Teriflunomide Plus High-dose Dexamethasone as First-line Treatment in Newly Diagnosed Primary Immune Thrombocytopenia

NCT06176235 | Withdrawn Phase 2 DMC

• 1 other identifier: PKU-TFITP-03
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 10

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).

Conditions

Immune Thrombocytopenia

Keywords

Teriflunomide; Dexamethasone

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 (7)

• Overall response

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

• Time to response

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

• Duration of 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

• Bleeding events

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

Study Arms (2)

Teriflunomide plus Dexamethasone

EXPERIMENTAL

Oral Teriflunomide was given at a dose of 7 mg once daily for 24 weeks and dexamethasone was given at a dose of 40mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Nonresponsive participants with platelets less than 20 x10\^9/L or with active bleeding were allowed to repeat the 4-day course of dexamethasone treatment.

Drug: Teriflunomide; Drug: Dexamethasone

Dexamethasone

ACTIVE COMPARATOR

Dexamethasone was given at a dose of 40mg, orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Participants in this treatment arm who failed to achieve a sustained response and had a platelet count of less than 20 x 10\^9/L or with active bleeding were also allowed to repeat the 4-day course of dexamethasone treatment.

Drug: Dexamethasone

Interventions

Teriflunomide DRUG

Teriflunomide 7 mg orally once daily for 24 weeks. Dose adjustments were made throughout the study based on individual platelet counts.

Also known as: AUBAGIO

Teriflunomide plus Dexamethasone

Dexamethasone DRUG

Dexamethasone 40 mg orally once daily for four consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 14).

Dexamethasone; Teriflunomide plus Dexamethasone

Eligibility Criteria

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

You may qualify if:

• Newly diagnosed, treatment naïve ITP patients
• Patients with a platelet count \<30,000/μL or a platelet count \<50,000/μL with bleeding manifestations at the enrollment;
• Willing and able to sign written informed consent.

You may not qualify if:

• Received first-line and second-line ITP-modifying therapy (any previous dose of corticosteroids or other immune-suppressive agents);
• Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
• Active or a history of malignancy;
• Positive test result for hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
• 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 before screening) clinically serious viral, bacterial, fungal, or parasitic infection;
• 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;
• Patients who are deemed unsuitable for the study by the investigator.

Sponsors & Collaborators

• Peking University People's Hospital: lead
• Beijing Luhe Hospital: collaborator
• Chinese PLA General Hospital: collaborator
• Navy General Hospital, Beijing: collaborator
• Beijing Hospital: collaborator
• Beijing Friendship Hospital: collaborator
• Peking University First Hospital: collaborator
• Peking University Third Hospital: collaborator
• China-Japan Friendship Hospital: collaborator
• Beijing Tsinghua Changgeng Hospital: collaborator

Study Sites (10)

Beijing Friendship Hospital

Beijing, China

Location

Beijing Hospital

Beijing, China

Location

Beijing Luhe Hospital

Beijing, China

Location

Beijing Tsinghua Changgeng Hospital

Beijing, China

Location

China-Japan Friendship Hospital

Beijing, China

Location

Chinese PLA General Hospital

Beijing, China

Location

Peking University First Hospital

Beijing, China

Location

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

Beijing, China

Location

Peking University Third Hospital

Beijing, China

Location

The Sixth Medical Center of PLA General Hospital

Beijing, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

teriflunomide; Dexamethasone

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

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Study Officials

• Xiao-Hui Zhang, MD

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

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: December 9, 2023
• First Posted: December 19, 2023
• Study Start: December 19, 2023
• Primary Completion: December 12, 2024
• Study Completion: May 12, 2025
• Last Updated: August 11, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (10)