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

Immune Thrombocytopenia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06176911
• Other study ID #: PKU-TFITP-01
• Status: Recruiting
• Phase: Phase 2
• Start date: December 5, 2023
• Est. completion date: July 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

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 124
• Est. completion date: July 15, 2025
• Est. primary completion date: December 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

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

Locations

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

Sponsors (10)

Lead Sponsor	Collaborator
Peking University People's Hospital	Beijing Friendship Hospital, Beijing Hospital, Beijing Luhe Hospital, Beijing Tsinghua Changgeng Hospital, China-Japan Friendship Hospital, Chinese PLA General Hospital, Navy General Hospital, Beijing, Peking University First Hospital, Peking University Third 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	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	Duration of response	The time from the achievement of a complete response or a partial response to the loss of response.	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	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