Immune Thrombocytopenia (ITP)
Conditions
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.
Interventions
DRUGTeriflunomide
Teriflunomide 14 mg orally once daily for 24 weeks.
DRUGDexamethasone
Dexamethasone 40 mg orally once daily for four consecutive days.
Sponsors
Peking University Health Science Center
Cisen Pharmaceutical CO., LTD.
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| Duration of response | Week 24 | The time from the achievement of a CR or R to the loss of CR or R. |
Countries
China
Contacts
Primary ContactXiao-Hui Zhang, MD
Backup ContactLi-Ping Yang, MD
Outcome results
None listed