Clinical trial NCT04289207: Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia
Official title: Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Lead sponsor: Seoul National University Hospital
Sponsor class: OTHER
Collaborators: name: Kyowa Kirin Korea Co., Ltd.
First posted: 2020-02-28
Enrollment: 55
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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

Eligibility criteria: 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

Primary outcomes:
measure: Response rate
timeFrame: week 8
Secondary outcomes:
measure: Time to best response
timeFrame: week 24
measure: Response duration
timeFrame: week 24
measure: Effects on quality of life
timeFrame: week 24
Other outcomes:
Locations:
Study officials:
affiliation: Seoul National University Hospital
name: Youngil Koh
role: PRINCIPAL_INVESTIGATOR
References:
citation: Cooper N, Bussel J. The pathogenesis of immune thrombocytopaenic purpura. Br J Haematol. 2006 May;133(4):364-74. doi: 10.1111/j.1365-2141.2006.06024.x.
citation: 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.
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.
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.