Teriflunomide for Steroid-resistant/Relapse Immune Thrombocytopenia

Phase 2

Recruiting

• Conditions: Immune Thrombocytopenia
• Interventions: Drug: Teriflunomide

• Registration Number: NCT06190145
• Lead Sponsor: Peking University People's Hospital

Brief 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).

Detailed 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.

Study Timeline

• Status Verified: 2023-12
• Study Start: 2023-12-05
• Study First Submitted: 2023-12-19
• Study First Submitted QC: 2023-12-19
• Last Update Submitted: 2023-12-19
• Study First Posted: 2024-01-05
• Last Update Posted: 2024-01-05
• Primary Completion: 2024-10-15
• Study Completion: 2025-06-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Teriflunomide	Teriflunomide	Oral Teriflunomide is given at a dose of 7 mg once daily with dose adjustments for 24 weeks. Treatment was discontinued if a dose-limiting toxic effect occurred, rescue medication was used, or concomitant medication for immune thrombocytopenia was changed beyond the 10% level as defined above.

Primary Outcome Measures

Name	Time	Method
Sustained response	From the start of study treatment (Day 1) to the end of week 24	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.

Secondary Outcome Measures

Name	Time	Method
Adverse events	From the start of study treatment (Day 1) to the end of week 24	Adverse events (AEs) were reported and graded according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.
Health-related quality of life (HRQoL)	From the start of study treatment (Day 1) to the end of week 24	ITP-PAQ was used to assess the Health-Related Quality of Life (HRQoL) before and after treatment.
Overall response	From the start of study treatment (Day 1) to the end of week 24	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.
Initial response	From the start of study treatment (Day 1) up to week 4 of treatment	The number of participants with achievement of CR or R at 4 weeks.
Time to response	From the start of study treatment (Day 1) to the end of week 24	The time from treatment initiation to achieve a CR or a R.
Bleeding events	From the start of study treatment (Day 1) to the end of week 24	Clinically significant bleeding was assessed using the World Health Organization (WHO) bleeding scale.

Trial Locations

Locations (1)

Peking University Insititute of Hematology, Peking University People's Hospital; 🇨🇳 Beijing, China