Teriflunomide Plus High-dose Dexamethasone as First-line Treatment in Newly Diagnosed Primary Immune Thrombocytopenia
- Conditions
- Immune Thrombocytopenia
- Interventions
- Registration Number
- NCT06176235
- Lead Sponsor
- Peking University People's Hospital
- Brief 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).
- Detailed 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 & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 132
- Newly diagnosed, treatment naïve ITP patients
- Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with bleeding manifestations at the enrollment;
- Willing and able to sign written informed consent.
- Received first-line and second-line ITP-modifying therapy (any previous dose of corticosteroids or other immune-suppressive agents);
- Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
- Active or a history of malignancy;
- Positive test result for hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
- Pregnancy or lactation;
- Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN);
- Current or recent (<4 weeks before screening) clinically serious viral, bacterial, fungal, or parasitic infection;
- 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;
- Patients who are deemed unsuitable for the study by the investigator.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Teriflunomide plus Dexamethasone Teriflunomide Oral Teriflunomide was given at a dose of 7 mg once daily for 24 weeks and dexamethasone was given at a dose of 40mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Nonresponsive participants with platelets less than 20 x10\^9/L or with active bleeding were allowed to repeat the 4-day course of dexamethasone treatment. Teriflunomide plus Dexamethasone Dexamethasone Oral Teriflunomide was given at a dose of 7 mg once daily for 24 weeks and dexamethasone was given at a dose of 40mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Nonresponsive participants with platelets less than 20 x10\^9/L or with active bleeding were allowed to repeat the 4-day course of dexamethasone treatment. Dexamethasone Dexamethasone Dexamethasone was given at a dose of 40mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Participants in this treatment arm who failed to achieve a sustained response and had a platelet count of less than 20 x 10\^9/L or with active bleeding were also allowed to repeat the 4-day course of dexamethasone treatment.
- 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 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.
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.
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.
Adverse events From the start of study treatment (Day 1) to the end of follow-up Adverse events (AEs) were reported and graded according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.
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.
Health-related quality of life (HRQoL) From the start of study treatment (Day 1) to the end of week 24 ITP-patient assessment questionnaire (ITP-PAQ) was used to assess the HRQoL before and after treatment.
Trial Locations
- Locations (10)
China-Japan Friendship Hospital
🇨🇳Beijing, China
Chinese PLA General Hospital
🇨🇳Beijing, China
Peking University Insititute of Hematology, Peking University People's Hospital
🇨🇳Beijing, China
Peking University First Hospital
🇨🇳Beijing, China
Beijing Hospital
🇨🇳Beijing, China
Beijing Friendship Hospital
🇨🇳Beijing, China
Beijing Luhe Hospital
🇨🇳Beijing, China
Beijing Tsinghua Changgeng Hospital
🇨🇳Beijing, China
Peking University Third Hospital
🇨🇳Beijing, China
The Sixth Medical Center of PLA General Hospital
🇨🇳Beijing, China