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

Primary Purpose

Immune Thrombocytopenia

Status
Unknown status
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Romiplostim and danazol
Sponsored by
Seoul National University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Immune Thrombocytopenia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Romiplostim and danazol

    Arm Description

    Treatment group (romiplostim and danazol)

    Outcomes

    Primary Outcome Measures

    Response rate
    proportion of patients with platelet count over 50x10^9/L

    Secondary Outcome Measures

    Time to best response
    time interval between initiation of treatment and best response
    Response duration
    period of time with platelet count over 50x10^9/L
    Effects on quality of life
    changes in EORTC QLQ-C30 scores

    Full Information

    First Posted
    February 25, 2020
    Last Updated
    February 26, 2020
    Sponsor
    Seoul National University Hospital
    Collaborators
    Kyowa Kirin Korea Co., Ltd.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04289207
    Brief Title
    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

    2. Study Status

    Record Verification Date
    February 2020
    Overall Recruitment Status
    Unknown status
    Study Start Date
    March 1, 2020 (Anticipated)
    Primary Completion Date
    September 1, 2020 (Anticipated)
    Study Completion Date
    March 1, 2021 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Seoul National University Hospital
    Collaborators
    Kyowa Kirin Korea Co., Ltd.

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Product Manufactured in and Exported from the U.S.
    No
    Data Monitoring Committee
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Immune Thrombocytopenia

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    55 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Romiplostim and danazol
    Arm Type
    Experimental
    Arm Description
    Treatment group (romiplostim and danazol)
    Intervention Type
    Drug
    Intervention Name(s)
    Romiplostim and danazol
    Intervention Description
    Treatment with romiplostim and danazol
    Primary Outcome Measure Information:
    Title
    Response rate
    Description
    proportion of patients with platelet count over 50x10^9/L
    Time Frame
    week 8
    Secondary Outcome Measure Information:
    Title
    Time to best response
    Description
    time interval between initiation of treatment and best response
    Time Frame
    week 24
    Title
    Response duration
    Description
    period of time with platelet count over 50x10^9/L
    Time Frame
    week 24
    Title
    Effects on quality of life
    Description
    changes in EORTC QLQ-C30 scores
    Time Frame
    week 24

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Youngil Koh
    Phone
    82-02-2072-7217
    Email
    go01@snu.ac.kr
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Youngil Koh
    Organizational Affiliation
    Seoul National University Hospital
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    No
    Citations:
    PubMed Identifier
    16643442
    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.
    Results Reference
    background
    PubMed Identifier
    18981291
    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. Erratum In: Blood. 2009 May 7;113(19):4822.
    Results Reference
    background
    PubMed Identifier
    29042367
    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.
    Results Reference
    background
    PubMed Identifier
    23445876
    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.
    Results Reference
    background

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

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