The Efficacy and Safety of Combined Teriflunomide and High-dose Dexamethasone in Newly Diagnosed Primary Immune Thrombocytopenia (TEMPO-2)

Not Applicable

Not yet recruiting

• Conditions: Immune Thrombocytopenia (ITP)
• Interventions: Drug: Teriflunomide; Drug: Dexamethasone

• Registration Number: NCT07065968
• Lead Sponsor: Peking University People's Hospital

Brief Summary

A multicenter, open-label, randomized study to report 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

The investigators are undertaking 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. Platelet count, bleeding, and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Study Timeline

• Status Verified: 2025-06
• Study First Submitted: 2025-07-04
• Study First Submitted QC: 2025-07-04
• Last Update Submitted: 2025-07-04
• Study Start: 2025-07-15
• Study First Posted: 2025-07-15
• Last Update Posted: 2025-07-15
• Primary Completion: 2027-09-10
• Study Completion: 2027-12-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 132

Inclusion Criteria

1. Patients aged ≥18 years;
2. Newly diagnosed, treatment naïve ITP patients;
3. Patients with a platelet count <30 x10^9/L or a platelet count <50 x10^9/L with bleeding manifestations at the enrollment;
4. Willing and able to sign written informed consent.

Exclusion Criteria

1. Secondary ITP such as drug-related thrombocytopenia, viral infection (HIV, hepatitis B virus, or hepatitis C virus);
2. Pre-existing acute or chronic liver disease, or ALT/AST greater than 2 times the upper limit of normal (ULN);
3. Severe cardiac, renal, hepatic, or respiratory insufficiency;
4. Severe immunodeficiency;
5. Pregnancy or lactation;
6. Active or a history of malignancy;
7. Active infection requiring systemic therapy;
8. Myelodysplastic syndrome, aplastic anemia, or myelofibrosis;
9. A known diagnosis of other autoimmune diseases;
10. 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 14 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 30 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 14 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 30 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). Nonresponsive participants with platelets less than 30 x10\^9/L or with active bleeding were allowed to repeat the 4-day course of dexamethasone treatment.

Primary Outcome Measures

Name	Time	Method
Sustained response	Week 24	Sustained response was defined as a platelet count ≥ 30 x10\^9/L and at least a 2-fold increase of the baseline count in the absence of bleeding and rescue therapy for at least three of the four visits of the last 8 weeks of treatment.

Secondary Outcome Measures

Name	Time	Method
Initial response	Week 4	The number of participants with achievement of CR or R at 4 weeks. Complete response (CR) was defined as a platelet count ≥ 100 x10\^9/L and absence of bleeding. Response (R) was defined as a platelet count ≥ 30 x10\^9/L and at least a 2-fold increase of the baseline count and absence of bleeding. No response was defined as a platelet count of less than 30 x10\^9/L, or less than two times increase from baseline platelet count, or bleeding.
Time to response	Week 24	The time from treatment initiation to time of achievement of CR or R.
Duration of response	Week 24	The time from the achievement of a CR or R to the loss of CR or R.
Bleeding events	Week 24	Clinically significant bleeding was assessed using the World Health Organization (WHO) bleeding scale.
Adverse events	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)	Week 24	ITP-patient assessment questionnaire (ITP-PAQ) was used to assess the HRQoL before and after treatment.

Trial Locations

Locations (3)

Peking University First Hospital; 🇨🇳 Beijing, Beijing, China

Peking University Insititute of Hematology, Peking University People's Hospital; 🇨🇳 Beijing, Beijing, China

Peking University Third Hospital; 🇨🇳 Beijing, Beijing, China