The Combination of Terbutaline and Danazol as the Treatment of Corticosteroid-resistant/Relapse Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

Official title:

A Multicenter Randomized Trial of Second Line Treatment for Corticosteroid-Resistant or Relapsed Immune Thrombocytopenia: Combined Terbutaline and Danazol Versus Danazol Monotherapy

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05494307
• Other study ID #: PKU-ITP039
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: September 1, 2022
• Est. completion date: February 28, 2024

Study information

• Verified date: August 2022
• Source: Peking University People's Hospital
• Contact: Ye-Jun Wu, MD
• Phone: 13522338836
• Email: wyejun1999[@]pku.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A randomized, open-label, multicenter study to compare the efficacy and safety of terbutaline plus danazol compared to danazol monotherapy for the second-line treatment of adults with corticosteroid-resistant or relapsed primary immune thrombocytopenia (ITP).

Description:

The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 228 adults with ITP in China. Patients were randomized to terbutaline plus danazol compared to danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 228
• Est. completion date: February 28, 2024
• Est. primary completion date: September 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

Inclusion Criteria: Patients >18 years old with corticosteroid-resistant or relapsed ITP who had either a platelet count of <30×10^9/L or a platelet count of <50×10^9/L and clinically significant bleeding.

1. Not achieving a sustained response to therapy with full-dose corticosteroids for a duration of at least 4 weeks or relapsed during the process of corticosteroid tapering or discontinuation;

2. Platelet counts <30×10^9/L or platelet counts < 50×10^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);

3. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Pregnant or lactating women;

2. Secondary ITP (have a known diagnosis of connective tissue diseases, malignancy, active infection, HIV infections or hepatitis B virus or hepatitis C virus infections);

3. Received drugs affecting the platelet counts within 6 months before the screening visit (e.g., chemotherapy, anticoagulants, etc);

4. Severe medical condition (lung, heart, hepatic or renal disorder);

5. Patients who are deemed unsuitable for the study by the investigator.

6. Patients who had hypertension, diabetes mellitus, hyperthyroidism or coronary heart disease.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Terbutaline
Orally terbutaline at a dose of 2.5 mg three times daily for 16 weeks
• Danazol
Orally danazol at a dose of 200 mg twice daily for 16 weeks

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Peking University People's Hospital

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall response	Achieving a platelet count = 30 × 10^9/L confirmed on at least two separate occasions (at least 7 days apart), at least a doubling of the baseline platelet count without any other ITP-specific treatment and the absence of bleeding.	From date of randomization until 1 years or the end of follow-up
Secondary	Sustained response	maintenance of a platelet count > 30 × 10^9/L, an absence of bleeding, and no requirement for any other ITP-specific treatment for 6 consecutive months after achievement of OR	From date of randomization until 1 years or the end of follow-up
Secondary	Complete response	a platelet count = 100 × 10^9/L measured on two occasions at least 7 days apart and the absence of bleeding	From date of randomization until 1 years or the end of follow-up
Secondary	Remission	a durable platelet count > 30 × 109/L without bleeding up to 12 months after randomization	at 12-month follow-up
Secondary	Time to response	the time from starting treatment to the time a response was achieved.	From date of randomization until 1 years or the end of follow-up
Secondary	Duration of response	time from OR until loss of response or until the last follow-up visit	From date of randomization until 1 years or the end of follow-up
Secondary	Rescue therapy	any new medical intervention taken to increase the platelet count or prevent bleeding events or an increase in the dose of concomitant treatments	From date of randomization until 1 years or the end of follow-up
Secondary	Associated factors of treatment failure, OR, SR and remission	Factors that are associated with treatment failure, OR, SR and remission	From date of randomization until 1 years or the end of follow-up
Secondary	Number of patients with bleeding	Number of patients with bleeding complication (WHO bleeding score)	From date of randomization until 1 years or the end of follow-up
Secondary	Number of patients with side effects	Number of patients with Medication adverse events.	From date of randomization until 1 years or the end of follow-up
Secondary	Relapse	Loss of OR	From date of randomization until 1 years or the end of follow-up
Secondary	Relapse-free survival	the time interval between achievement of OR and relapse or the end of the follow-up	From date of randomization until 1 years or the end of follow-up