The Efficacy and Safety of HCQ Plus Pred in ANA Positive ITP

NCT06479304 | Recruiting DMC

• 1 other identifier: B2024-028R2
• Study Type: interventional
• Target: 129
• Locations: 3 countries
• Sites: 7

Brief Summary

The goal of this clinical trial is to learn if hydroxychloroquine (HCQ) plus prednisone (Pred) works to treat primary immune thrombocytopenia with positive anti-nuclear antibodies in adults. It will also learn about the safety of HCQ plus Pred. The main questions it aims to answer are: Does HCQ plus Pred raise the response rate in participants, compared to Pred alone? Does HCQ plus Pred prolong the response duration in participants, compared to Pred alone? What medical problems do participants have when taking HCQ plus Pred? Researchers will compare HCQ plus Pred with Pred alone to see if HCQ plus Pred works better to treat primary immune thrombocytopenia with positive anti-nuclear antibodies. Participants will: Take Pred every day for 6 weeks, with or without HCQ twice a day for 1 year , Visit the clinic once every 1 weeks for the first 4 weeks, and once every 2-4 weeks in the following 11 months for checkups and tests, Keep a diary of their symptoms.

Conditions

Immune Thrombocytopenia With Positive ANA Antibodies

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• Complete response rate

  1 year

• Duration of response

  1 year

• Durable response rate

  1 year

• Platelet count at each visit

  1 year

• Time to response

  4 weeks

Study Arms (2)

HCQ plus Pred group

EXPERIMENTAL

This group is experiment group. Participants will take Pred every day for 6 weeks with HCQ twice a day for 1 year

Drug: Hydroxychloroquine Oral Tablet; Drug: Prednisone tablet

Pred group

PLACEBO COMPARATOR

This group is control group. Participants will take Pred every day for 6 weeks.

Drug: Prednisone tablet

Interventions

Hydroxychloroquine Oral Tablet DRUG

Hydroxychloroquine is taken at the dose of 0.1g / dose, twice a day for 1 year, regardless of food intake.

Also known as: Hydroxychloroquine

HCQ plus Pred group

Prednisone tablet DRUG

Prednisone is given at the dose of 1mg/kg every morning after meals for 2 weeks ( if platelet count does not recover higher than 30×10\^9/L after 2 weeks, prednisone will be given at the dose for 2 more weeks ), then tapering off.

Also known as: Prednisone

HCQ plus Pred group; Pred group

Eligibility Criteria

• Age: 15 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.
• The antinuclear antibody is positive.
• Other autoantibodies (mainly including dsDNA antibodies, SSA, SSB, RNP, β 2-GP, ACA, ANCA) are negative.
• 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.
• Understand the study procedures and sign the written informed consent form.

You may not qualify if:

• 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.
• 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.
• 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.
• With severe hemorrhage (intracranial hemorrhage) or coagulation dysfunction (INR and APTT\> 125% upper limit of normal).
• With severe digestive tract diseases affecting drug absorption.
• With serious mental illness patient.
• Having participated in other clinical trials within 3 months prior to screening.
• Having received any immunomodulatory medication for other diseases 3 months before screening.
• 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.
• With Glucose-6-phosphate dehydrogenase deficiency.
• With retinal or visual field changes caused by 4-aminoquinoline compounds.
• Being allergic to 4-aminoquinoline compounds.
• 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.
• 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.
• With liver cirrhosis or portal hypertension.

Sponsors & Collaborators

• Yunfeng Cheng: lead
• Shanghai Zhongshan Hospital: collaborator
• Shanghai Jinshan Hospital: collaborator
• Zhongshan Qingpu Hospital, Fudan University: collaborator
• Zhongshan Wusong Hospital, Fudan University: collaborator
• Macau University of Science and Technology Hospital: collaborator
• Health and Humanity Research Centre, Hongkong: collaborator
• Dr. Stanley Ho Medical Foundation, Macau: collaborator

Study Sites (7)

Shanghai Zhongshan Hospital

Shanghai, Shanghai Municipality, 200032, China

RECRUITING

Zhongshan Wusong Hospital, Fudan University

Shanghai, Shanghai Municipality, 200094, China

RECRUITING

Shanghai Jinshan Hospital

Shanghai, Shanghai Municipality, 201508, China

RECRUITING

Zhongshan Qingpu Hospital, Fudan University

Shanghai, Shanghai Municipality, 201700, China

RECRUITING

Health and Humanity Research Centre, Hongkong, China.

Hong Kong, 999077, Hong Kong

RECRUITING

Dr. Stanley Ho Medical Foundation

Macao, 999078, Macau

RECRUITING

University Hospital, Macau University of Science and Technology.

Macao, 999078, Macau

RECRUITING

Related Publications (4)

• Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kuhne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866. doi: 10.1182/bloodadvances.2019000966.

  PMID: 31794604 BACKGROUND

• Khellaf M, Chabrol A, Mahevas M, Roudot-Thoraval F, Limal N, Languille L, Bierling P, Michel M, Godeau B. Hydroxychloroquine is a good second-line treatment for adults with immune thrombocytopenia and positive antinuclear antibodies. Am J Hematol. 2014 Feb;89(2):194-8. doi: 10.1002/ajh.23609. Epub 2013 Nov 20.

  PMID: 24254965 RESULT

• Mejdoub S, Hachicha H, Gargouri L, Feki S, Mahfoudh A, Masmoudi H. Antinuclear antibodies in children: clinical signification and diagnosis utility. Tunis Med. 2021 Octobre;99(10):980-984.

  PMID: 35288899 RESULT

• Lambert MP, Gernsheimer TB. Clinical updates in adult immune thrombocytopenia. Blood. 2017 May 25;129(21):2829-2835. doi: 10.1182/blood-2017-03-754119. Epub 2017 Apr 17.

  PMID: 28416506 RESULT

MeSH Terms

Interventions

Hydroxychloroquine; Prednisone

Intervention Hierarchy (Ancestors)

Chloroquine; Aminoquinolines; Quinolines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Yunfeng Cheng

  Shanghai Zhongshan Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lili Ji

CONTACT

86-021-64041990; ji.lili@zs-hospital.sh.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Institute of Clinical Science, Zhongshan Hospital

Study Record Dates

• First Submitted: June 22, 2024
• First Posted: June 28, 2024
• Study Start: April 24, 2024
• Primary Completion: January 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: June 28, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: IPD information will become available starting 6 months after publication, for 1 year.

Locations

CN (4); HK (1); MA (2)