NCT03838354

A Clinical Trial of Chidamide in the Management of Refractory ITP

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 61 (actual)
• Sponsor: Shandong University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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.

Conditions

• Thrombocytopenia

Interventions

Type	Name	Description
DRUG	Chidamide	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.
DRUG	Chidamide	In the 5 mg group, chidamide will be administered orally at an initial dose of 5 mg twice per week for 12 weeks. If an initial response was achieved by week 12, the allocated treatment could continue. Patients who did not respond to chidamide at 5 mg for 12 weeks would stop the allocated treatment and were continuously followed up.

Timeline

• Start date: 2021-06-01
• Primary completion: 2022-09-01
• Completion: 2023-09-01
• First posted: 2019-02-12
• Last updated: 2025-08-26

Locations

1 site across 1 country: China