The Efficacy and Safety of HCQ Plus TPO-RA in ANA Positive ITP
NCT Number: NCT06479291
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: NA
Conditions: Immune Thrombocytopenia With Positive ANA Antibodies
Interventions: DRUG: Hydroxychloroquine Oral Tablet|DRUG: Eltrombopag
Sponsor: Yunfeng Cheng
Collaborators: Shanghai Zhongshan Hospital|Shanghai Jinshan Hospital|Zhongshan Qingpu Hospital, Fudan University|Zhongshan Wusong Hospital, Fudan University|Macau University of Science and Technology Hospital|Health and Humanity Research Centre, Hongkong|Dr. Stanley Ho Medical Foundation, Macau
Sex: ALL
Age: CHILD, ADULT, OLDER_ADULT
Enrollment: 126
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: SINGLE (OUTCOMES_ASSESSOR)|Primary Purpose: TREATMENT
Start Date: 2024-07
Completion Date: 2026-12
Locations: Shanghai Zhongshan Hospital, Shanghai, Shanghai, 200032, China|Wusong Hospital, Zhongshan Hospital, Fudan University, Shanghai, Shanghai, 200094, China|Shanghai Jinshan Hospital, Shanghai, Shanghai, 201508, China|Qingpu Branch of Zhongshan Hospital, Fudan University, Shanghai, Shanghai, 201700, China|Health and Humanity Research Centre, Hongkong, China., Hong Kong, 999077, Hong Kong|Dr. Stanley Ho Medical Foundation, Macau, 999078, Macau|University Hospital, Macau University of Science and Technology., Macau, 999078, Macau

Summary:
The goal of this clinical trial is to learn if hydroxychloroquine (HCQ) plus thrombopoietin receptor agonists (TPO-RA) works to treat primary immune thrombocytopenia with positive anti-nuclear antibodies in adults. It will also learn about the safety of HCQ plus TPO-RA. The main questions it aims to answer are:

Does HCQ plus TPO-RA raise the response rate in participants, compared to TPO-RA alone? Does HCQ plus TPO-RA prolong the response duration in participants, compared to Pred alone? Does HCQ plus TPO-RA decrease the dose of TPO-RA to maintain response in participants, compared to TPO-RA alone? What medical problems do participants have when taking HCQ plus TPO-RA? Researchers will compare HCQ plus TPO-RA with TPO-RA alone to see if HCQ plus TPO-RA works better to treat primary immune thrombocytopenia with positive anti-nuclear antibodies.

Participants will:

Take TPO-RA every day for no more than 24 weeks, adjust the dose of TPO-RA according to the platelet level, with or without HCQ twice a day for 1 year; Visit the clinic once every 1 weeks for the first 8 weeks, and once every 2-4 weeks in the following 10 months for checkups and tests; Keep a diary of their symptoms

Primary Outcome Measures: Overall response rate, The percentage of participants with platelet counts higher than 30×10\^9/L and at least twice the baseline platelet count , for at least two consecutive tests (7 days apart)., 4 weeks
Secondary Outcome Measures: Complete response rate, The percentage of participants with platelet counts higher than 100×10\^9/L , for at least two consecutive tests (7 days apart)., 1 year|Duration of response, Time from response to disease relapse (platelet count ≤ 30×10\^9/L on any test or occurance of bleeding symptoms ), 1 year|Durable response rate, Percentage of patients with complete remission lasting at least 6 months without any additional ITP-specific therapy, 1 year|Platelet count at each visit, Average platelet count at each visit, 1 year|Time to response, Time from starting treatment to response, 4 weeks|Time to TPO-RA withdrawal, Time from starting treatment to TPO-RA withdrawal, 24 weeks|Rate of TPO-RA withdrawal, Rate of participants who withdraw TPO-RA with platelet count over 30×10\^9/L, 24 weeks|The maximum dose of TPO-RA to attain response, The maximum dose of TPO-RA to attain response (platelet count over 30×10\^9/L), 8 weeks|Response rate throughout the trial, The percentage of response participants (platelet counts higher than 30×10\^9/L and at least twice the baseline platelet count ) at each visit, 1 year|Once response rate throughout the trial, The percentage of once response participants (platelet counts higher than 30×10\^9/L and over twice the baseline platelet count for at least one visit) throughout 1 year, 1 year|WHO bleeding score, WHO bleeding score at each visit, 1 year|Adverse reaction, Adverse reaction at each visit, 1 year