Clinical trial NCT06190145: Teriflunomide for Steroid-resistant/Relapse Immune Thrombocytopenia
Official title: A Single-arm, Open-label Phase II Study to Determine the Safety and Efficacy of Teriflunomide in Patients With Steroid-resistant/Relapse Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Keywords: Teriflunomide
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2024-01-05
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

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: Initial response
timeFrame: From the start of study treatment (Day 1) up to week 4 of treatment
measure: Time to response
timeFrame: From the start of study treatment (Day 1) to the end of week 24
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 week 24
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: Peking University Insititute of Hematology, Peking University People's Hospital
Study officials:
affiliation: Peking University Institute of Hematology, Peking University People's Hospital
name: Xiao-Hui Zhang
role: PRINCIPAL_INVESTIGATOR
References: