Skip to main content
OpenTrials
Completed

NCT Number: NCT03838354

A Clinical Trial of Chidamide in the Management of Refractory ITP

Primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Increased macrophage phagocytosis of antibody-coated platelet as well as decreased number and/or impaired function of CD4+CD25+Foxp3+ regulatory T (Treg) cells have been shown to participate in the pathogenesis of ITP. Our preclinical data revealed that chidamide, a histone deacetylase inhibitor (HDACi), could attenuate macrophage phagocytosis of antibody-coated platelets, stimulate production of natural Foxp3+ Tregs, and upregulate CTLA4 expression through modulation of histone H3K27 acetylation. The project was undertaking by Qilu Hospital of Shandong University in China. In order to evaluate the efficacy and safety of chidamide at two different dosage regimens in adult patients with refractory ITP.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Thrombocytopenia Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Qilu hospital, Shandong University

Jinan, Shandong, China

About this study

In this prospective, open-label, multicenter, randomized clinical trial, refractory ITP adult patients will be enrolled from five medical centers in China. Eligible participants will be randomly assigned 1:1 to receive chidamide at either 2.5 or 5 mg twice per week. The primary endpoint is the overall response at week 12. Complete response was defined as a platelet count at or above 100×10^9/L and an absence of bleeding. Partial response was defined as a platelet count at or above 30×10^9/L but less than 100×10^9/L and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30×10^9 cells per L, or less than two-times increase from baseline platelet count, or bleeding. The secondary enpoints included 6-month sustained response, time to response (TTR), duration of response, bleeding scores, health-related quality of life assessment and adverse events (AEs). This study will compare the efficacy and safety of chidamide in two different dosage regimens in adult patients with refractory.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participant must be at least 18 years of age at the time of the screening.
  • Participant may be male or female.
  • Participant has a confirmed diagnosis of ITP according to the 2019 International Working Group assessment for more than 6 months at screening.
  • Participant who didn't respond or relapsed after previous first-line treatment, and lack of response to rituximab, TPO agents, or splenectomy.
  • Bone marrow biopsy is performed in participants over 60 years to exclude hematological malignancies.

Exclusion criteria

  • Participant has evidence of a secondary cause of immune thrombocytopenia or to drug treatments or participant has a multiple immune cytopenia, e.g. Evan's syndrome.
  • Participant with the following conditions:severe dysfunction of the heart, kidney, liver, or lung; severe immunodeficiency; malignancy; HIV; hepatitis B or C virus infection; pregnancy or lactation.

Treatment and study plan

Chidamide

Drug

In the 2.5 mg group, chidamide will be administered orally at an initial dose of 2.5 mg twice per week for 12 weeks. If an initial response was achieved by week 12, the allocated treatment could continue. Patients in 2.5 mg group were allowed to increase dose to 5 mg if platelet counts were less than 30×10^9 cells per L or less than two-times increase from baseline platelet count at week 12 according to investigators' advice and the patients' decision.

Other names: HBI-8000

Primary outcomes

  1. Overall response at week 12

    Time frame: week 12

    The primary endpoint was the overall response at week 12.Complete response was defined as a platelet count at or above 100×10^9/L and an absence of bleeding. Partial response was defined as a platelet count at or above 30×109/L but less than 100×10^9/L and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30×10^9 cells per L, or less than two-times increase from baseline platelet count, or bleeding.

Secondary outcomes

  1. Therapy associated adverse events

    Time frame: up to 1 year per subject

    The number and frequency of therapy associated adverse events

  2. Sustained response

    Time frame: 6 months

    A response lasting for at least 6 months was defined as a sustained response.

  3. Time to response

    Time frame: 12 weeks

    Time to response was defined as the time from treatment initiation to achieve a complete response or a partial response, whichever came first, assessed up to week 12.

  4. Duration of response

    Time frame: 12 months

    Duration of response was defined as the time from achievement of a complete response or a partial response to the loss of response.

  5. Bleeding scores

    Time frame: 12 months

    Bleeding symptoms were graded according a standardized bleeding scale specific to primary immune thrombocytopenia on the basis of site and severity of bleeding by Khellaf et al (PMID: 15951296). A modification was made to exclude age from the original scale so that only bleeding symptoms were described. At each visit, we recorded bleeding scores. Routine visits were scheduled once a week for the first 4 weeks and once a month thereafter. Scores ranged from 0 to 59, with higher values indicating higher bleeding risk.

  6. Health-related quality of life assessment

    Time frame: 12 weeks

    Health-related quality of life was assessed using a self-administered immune thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) at baseline and at week 12. Scores ranged from 0 to 100, with higher values indicating better quality of life.

Sponsors and collaborators

Lead sponsor

Shandong University

Other

Registry information

Official study title

A Prospective, Multicenter Clinical Trial of Chidamide in the Management of Refractory Immune Thrombocytopenia

Important dates

Study start
2021
Primary completion
2022
Study completion
2023
First posted
Feb 12, 2019
Registry last updated
Aug 26, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.