Using teriflunomide to treat immune thrombocytopenia that doesn't respond to steroids

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

This study is testing if the medication teriflunomide can help adults with immune thrombocytopenia who haven't responded to steroids feel better.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Peking University People's Hospital (other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Beijing)
• Trial ID: NCT06190145 on ClinicalTrials.gov

What this trial studies

This is a single-arm, open-label study evaluating the efficacy and safety of teriflunomide in approximately 40 adults with steroid-resistant or relapsed immune thrombocytopenia (ITP). Participants will receive teriflunomide at a dose of 7 mg orally once daily for 24 weeks, with adjustments as needed. The study will assess both efficacy and safety outcomes during scheduled visits throughout the treatment period.

Who should consider this trial

Good fit: Ideal candidates are adults diagnosed with primary immune thrombocytopenia who have not achieved a sustained response to corticosteroids.

Not a fit: Patients with secondary immune thrombocytopenia or those who have received certain medications affecting platelet counts recently may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new option for patients with ITP who do not respond to standard steroid therapies.

How similar studies have performed: While this approach is novel for ITP, similar studies using teriflunomide in other conditions have shown promising results.

Eligibility criteria

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.

Where this trial is running

Beijing

• Peking University Insititute of Hematology, Peking University People's Hospital — Beijing, China (RECRUITING)

Study contacts

• Principal investigator: Xiao-Hui Zhang — Peking University Institute of Hematology, Peking University People's Hospital
• Study coordinator: Xiao-Hui Zhang, MD

Conditions: Immune Thrombocytopenia, Teriflunomide