Efficacy and Safety of Iguratimod in the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia

Efficacy and Safety of Iguratimod in the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia: a Phase 2, Open-label, Single-arm Trial

A phase 2, open-label, single-arm study to evaluate the efficacy and safety of iguratimod for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP)

Study Overview

• Status: Not yet recruiting
• Conditions: Immune Thrombocytopenia
• Intervention / Treatment: Drug: Iguratimod

Detailed Description

The investigators are undertaking an open-label, single-arm study of 100 adults with steroid-resistant/ relapse ITP in China. Patients were received Iguratimod treatment. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

• Study Type: Interventional
• Enrollment (Anticipated): 100
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Xiao-Hui Zhang, MD; Phone Number: 86-10-88324577; Email: zhangxh@bjmu.edu.cn

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100010
      • Peking University Insititute of Hematology, Peking University People's Hospital
      • Contact: Xiao Hui Zhang, doctor; Email: zhangxh100@sina.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
• Platelet count of less than 30×10^9/L at enrollment;
• Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation;
• 18 years older;

Exclusion Criteria:

• Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
• Congestive heart failure
• Severe arrhythmia
• Nursing or pregnant women
• Aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria
• Creatinine or serum bilirubin levels each 1•5 times or more than the normal range
• Active or previous malignancy
• Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

EXPERIMENTAL: Iguratimod treatment; Iguratimod is given at a dose of 25 mg bid for 12 weeks.

Drug: Iguratimod; Oral iguratimod (25 mg twice daily) for 12 weeks. Iguratimod is a new drug for the treatment of rheumatoid arthritis (RA) and osteoarthritis (OA), which was filed for marketing in Japan in 2003. It can significantly reduce the inflammatory response, not only selectively inhibit COX-2, but also inhibit the production of inflammatory cytokines, tumor necrosis factor, lymphocytes and immunoglobulins, and has an autoimmunomodulatory effect; it has a rapid onset of action, better efficacy and fewer adverse effects than existing drugs, and is effective in patients for whom other drugs are ineffective. It has been reported in the literature that in vitro iguratimod can inhibit the activity of nuclear factor-κB (NF-κB), which in turn inhibits the production of inflammatory cytokines (interleukin-1, interleukin-6, interleukin-8, tumor necrosis factor alpha). Iguratimod also interacts directly with mouse and human B cells in vitro to inhibit the production of immunoglobulins.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Durable response	The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Initial response	Platelet count ≥ 30 x 10^9/L and at least doubling baseline at 1 month	1 month
Remission	109/ Platelet count >100x 10^9/L at 12 mon	12 months
Time to response	Time to response was defined as the time from starting treatment to the time to achieve the response	12 months
Bleeding	Major bleeding: (1) WHO grade 3 or 4 bleeding, (2) Buchanan severe grade, (3) Bolton-Maggs and Moon "major bleeding," (4) IBLS grade 2 or higher, or (5) life-threatening or intracerebral hemorrhage bleeding	12 months
Adverse events	Adverse events	12 months

Collaborators and Investigators

• Sponsor: Peking University People's Hospital

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): March 22, 2022
• Primary Completion (ANTICIPATED): December 12, 2023
• Study Completion (ANTICIPATED): December 12, 2023

Study Registration Dates

• First Submitted: March 21, 2022
• First Submitted That Met QC Criteria: March 21, 2022
• First Posted (ACTUAL): March 31, 2022

Study Record Updates

• Last Update Posted (ACTUAL): March 31, 2022
• Last Update Submitted That Met QC Criteria: March 21, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: I-ITP 001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No