ACR criteria for systemic lupus erythematosus
• Meet ITP diagnostic criteria: platelet count ≤ 30×10\^9/L, or ≤ 50×10\^9/L with bleeding tendency
• At least two CBC tests showing thrombocytopenia
• Bone marrow smear showing megakaryocyte maturation disorder
• Willing to sign informed consent form

You may not qualify if:

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

Sponsors & Collaborators

• Chinese SLE Treatment And Research Group: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Purpura, Thrombocytopenic, Idiopathic

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Central Study Contacts

Liying Peng

CONTACT

86 010 69159958; liying_peng@qq.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: SPONSOR

Study Record Dates

• First Submitted: June 15, 2026
• First Posted: June 18, 2026
• Study Start: June 7, 2026
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): September 30, 2029
• Last Updated: June 18, 2026

Record last verified: 2026-06

Locations

CN (1)