Teriflunomide Plus High-dose Dexamethasone as First-line Treatment in Newly Diagnosed Primary Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

Official title:

The Combination of Teriflunomide and High-dose Dexamethasone vs High-dose Dexamethasone Alone as First-line Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia (ITP): A Prospective, Multicenter, Randomized Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06176235
• Other study ID #: PKU-TFITP-03
• Status: Recruiting
• Phase: Phase 2
• Start date: December 19, 2023
• Est. completion date: May 12, 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

A randomized, open-label, multicenter study to compare the efficacy and safety of teriflunomide plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with newly diagnosed primary immune thrombocytopenia (ITP).

Description:

This is a parallel-group, multicenter, randomized controlled trial of 132 adults with ITP in China. Patients were randomized to teriflunomide plus high-dose dexamethasone and high-dose dexamethasone monotherapy group. Patients who do not respond to dexamethasone may receive another cycle of high-dose dexamethasone therapy within 2 weeks. Platelet count, bleeding, and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 132
• Est. completion date: May 12, 2025
• Est. primary completion date: December 12, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

Inclusion Criteria:

1. Newly diagnosed, treatment naïve ITP patients

2. Patients with a platelet count <30,000/µL or a platelet count <50,000/µL with bleeding manifestations at the enrollment;

3. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Received first-line and second-line ITP-modifying therapy (any previous dose of corticosteroids or other immune-suppressive agents);

2. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;

3. Active or a history of malignancy;

4. Positive test result for hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);

5. Pregnancy or lactation;

6. Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN);

7. Current or recent (<4 weeks before screening) clinically serious viral, bacterial, fungal, or parasitic infection;

8. A known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;

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
Teriflunomide 7 mg orally once daily for 24 weeks. Dose adjustments were made throughout the study based on individual platelet counts.
• Dexamethasone
Dexamethasone 40 mg orally once daily for four consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 14).

Locations

Country	Name	City	State
China	Beijing Friendship Hospital	Beijing
China	Beijing Hospital	Beijing
China	Beijing Luhe Hospital	Beijing
China	Beijing Tsinghua Changgeng Hospital	Beijing
China	China-Japan Friendship Hospital	Beijing
China	Chinese PLA General Hospital	Beijing
China	Peking University First Hospital	Beijing
China	Peking University Insititute of Hematology, Peking University People's Hospital	Beijing
China	Peking University Third Hospital	Beijing
China	The Sixth Medical Center of PLA General Hospital	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 follow-up
Secondary	Health-related quality of life (HRQoL)	ITP-patient assessment questionnaire (ITP-PAQ) was used to assess the HRQoL before and after treatment.	From the start of study treatment (Day 1) to the end of week 24