Combining Teriflunomide and Danazol for Immune Thrombocytopenia

A Multicenter, Open-label, Randomized, Controlled, Phase 2 Trial Comparing the Efficacy and Safety of Teriflunomide Plus Danazol in Patients With Steroid-resistant/Relapse ITP

This study is testing if a combination of two medications, teriflunomide and danazol, can help adults with immune thrombocytopenia who haven't responded to steroids feel better compared to using danazol alone.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 124 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Peking University People's Hospital (other)
• Locations: 1 site (Beijing, Beijing)
• Trial ID: NCT06176911 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the efficacy and safety of a combination treatment of teriflunomide and danazol compared to danazol alone in adults with steroid-resistant or relapsed immune thrombocytopenia (ITP). It is a multicenter, randomized, controlled trial involving 124 patients in China, who will be assigned to one of the two treatment groups. The study will monitor patients for adverse events and treatment responses over the course of the trial.

Who should consider this trial

Good fit: Ideal candidates are adults diagnosed with primary immune thrombocytopenia who have not responded to corticosteroids and have low platelet counts.

Not a fit: Patients with secondary immune thrombocytopenia or those with active malignancies or liver disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment combination could provide a new effective option for patients with difficult-to-treat immune thrombocytopenia.

How similar studies have performed: While there have been studies on individual treatments for ITP, this specific combination approach is novel and has not been extensively tested.

Eligibility criteria

Inclusion Criteria:

1. Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
2. 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;
3. Patients with a platelet count \<30,000/μL or a platelet count \<50,000/μL with bleeding manifestations at the enrollment;
4. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, or patients with confirmed autoimmune disease);
2. Active or a history of malignancy;
3. Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than two times the upper limit of normal (ULN);
4. Pregnancy or lactation;
5. Current or recent (\< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
6. Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
7. 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;
8. Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled;
9. Patients who are deemed unsuitable for the study by the investigator.

Where this trial is running

Beijing, Beijing

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

Study contacts

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

Conditions: Immune Thrombocytopenia, Teriflunomide, Danazol