Clinical trial NCT06176235: Teriflunomide Plus High-dose Dexamethasone as First-line Treatment in Newly Diagnosed Primary Immune Thrombocytopenia
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Keywords: Teriflunomide
Dexamethasone
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
Collaborators: name: Beijing Luhe Hospital
name: Chinese PLA General Hospital
name: Navy General Hospital, Beijing
name: Beijing Hospital
name: Beijing Friendship Hospital
name: Peking University First Hospital
name: Peking University Third Hospital
name: China-Japan Friendship Hospital
name: Beijing Tsinghua Changgeng Hospital
First posted: 2023-12-19
Enrollment: 132
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Sustained response
timeFrame: From the start of study treatment (Day 1) to the end of week 24
Secondary outcomes:
measure: Overall response
timeFrame: From the start of study treatment (Day 1) to the end of week 24
measure: Time to response
timeFrame: From the start of study treatment (Day 1) to the end of week 24
measure: Duration of response
timeFrame: From the start of study treatment (Day 1) to the end of week 24
measure: Initial response
timeFrame: From the start of study treatment (Day 1) up to week 4 of treatment
measure: Bleeding events
timeFrame: From the start of study treatment (Day 1) to the end of week 24
measure: Adverse events
timeFrame: From the start of study treatment (Day 1) to the end of follow-up
measure: Health-related quality of life (HRQoL)
timeFrame: From the start of study treatment (Day 1) to the end of week 24
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Friendship Hospital
city: Beijing
country: China
facility: Beijing Hospital
city: Beijing
country: China
facility: Beijing Luhe Hospital
city: Beijing
country: China
facility: Beijing Tsinghua Changgeng Hospital
city: Beijing
country: China
facility: China-Japan Friendship Hospital
city: Beijing
country: China
facility: Chinese PLA General Hospital
city: Beijing
country: China
facility: Peking University First Hospital
city: Beijing
country: China
facility: Peking University Insititute of Hematology, Peking University People's Hospital
city: Beijing
country: China
facility: Peking University Third Hospital
city: Beijing
country: China
facility: The Sixth Medical Center of PLA General Hospital
Study officials:
affiliation: Peking University Institute of Hematology, Peking University People's Hospital
name: Xiao-Hui Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: