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

Immune Thrombocytopenia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04481282
• Other study ID #: ITP-PKU010
• Status: Recruiting
• Phase: Phase 2
• Start date: July 8, 2020
• Est. completion date: July 31, 2021

Study information

• Verified date: July 2020
• Source: Peking University People's Hospital
• Contact: Xiaohui Zhang, MD
• Phone: 010-88324981
• Email: zhangxh100[@]sina.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Single-arm, open-label, single center study to assess the efficacy and safety of terbutaline plus danazol in patients with corticosteroid resistant/relapsed ITP.

Description:

Immune thrombocytopenia (ITP) is a severe bleeding disorder. Approximately 2/3 of patients achieve remission from first-line therapies. However, the underlying mechanism of corticosteroid-resistant or relapsed ITP is not well understood; thus, treatment remains a great challenge. β2-AR agonist terbutaline modulates T cell differentiation and effector cell function.

A single center prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were assigned to terbutaline plus danazol group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study, in order to assess the efficacy and safety of terbutaline plus danazol in patients with corticosteroid-resistant/relapsed ITP.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: July 31, 2021
• Est. primary completion date: March 31, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• ITP confirmed by excluding other supervened causes of thrombocytopenia;

• 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;

• Subject has signed and dated written informed consent.

• Fertile patients must use effective contraception during treatment and observational period

• Negative pregnancy test.

Exclusion Criteria:

• Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)

• congestive heart failure

• severe arrhythmia

• nursing or pregnant women

• aspartate aminotransferase and alanine transaminase levels = 3× the upper limit of the normal threshold criteria

• creatinine or serum bilirubin levels each 1•5 times or more than the normal range

• active or previous malignancy

• Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.

• diagnosis with any of the following diseases: chronic hypertension, hyperthyroidism, diabetes, or seizure disorder.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Terbutaline
2.5mg po tid for 12 weeks
• Danazol
200mg po bid for 12 weeks

Locations

Country	Name	City	State
China	Peking University Insititute of Hematology, Peking University People's Hospital	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Peking University People's Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Sustained response	The maintenance of platelet count = 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up. Interim analysis was scheduled at 50% through recruitment.	6 months
Secondary	complete remission	The number of participants (responders) with platelet count>=100x10^9/L (CR) and the absence of bleeding.	6 months
Secondary	partial remission	The number of participants (responders) with platelet count >=30x10^9/L and at least a 2-fold increase in the baseline count (PR) without the administration of any other platelet increasing therapy.	6 months
Secondary	time to response	Time to response was defined as the time from starting treatment to the time to achieve the response. Interim analysis	6 months
Secondary	duration of response	Duration of response was measured from the achievement of response to the loss of response.	6 months
Secondary	incidence of treatment-emergent adverse events	Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.	6 months