Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

Official title:

Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04289207
• Other study ID #: 0620200130
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: March 1, 2020
• Est. completion date: March 1, 2021

Study information

• Verified date: February 2020
• Source: Seoul National University Hospital
• Contact: Youngil Koh
• Phone: 82-02-2072-7217
• Email: go01[@]snu.ac.kr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

In this study, we tried to demonstrate clinical benefit of additive danazol to romiplostim therapy in patients with eltrombopag-resistant immune thrombocytopenia

Description:

The patients with eltrombopag-resistant immune thrombocytopenia are treated with romiplostim and danazol. The efficacy and toxicity of treatment will be evaluated.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 55
• Est. completion date: March 1, 2021
• Est. primary completion date: September 1, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• aged over 18 years

• diagnosed with immune thrombocytopenia (bone marrow examination is not necessary)

• failed to achieve platelet count over 50X10^9/L with eltrombopag

• ECOG performance status 0,1,2

• available to obtain informed consent

Exclusion Criteria:

• Hepatitis B or C carriers

• HIV positive patients

• diagnosed with systemic lupus erythematosus or other autoimmune disorders

• unable to intake orally or absorb through gastrointestinal tract

• pregnant or breast-feeding

• diagnosed with uncontrolled seizure or other neuropsychiatric disorders

• diagnosed with clinically significant cardiovascular events within 6 months or dyspnea on exertion evaluated to New York Heart Association Functional Classification III or IV

• diagnosed with clinically significant cerebrovascular disorders

• previously diagnosed or treated with thromboembolism

• current treating malignant diseases

• currently accompanied by uncontrolled infection or active bleeding

• with blood test results as follows; total bilirubin > 2xUNL(upper normal limit), AST/ALT > 1.5xUNL, creatinine > 1.5xUNL, glomerular filtration rate < 30ml/min/1.73m^2

• registered to other clinical trials for treatment of immune thrombocytopenia

• judged to be inappropriate for clinical trial by doctor in charge

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Romiplostim and danazol
Treatment with romiplostim and danazol

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Seoul National University Hospital	Kyowa Kirin Korea Co., Ltd.

References & Publications (4)

Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009 Mar 5;113(10):2161-71. doi: 10.1182/blood-2008-04-150078. Epub 2008 Nov 3. Erratum in: Blood. 2009 May 7;113(19):4822. — View Citation

Cooper N, Bussel J. The pathogenesis of immune thrombocytopaenic purpura. Br J Haematol. 2006 May;133(4):364-74. Review. — View Citation

Khellaf M, Viallard JF, Hamidou M, Cheze S, Roudot-Thoraval F, Lefrere F, Fain O, Audia S, Abgrall JF, Michot JM, Dauriac C, Lefort S, Gyan E, Niault M, Durand JM, Languille L, Boutboul D, Bierling P, Michel M, Godeau B. A retrospective pilot evaluation of switching thrombopoietic receptor-agonists in immune thrombocytopenia. Haematologica. 2013 Jun;98(6):881-7. doi: 10.3324/haematol.2012.074633. Epub 2013 Feb 26. — View Citation

Wong RSM, Saleh MN, Khelif A, Salama A, Portella MSO, Burgess P, Bussel JB. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017 Dec 7;130(23):2527-2536. doi: 10.1182/blood-2017-04-748707. Epub 2017 Oct 17. Erratum in: Blood. 2018 Feb 8;131(6):709. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Response rate	proportion of patients with platelet count over 50x10^9/L	week 8
Secondary	Time to best response	time interval between initiation of treatment and best response	week 24
Secondary	Response duration	period of time with platelet count over 50x10^9/L	week 24
Secondary	Effects on quality of life	changes in EORTC QLQ-C30 scores	week 24