Treatment of immune thrombocytopenia with hydroxychloroquine and TPO-RA

The Efficacy and Safety of Hydroxychloroquine Plus TPO-RA in Antinuclear Antibody-positive Patients With Primary Immune Thrombocytopenia-- The Multicenter, Randomized, Open-labled Clinical Trial

This study is testing if adding hydroxychloroquine to a standard treatment can help adults with immune thrombocytopenia who have certain antibodies feel better and have better platelet levels.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 126 (estimated)
• Ages: 15 Years to 99 Years
• Sex: All
• Sponsor: Shanghai Zhongshan Hospital (Academic / other)
• Drugs / interventions: prednisone
• Locations: 7 sites (Shanghai, Shanghai Municipality and 6 other locations)
• Trial ID: NCT06479291 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the efficacy and safety of hydroxychloroquine (HCQ) combined with thrombopoietin receptor agonists (TPO-RA) in adults diagnosed with primary immune thrombocytopenia (ITP) who also test positive for anti-nuclear antibodies (ANA). Participants will receive TPO-RA daily for up to 24 weeks, with adjustments based on platelet levels, while the study will compare the outcomes of those receiving HCQ plus TPO-RA against those receiving TPO-RA alone. The trial seeks to determine if the combination treatment improves response rates, prolongs response duration, and reduces the necessary dosage of TPO-RA. Additionally, the study will monitor any medical issues arising from the treatment regimen.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 15 and older with a clinical diagnosis of primary immune thrombocytopenia and positive ANA.

Not a fit: Patients who do not have primary immune thrombocytopenia or those with negative ANA results may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a more effective and safer option for patients with immune thrombocytopenia and positive ANA, potentially leading to better management of their condition.

How similar studies have performed: While previous studies have explored HCQ in related conditions, this specific combination of HCQ and TPO-RA for ITP with positive ANA is novel and has not been previously assessed.

Eligibility criteria

Inclusion Criteria:

1. Age is above 15 years old.
2. Before randomization, the clinical diagnosis is primary immune thrombocytopenia. The platelet count is less than 30×10\^9 / L within 1 week before enrollment, or platelet count is less than 50×10\^9 / L with bleeding symptoms within 1 week before enrollment.
3. The antinuclear antibody is positive.
4. Other autoantibodies (mainly including dsDNA antibodies, SSA, SSB, RNP, β 2-GP, ACA, ANCA) are negative.
5. Participants who had received at least two HD-DXM 40 mg/d ×4 d, failed or relapsed, or received standard dose prednisone (1-2 mg/kg/d) for 4 weeks, the platelet count remained \<30×10 9 / L, or the platelet count normalized but decreased with prednisone tappering off, or prednisone 30mg to maintain the platelet number.
6. Prothrombin time does not exceed ± 3s of the normal value ranget, activated partial thrombin time is not outside normal range ± 10s; no history of coagulopathy except ITP.

(6)Understand the study procedures and sign the written informed consent form.

Exclusion Criteria:

1. Secondary thrombocytopenia caused by myelodysplastic syndrome, immune diseases such as systemic lupus erythematosus, early aplastic anemia, atypical reanemia, antiphospholipid syndrome, thrombotic thrombocytopenic purpura and various other causes.
2. The participant has experienced any arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism), or clinical symptoms and medical history indicate thrombophilia.
3. Congestive heart disease, including New York Heart Association (NHYA) Grade III / IV, occurred within 3 months prior to screening, arrhythmia requiring medication or myocardial infarction, or arrhythmia known to increase the risk of thrombotic events (such as atrial fibrillation), or corrected QT interval (QTc) is longer than 450 ms, or QTc\> 480 ms in paricipants with bundle branch block.
4. A medical history of parenchymal organ transplantation or allogeneic bone marrow transplantation.
5. Having received any medication affecting platelet function ( Including but not limited to aspirin, aspirin-containing complexes, clopidogrel, salicylates, and / or non-steroidal anti-inflammatory drugs NSAIDs ) or anticoagulant therapy for over consecutive 3 days within 2 weeks before screening.
6. With Glucose-6-phosphate dehydrogenase deficiency.
7. With retinal or visual field changes caused by 4-aminoquinoline compounds.
8. Being allergic to 4-aminoquinoline compounds.
9. Having evidence of Human Immunodeficiency Virus (HIV)/ hepatitis C virus(HCV)/ hepatitis B virus(HBV) infection (HIV antibody or HCV antibody is positive, HBV surface antigen is positive, or HBV surface antigen is negative but HBV-DNA indicating viral replication.
10. Glutamate transaminotransferase (ALT) or glutamate transaminase (AST) is higher than 1.5 times the upper limit of normal value (ULN), or total bilirubin or blood creatinine is higher than 1.2 times the ULN.
11. With liver cirrhosis or portal hypertension.
12. With evidence of malignant tumor activity, or receiving anti-tumor treatment within 5 years prior to the screening.
13. Participants being pregnant or lactating, or with potential fertility, reluctance to use effective contraception within the entire trial cycle and within 28 days after the end of the trial (or within 28 days after premature withdraw).

Where this trial is running

Shanghai, Shanghai Municipality and 6 other locations

• Shanghai Zhongshan Hospital — Shanghai, Shanghai Municipality, China (Recruiting)
• Wusong Hospital, Zhongshan Hospital, Fudan University — Shanghai, Shanghai Municipality, China (Recruiting)
• Shanghai Jinshan Hospital — Shanghai, Shanghai Municipality, China (Recruiting)
• Qingpu Branch of Zhongshan Hospital, Fudan University — Shanghai, Shanghai Municipality, China (Recruiting)
• Health and Humanity Research Centre, Hongkong, China. — Hong Kong, Hong Kong (Recruiting)
• Dr. Stanley Ho Medical Foundation — Macao, Macau (Recruiting)
• University Hospital, Macau University of Science and Technology. — Macao, Macau (Recruiting)

Study contacts

• Principal investigator: Yunfeng Cheng — Shanghai Zhongshan Hospital
• Study coordinator: Lili Ji

Conditions Immune Thrombocytopenia With Positive ANA Antibodies