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

NCT ID: NCT06479304

Last Updated: 2024-06-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

129 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-24

Study Completion Date

2026-12-31

Brief Summary

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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.

Detailed Description

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Primary immune thrombocytopenia (Primary immune thrombocytopenia, ITP) is an acquired autoimmune hemorrhagic disease characterized by a decreased peripheral platelet count and an increased risk of bleeding. It has been reported that 33.3% -39.2% of ITP patients have positive antinuclear antibodies (ANA) in the course of the disease.In the meantime, they do not meet the diagnostic criteria for rheumatic diseases such as lupus erythematosus(SLE). ITP patients with positive ANA are prone to relapse and chronicity. Therefore, it is necessary to explore new clinical treatments to attain long-term remission in these patients.

Hydroxychloroquine (HCQ) has immune modulating role on a variety of immune cells.A clinical trial enrolled immune thrombocytopenia secondary to SLE, and ITP with positive anti-nuclear antibodiy (ANA) were treated with HCQ combined with glucocorticoids. The results showed an overall response rate of 60% (24 / 40), including 18 continuous complete response (CR) and 6 continuous response (R), and some patients had continued elevated platelet counts 3 months after treatment initiation. The above studies illustrate that HCQ contributes to the treatment of chronic ITP, especially as a long-term therapeutic agent with low economic burden and good tolerance. In conclusion, it can be seen that HCQ and prednisone have complementary mechanism of action and complementary time window, which can be used as a combination for the treatment of ITP select.

Conditions

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Immune Thrombocytopenia With Positive ANA Antibodies

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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HCQ plus Pred group

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

Group Type EXPERIMENTAL

Hydroxychloroquine Oral Tablet

Intervention Type DRUG

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

Prednisone tablet

Intervention Type 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.

Pred group

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

Group Type PLACEBO_COMPARATOR

Prednisone tablet

Intervention Type 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.

Interventions

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Hydroxychloroquine Oral Tablet

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

Intervention Type DRUG

Prednisone tablet

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.

Intervention Type DRUG

Other Intervention Names

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Hydroxychloroquine Prednisone

Eligibility Criteria

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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).
Minimum Eligible Age

15 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shanghai Zhongshan Hospital

OTHER

Sponsor Role collaborator

Shanghai Jinshan Hospital

OTHER

Sponsor Role collaborator

Zhongshan Qingpu Hospital, Fudan University

UNKNOWN

Sponsor Role collaborator

Zhongshan Wusong Hospital, Fudan University

UNKNOWN

Sponsor Role collaborator

Macau University of Science and Technology Hospital

OTHER

Sponsor Role collaborator

Health and Humanity Research Centre, Hongkong

UNKNOWN

Sponsor Role collaborator

Dr. Stanley Ho Medical Foundation, Macau

UNKNOWN

Sponsor Role collaborator

Yunfeng Cheng

OTHER

Sponsor Role lead

Responsible Party

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Yunfeng Cheng

Professor of Institute of Clinical Science, Zhongshan Hospital

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Yunfeng Cheng

Role: PRINCIPAL_INVESTIGATOR

Shanghai Zhongshan Hospital

Locations

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Shanghai Zhongshan Hospital

Shanghai, Shanghai Municipality, China

Site Status RECRUITING

Zhongshan Wusong Hospital, Fudan University

Shanghai, Shanghai Municipality, China

Site Status RECRUITING

Shanghai Jinshan Hospital

Shanghai, Shanghai Municipality, China

Site Status RECRUITING

Zhongshan Qingpu Hospital, Fudan University

Shanghai, Shanghai Municipality, China

Site Status RECRUITING

Health and Humanity Research Centre, Hongkong, China.

Hong Kong, , Hong Kong

Site Status RECRUITING

Dr. Stanley Ho Medical Foundation

Macao, , Macau

Site Status RECRUITING

University Hospital, Macau University of Science and Technology.

Macao, , Macau

Site Status RECRUITING

Countries

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China Hong Kong Macau

Central Contacts

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Lili Ji

Role: CONTACT

86-021-64041990

Facility Contacts

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Lili Ji

Role: primary

086-021-64041990

Hongjuan Lu

Role: primary

13564618950

Yunfeng Cheng

Role: primary

15921398238

Fanli Hua

Role: primary

18116017032

Gregory Cheng

Role: primary

852-60269018

Gregory Cheng

Role: primary

852-60269018

Gregory Cheng

Role: primary

852-60269018

References

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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.

Reference Type BACKGROUND
PMID: 31794604 (View on PubMed)

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.

Reference Type RESULT
PMID: 24254965 (View on PubMed)

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.

Reference Type RESULT
PMID: 35288899 (View on PubMed)

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.

Reference Type RESULT
PMID: 28416506 (View on PubMed)

Other Identifiers

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B2024-028R2

Identifier Type: -

Identifier Source: org_study_id

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