A Clinical Trial of Chidamide in the Management of Refractory ITP

NCT03838354 | Completed Phase 1 DMC

• 1 other identifier: ITP-Chidamide
• Study Type: interventional
• Target: 61
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Thrombocytopenia

Keywords

ITP; Chidamide

Outcome Measures

Primary Outcomes (1)

• Overall response at 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.

  week 12

Secondary Outcomes (6)

• Therapy associated adverse events

  up to 1 year per subject

• Sustained response

  6 months

• Time to response

  12 weeks

• Duration of response

  12 months

• Bleeding scores

  12 months

Study Arms (2)

A

EXPERIMENTAL

Chidamide 2.5 mg po twice per week

Drug: Chidamide

B

EXPERIMENTAL

Chidamide 5 mg po twice per week

Drug: Chidamide

Interventions

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.

Also known as: HBI-8000

A

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shandong University: lead

Study Sites (1)

Qilu hospital, Shandong University

Jinan, Shandong, China

Location

MeSH Terms

Conditions

Thrombocytopenia

Interventions

N-(2-amino-5-fluorobenzyl)-4-(N-(pyridine-3-acrylyl)aminomethyl)benzamide; HBI-8000

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor and Director

Study Record Dates

• First Submitted: November 27, 2018
• First Posted: February 12, 2019
• Study Start: June 1, 2021
• Primary Completion: September 1, 2022
• Study Completion: September 1, 2023
• Last Updated: August 26, 2025

Record last verified: 2025-08

Locations

CN (1)