Teriflunomide for Steroid-resistant/Relapse Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

Official title:

A Single-arm, Open-label Phase II Study to Determine the Safety and Efficacy of Teriflunomide in Patients With Steroid-resistant/Relapse Thrombocytopenia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06190145
• Other study ID #: PKU-TFITP-02
• Status: Recruiting
• Phase: Phase 2
• Start date: December 5, 2023
• Est. completion date: June 15, 2025

Study information

• Verified date: December 2023
• Source: Peking University People's Hospital
• Contact: Xiao-Hui Zhang, MD
• Phone: +8613522338836
• Email: zhangxh100[@]sina.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Single-arm, open-label, single-center study to evaluate the efficacy and safety of teriflunomide for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).

Description:

This is a prospective, single-arm, open-label study of teriflunomide in approximately 40 patients with ITP who have relapsed or have an insufficient response to prior therapies. Teriflunomide is administered as 7 mg orally once daily with dose adjustments for 24 weeks. Efficacy and safety outcomes are assessed on scheduled study visits.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: June 15, 2025
• Est. primary completion date: October 15, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

Inclusion Criteria:

1. Diagnosis of primary immune thrombocytopenia (ITP) by excluding other supervened causes of thrombocytopenia

2. Patients with a platelet count <30,000/µL or a platelet count <50,000/µL with bleeding manifestations at the enrollment;

3. 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;

4. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;

2. Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, malignancy, or patients with confirmed autoimmune disease);

3. Pregnancy or lactation;

4. Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN);

5. Current or recent (< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;

6. Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB;

7. Patients who are deemed unsuitable for the study by the investigator.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Teriflunomide
Starting dose of 7 mg once daily. Dose adjustments were made throughout the study based on individual platelet counts.

Locations

Country	Name	City	State
China	Peking University Insititute of Hematology, Peking University People's Hospital	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Peking University People's Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Sustained response	Platelet count over 30,000/µL and at least a 2-fold increase of the baseline count in the absence of bleeding and rescue therapy for at least four of the six visits between weeks 19 and 24.	From the start of study treatment (Day 1) to the end of week 24
Secondary	Overall response	Complete response (CR) was defined as platelet count over 100,000/µL and absence of bleeding. Response (R) was defined as platelet count over 30,000/µL and at least a 2-fold increase of the baseline count and absence of bleeding.	From the start of study treatment (Day 1) to the end of week 24
Secondary	Initial response	The number of participants with achievement of CR or R at 4 weeks.	From the start of study treatment (Day 1) up to week 4 of treatment
Secondary	Time to response	The time from treatment initiation to achieve a CR or a R.	From the start of study treatment (Day 1) to the end of week 24
Secondary	Bleeding events	Clinically significant bleeding was assessed using the World Health Organization (WHO) bleeding scale.	From the start of study treatment (Day 1) to the end of week 24
Secondary	Adverse events	Adverse events (AEs) were reported and graded according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.	From the start of study treatment (Day 1) to the end of week 24
Secondary	Health-related quality of life (HRQoL)	ITP-PAQ was used to assess the Health-Related Quality of Life (HRQoL) before and after treatment.	From the start of study treatment (Day 1) to the end of week 24