The Combination of Teriflunomide and Danazol for Steroid-resistant/Relapse Immune Thrombocytopenia

Phase 2

Recruiting

• Conditions: Immune Thrombocytopenia
• Interventions: Drug: Danazol; Drug: Teriflunomide

• Registration Number: NCT06176911
• Lead Sponsor: Peking University People's Hospital

Brief Summary

To compare the efficacy and safety of teriflunomide plus danazol versus danazol in patients with steroid-resistant/relapse ITP

Detailed Description

This is a prospective, multicenter, randomized, controlled trial of 124 adult patients with steroid-resistant/relapse ITP in China. Patients were randomized to receive either experimental teriflunomide plus danazol or active comparator danazol monotherapy. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.

Study Timeline

• Status Verified: 2023-12
• Study Start: 2023-12-05
• Study First Submitted: 2023-12-06
• Study First Submitted QC: 2023-12-19
• Last Update Submitted: 2023-12-19
• Study First Posted: 2023-12-20
• Last Update Posted: 2023-12-20
• Primary Completion: 2024-12-30
• Study Completion: 2025-07-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 124

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Danazol	Danazol	Danazol was given at a dose of 200 mg twice a day for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.
Teriflunomide plus danazol	Teriflunomide	Oral teriflunomide was given at a starting dose of 7 mg once daily and danazol was given at a dose of 200mg twice daily for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.
Teriflunomide plus danazol	Danazol	Oral teriflunomide was given at a starting dose of 7 mg once daily and danazol was given at a dose of 200mg twice daily for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.

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
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.
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.
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.
Duration of response	From the start of study treatment (Day 1) to the end of week 24	The time from the achievement of a complete response or a partial response to the loss of response.
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.
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, Beijing, China