The Combination of Teriflunomide and Danazol for Steroid-resistant/Relapse Immune Thrombocytopenia

NCT06176911 | Unknown Phase 2 DMC

• 1 other identifier: PKU-TFITP-01
• Study Type: interventional
• Target: 124
• Locations: 1 country
• Sites: 1

Brief Summary

To compare the efficacy and safety of teriflunomide plus danazol versus danazol in patients with steroid-resistant/relapse ITP

Conditions

Immune Thrombocytopenia

Keywords

Teriflunomide; Danazol

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 danazol

EXPERIMENTAL

Oral teriflunomide was given at a starting dose of 7 mg once daily and danazol was given at a dose of 200mg twice daily for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.

Drug: Teriflunomide; Drug: Danazol

Danazol

ACTIVE COMPARATOR

Danazol was given at a dose of 200 mg twice a day for 24 weeks. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.

Drug: Danazol

Interventions

Teriflunomide DRUG

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

Also known as: AUBAGIO

Teriflunomide plus danazol

Danazol DRUG

200 mg twice daily.

Danazol; Teriflunomide plus danazol

Eligibility Criteria

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

You may qualify if:

• Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
• 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;
• 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:

• Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, or patients with confirmed autoimmune disease);
• Active or a history of malignancy;
• Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than two times the upper limit of normal (ULN);
• Pregnancy or lactation;
• Current or recent (\< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
• Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
• 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;
• Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled;
• 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 (1)

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

Beijing, Beijing Municipality, 100010, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

teriflunomide; Danazol

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)

Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Xiao-Hui Zhang, MD

  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: 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

Model Details: Patients were randomly assigned at a 1:1 ratio to receive teriflunomide plus danazol or danazol. Each group requires 62 patients (considering a 10% drop-off).

Study Record Dates

• First Submitted: December 6, 2023
• First Posted: December 20, 2023
• Study Start: December 5, 2023
• Primary Completion: December 30, 2024
• Study Completion: July 15, 2025
• Last Updated: December 20, 2023

Record last verified: 2023-12

Locations

CN (1)