Immune Thrombocytopenia
Conditions
Keywords
Teriflunomide
Brief summary
Single-arm, open-label, single-center study to evaluate the efficacy and safety of teriflunomide for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
Detailed description
This is a prospective, single-arm, open-label study of teriflunomide in approximately 40 patients with ITP who have relapsed or have an insufficient response to prior therapies. Teriflunomide is administered as 7 mg orally once daily with dose adjustments for 24 weeks. Efficacy and safety outcomes are assessed on scheduled study visits.
Interventions
DRUGTeriflunomide
Starting dose of 7 mg once daily. Dose adjustments were made throughout the study based on individual platelet counts.
Sponsors
Peking University People's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Diagnosis of primary immune thrombocytopenia (ITP) by excluding other supervened causes of thrombocytopenia 2. Patients with a platelet count \<30,000/μL or a platelet count \<50,000/μL with bleeding manifestations at the enrollment; 3. Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid tapering or after its discontinuation; 4. Willing and able to sign written informed consent.
Exclusion criteria
1. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit; 2. Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, malignancy, or patients with confirmed autoimmune disease); 3. Pregnancy or lactation; 4. Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN); 5. Current or recent (\< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection; 6. Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB; 7. Patients who are deemed unsuitable for the study by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
| Adverse events | From the start of study treatment (Day 1) to the end of 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) | From the start of study treatment (Day 1) to the end of week 24 | ITP-PAQ was used to assess the Health-Related Quality of Life (HRQoL) before and after treatment. |
| 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. |
Countries
China
Contacts
Primary ContactXiao-Hui Zhang, MD
Backup ContactLi-Ping Yang, MD
Outcome results
None listed