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出境医 / 临床实验 / Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

Study Description
Brief Summary:
To evaluate the hematological responses based on the response assessment criteria when AMG531 is subcutaneous (SC)-administered with anti-human thymocyte immunoglobulin (ATG) + ciclosporin A (CsA) therapy for 6 months in patients with aplastic anemia (AA) who were previously untreated with immunosuppressive therapy.

Condition or disease Intervention/treatment Phase
Aplastic Anemia Drug: Romiplostim Phase 2 Phase 3

Study Design
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 14 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: multi-national, open-label, phase 2/3 study
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy
Actual Study Start Date : April 25, 2019
Estimated Primary Completion Date : November 2020
Estimated Study Completion Date : December 2021
Arms and Interventions
Arm Intervention/treatment
Experimental: AMG531 Drug: Romiplostim
Romiplostim SC. Initial dose is 10 ug/kg/. Maximum dose is 20 ug/kg. Original duration is 6 months from first administration but if investigator decide that the patient is needed more treatment, they move to extention period and take treatment up to 1 year.
Outcome Measures
Primary Outcome Measures :
  1. Achievement of complete response (CR) or partial response (PR) [ Time Frame: 27 weeks post-dose ]

Secondary Outcome Measures :
  1. Achievement of CR or PR [ Time Frame: Week 14 ]
  2. Achievement of CR [ Time Frame: Weeks 14 and 27 ]
  3. The time to CR or PR [ Time Frame: Each time point evaluated weekly until Week 27 ]
  4. Reduction or independence of platelet and/or erythrocyte transfusion [ Time Frame: Week 27 ]
  5. Change from baseline in platelet count (/µL) [ Time Frame: Each time point evaluated weekly until Week 27 ]
  6. Change from baseline in hemoglobin (Hb) concentration (g/dL) [ Time Frame: Each time point evaluated weekly until Week 27 ]
  7. Change from baseline in neutrophil count (/µL) [ Time Frame: Each time point evaluated weekly until Week 27 ]
  8. Change from baseline in reticulocyte count (/µL) [ Time Frame: Each time point evaluated weekly until Week 27 ]

Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Voluntary signed informed consent to participate in the study;
  2. A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;
  3. Considered to require new treatment with ATG and CsA provided that NSAA must be platelet or erythrocyte transfusion-dependent.
  4. An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening

Exclusion Criteria:

  1. Previously treated with ATG, CsA, or Alemtuzumab;
  2. Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
  3. Diagnosed as having AML or chronic myelomonocytic leukemia;
  4. Concurrent thrombocytopenia of other etiologies (e.g., MDS, ITP, cirrhosis);
  5. Concurrent active infection not adequately responding to appropriate therapy;
  6. Concurrent clinically significant illness(es) items which are deemed by the Investigator to be likely to affect the study conduct and assessments.
  7. Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.
  8. Concurrent PNH
  9. Having Grade 2 or higher bone marrow reticulin based on Bone Marrow Pathology (2nd edition) ;
  10. History of chromosome aberrations discovered in bone marrow cells.
  11. Having blast cells > 2% in bone marrow;
  12. Positive for anti-human immunodeficiency virus (HIV) antibody;
  13. Receiving prophylactic or therapeutic treatment for hepatitis type B
  14. Positive for hepatitis C virus (HCV) antibody, and HCV infection being confirmed
  15. Planned hematopoietic stem cell transplantation during the study;

  16. Systemic treatment with any of the following medication for the treatment of AA within 4 weeks before Day 1, however, excluding their use as premedication:

    • Anabolic steroids
    • Corticosteroids;
  17. Pregnant or breastfeeding women, or women willing to become pregnant;
  18. Other conditions unsuitable for participation in the study in the opinion of the Investigator.
Contacts and Locations

Locations
Layout table for location information
Japan
Matsuyama Red Cross Hospital
Ehime, Japan
Sponsors and Collaborators
Kyowa Kirin Co., Ltd.
Tracking Information
First Submitted Date  ICMJE May 9, 2019
First Posted Date  ICMJE May 21, 2019
Last Update Posted Date August 3, 2020
Actual Study Start Date  ICMJE April 25, 2019
Estimated Primary Completion Date November 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 19, 2019) 		Achievement of complete response (CR) or partial response (PR) [ Time Frame: 27 weeks post-dose ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 19, 2019)
  • Achievement of CR or PR [ Time Frame: Week 14 ]
  • Achievement of CR [ Time Frame: Weeks 14 and 27 ]
  • The time to CR or PR [ Time Frame: Each time point evaluated weekly until Week 27 ]
  • Reduction or independence of platelet and/or erythrocyte transfusion [ Time Frame: Week 27 ]
  • Change from baseline in platelet count (/µL) [ Time Frame: Each time point evaluated weekly until Week 27 ]
  • Change from baseline in hemoglobin (Hb) concentration (g/dL) [ Time Frame: Each time point evaluated weekly until Week 27 ]
  • Change from baseline in neutrophil count (/µL) [ Time Frame: Each time point evaluated weekly until Week 27 ]
  • Change from baseline in reticulocyte count (/µL) [ Time Frame: Each time point evaluated weekly until Week 27 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of AMG531(Romiplostim) in Patients With Aplastic Anemia
Official Title  ICMJE A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy
Brief Summary To evaluate the hematological responses based on the response assessment criteria when AMG531 is subcutaneous (SC)-administered with anti-human thymocyte immunoglobulin (ATG) + ciclosporin A (CsA) therapy for 6 months in patients with aplastic anemia (AA) who were previously untreated with immunosuppressive therapy.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
multi-national, open-label, phase 2/3 study
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Aplastic Anemia
Intervention  ICMJE Drug: Romiplostim
Romiplostim SC. Initial dose is 10 ug/kg/. Maximum dose is 20 ug/kg. Original duration is 6 months from first administration but if investigator decide that the patient is needed more treatment, they move to extention period and take treatment up to 1 year.
Study Arms  ICMJE Experimental: AMG531
Intervention: Drug: Romiplostim
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: May 19, 2019) 		14
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2021
Estimated Primary Completion Date November 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Voluntary signed informed consent to participate in the study;
  2. A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;
  3. Considered to require new treatment with ATG and CsA provided that NSAA must be platelet or erythrocyte transfusion-dependent.
  4. An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening

Exclusion Criteria:

  1. Previously treated with ATG, CsA, or Alemtuzumab;
  2. Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
  3. Diagnosed as having AML or chronic myelomonocytic leukemia;
  4. Concurrent thrombocytopenia of other etiologies (e.g., MDS, ITP, cirrhosis);
  5. Concurrent active infection not adequately responding to appropriate therapy;
  6. Concurrent clinically significant illness(es) items which are deemed by the Investigator to be likely to affect the study conduct and assessments.
  7. Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.
  8. Concurrent PNH
  9. Having Grade 2 or higher bone marrow reticulin based on Bone Marrow Pathology (2nd edition) ;
  10. History of chromosome aberrations discovered in bone marrow cells.
  11. Having blast cells > 2% in bone marrow;
  12. Positive for anti-human immunodeficiency virus (HIV) antibody;
  13. Receiving prophylactic or therapeutic treatment for hepatitis type B
  14. Positive for hepatitis C virus (HCV) antibody, and HCV infection being confirmed
  15. Planned hematopoietic stem cell transplantation during the study;

  16. Systemic treatment with any of the following medication for the treatment of AA within 4 weeks before Day 1, however, excluding their use as premedication:

    • Anabolic steroids
    • Corticosteroids;
  17. Pregnant or breastfeeding women, or women willing to become pregnant;
  18. Other conditions unsuitable for participation in the study in the opinion of the Investigator.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 20 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Japan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03957694
Other Study ID Numbers  ICMJE 531-003
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Kyowa Kirin Co., Ltd.
Study Sponsor  ICMJE Kyowa Kirin Co., Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Kyowa Kirin Co., Ltd.
Verification Date July 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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