The Combination of Tacrolimus and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia

NCT05471050 | Unknown Phase 2 DMC

• 1 other identifier: PKU-ITP036
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

Randomized, open-label, multicenter study to compare the efficacy and safety of combination of tacrolimus and danazol versus danazol for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).

Conditions

Immune Thrombocytopenia

Keywords

Immune Thrombocytopenia; tacrolimus; danazol

Outcome Measures

Primary Outcomes (1)

• Sustained response

  Sustained response was defines as the maintenance of platelet count ≥ 30 x 10\^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication at the 6-month follow-up.

  6 months

Secondary Outcomes (5)

• Complete response

  6 months

• Response

  6 months

• Time to response

  6 months

• Duration of response

  6 months

• Adverse events

  6 months

Study Arms (2)

tacrolimus and Danazol

EXPERIMENTAL

Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.

Drug: TAC; Drug: Danazol

Danazol

ACTIVE COMPARATOR

Danazol is given at 200mg bid for 12 weeks.

Drug: Danazol

Interventions

TAC DRUG

Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.

tacrolimus and Danazol

Danazol DRUG

Danazol is given at 200mg bid for 12 weeks.

Danazol; tacrolimus and Danazol

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Primary immune thrombocytopenia (ITP);
• years older;
• Platelet count of less than 30×10\^9/L at 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;
• Patients who were receiving other maintenance regimens, primarily corticosteroids, ciclosporin, or mycophenolate mofetil, were also eligible if the dose of treatment had been stable in the past month and the dose was expected to be stable after enrolment and remained unchanged at least for the first 4 weeks of study until initial response was assessed, unless severe adverse events were suspected.

You may not qualify if:

• Secondary ITP (e.g., patients with HIV, HBV, HCV, Helicobacter pylori infection or SLE);
• Congestive heart failure, severe arrhythmia;
• Nursing or pregnant women;
• ALT or AST levels ≥ 3× the upper limit of the normal threshold;
• Creatinine or serum bilirubin levels ≥ 1.5× the upper limit;
• Active or previous malignancy ;
• Patients who had received danazol treatment or did not respond to danazol;
• Patients unable to have routine blood tests because of reasons such as insufficient time.

Sponsors & Collaborators

• Peking University People's Hospital: lead

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

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 People's Hospital, Peking University Insititute of Hematology

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiao-Hui Zhang, MD

CONTACT

+8613522338836; zhangxh@bjmu.edu.cn

Xuan Cai, MD

CONTACT

+8618811729606; 2206385254@qq.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: The investigators are undertaking a randomized controlled trial of 120 adults with steroid-resistant/ relapse ITP in China. Patients were randomized to tacrolimus plus danazol and danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Study Record Dates

• First Submitted: July 17, 2022
• First Posted: July 22, 2022
• Study Start: March 2, 2022
• Primary Completion: March 1, 2023
• Study Completion: June 1, 2023
• Last Updated: July 22, 2022

Record last verified: 2022-07

Locations

CN (1)