Clinical trial NCT06176911: The Combination of Teriflunomide and Danazol for Steroid-resistant/Relapse Immune Thrombocytopenia
Official title: A Multicenter, Open-label, Randomized, Controlled, Phase 2 Trial Comparing the Efficacy and Safety of Teriflunomide Plus Danazol in Patients With Steroid-resistant/Relapse ITP
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Keywords: Teriflunomide
Danazol
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
Collaborators: name: Beijing Luhe Hospital
name: Chinese PLA General Hospital
name: Navy General Hospital, Beijing
name: Beijing Hospital
name: Beijing Friendship Hospital
name: Peking University First Hospital
name: Peking University Third Hospital
name: China-Japan Friendship Hospital
name: Beijing Tsinghua Changgeng Hospital
First posted: 2023-12-20
Enrollment: 124
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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).
Primary purpose: TREATMENT

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

Detailed description: This is a prospective, multicenter, randomized, controlled trial of 124 adult patients with steroid-resistant/relapse ITP in China. Patients were randomized to receive either experimental teriflunomide plus danazol or active comparator danazol monotherapy. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.

Eligibility criteria: Inclusion Criteria:

1. Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia;
2. 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;
3. Patients with a platelet count \<30,000/μL or a platelet count \<50,000/μL with bleeding manifestations at the enrollment;
4. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, or patients with confirmed autoimmune disease);
2. Active or a history of malignancy;
3. Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than two times the upper limit of normal (ULN);
4. Pregnancy or lactation;
5. Current or recent (\< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
6. Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
7. 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;
8. 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;
9. 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: Time to response
timeFrame: From the start of study treatment (Day 1) to the end of week 24
measure: Duration of 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: 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
state: Beijing
Study officials:
affiliation: Peking University Institute of Hematology, Peking University People's Hospital
name: Xiao-Hui Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: