Teriflunomide for Steroid-resistant/Relapse Immune Thrombocytopenia

NCT06190145 | Unknown Phase 2 DMC

• 1 other identifier: PKU-TFITP-02
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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).

Conditions

Immune Thrombocytopenia

Keywords

Teriflunomide

Outcome Measures

Primary Outcomes (1)

• Sustained response

  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.

  From the start of study treatment (Day 1) to the end of week 24

Secondary Outcomes (6)

• Overall response

  From the start of study treatment (Day 1) to the end of week 24

• Initial response

  From the start of study treatment (Day 1) up to week 4 of treatment

• Time to response

  From the start of study treatment (Day 1) to the end of week 24

• Bleeding events

  From the start of study treatment (Day 1) to the end of week 24

• Adverse events

  From the start of study treatment (Day 1) to the end of week 24

Study Arms (1)

Teriflunomide

EXPERIMENTAL

Oral Teriflunomide is given at a dose of 7 mg once daily with dose adjustments for 24 weeks. Treatment was discontinued if a dose-limiting toxic effect occurred, rescue medication was used, or concomitant medication for immune thrombocytopenia was changed beyond the 10% level as defined above.

Drug: Teriflunomide

Interventions

Teriflunomide DRUG

Starting dose of 7 mg once daily. Dose adjustments were made throughout the study based on individual platelet counts.

Also known as: AUBAGIO

Teriflunomide

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of primary immune thrombocytopenia (ITP) by excluding other supervened causes of thrombocytopenia
• Patients with a platelet count \<30,000/μL or a platelet count \<50,000/μL with bleeding manifestations at the enrollment;
• 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;
• Willing and able to sign written informed consent.

You may not qualify if:

• Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
• Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, malignancy, or patients with confirmed autoimmune disease);
• Pregnancy or lactation;
• Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN);
• Current or recent (\< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
• 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;
• Patients who are deemed unsuitable for the study by the investigator.

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Peking University Insititute of Hematology, Peking University People's Hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

teriflunomide

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Xiao-Hui Zhang

  Peking University Institute of Hematology, Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiao-Hui Zhang, MD

CONTACT

+8613522338836; zhangxh100@sina.com

Li-Ping Yang, MD

CONTACT

+8618519172033; lpyangvip@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking Univeristy Institute of Hematology

Study Record Dates

• First Submitted: December 19, 2023
• First Posted: January 5, 2024
• Study Start: December 5, 2023
• Primary Completion: October 15, 2024
• Study Completion: June 15, 2025
• Last Updated: January 5, 2024

Record last verified: 2023-12

Locations

CN (1)