Treatment of immune thrombocytopenia with hydroxychloroquine and prednisone

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

This study is testing if a combination of hydroxychloroquine and prednisone can help adults with immune thrombocytopenia feel better and have longer-lasting results than just using prednisone alone.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 129 (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: NCT06479304 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the efficacy and safety of hydroxychloroquine (HCQ) combined with prednisone (Pred) in treating adults with primary immune thrombocytopenia (ITP) who test positive for anti-nuclear antibodies (ANA). Participants will receive Pred daily for six weeks, with the option of taking HCQ twice daily for one year. The study will assess whether the combination treatment improves response rates and prolongs response duration compared to Pred alone, while also monitoring any associated medical issues. Regular clinic visits and symptom diaries will be part of the participant's involvement.

Who should consider this trial

Good fit: Ideal candidates include adults over 15 years old with primary immune thrombocytopenia, a platelet count below 30×10^9/L, and positive ANA.

Not a fit: Patients with secondary thrombocytopenia due to conditions like myelodysplastic syndrome or systemic lupus erythematosus may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a more effective long-term management option for patients with ITP and positive ANA.

How similar studies have performed: Previous studies have shown promising results for HCQ in treating chronic ITP, indicating potential for success with this combination approach.

Eligibility criteria

Inclusion Criteria:

1. Age is above 75 years old, or participants with uncontrolled hypertension and diabetes mellitus at the age between 15-75 years old, gender is unlimited.
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. 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. With severe hemorrhage (intracranial hemorrhage) or coagulation dysfunction (INR and APTT\> 125% upper limit of normal).
5. With severe digestive tract diseases affecting drug absorption.
6. With serious mental illness patient.
7. Having participated in other clinical trials within 3 months prior to screening.
8. Having received any immunomodulatory medication for other diseases 3 months before screening.
9. 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.
10. With Glucose-6-phosphate dehydrogenase deficiency.
11. With retinal or visual field changes caused by 4-aminoquinoline compounds.
12. Being allergic to 4-aminoquinoline compounds.
13. 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.
14. 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.
15. With liver cirrhosis or portal hypertension.
16. With evidence of malignant tumor activity, or receiving anti-tumor treatment within 5 years prior to the screening.
17. Addicted to alcohol or drugs.
18. 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)
• Zhongshan Wusong Hospital, Fudan University — Shanghai, Shanghai Municipality, China (Recruiting)
• Shanghai Jinshan Hospital — Shanghai, Shanghai Municipality, China (Recruiting)
• Zhongshan Qingpu 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