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Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03957694
Recruitment Status : Completed
First Posted : May 21, 2019
Last Update Posted : May 26, 2022
Sponsor:
Information provided by (Responsible Party):
Kyowa Kirin Co., Ltd.

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 17 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
Actual Primary Completion Date : November 5, 2020
Actual Study Completion Date : May 26, 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Romiplostim

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.




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 ]


Information from the National Library of Medicine

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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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03957694


Locations
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Japan
Matsuyama Red Cross Hospital
Ehime, Japan
Sponsors and Collaborators
Kyowa Kirin Co., Ltd.
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Responsible Party: Kyowa Kirin Co., Ltd.
ClinicalTrials.gov Identifier: NCT03957694    
Other Study ID Numbers: 531-003
First Posted: May 21, 2019    Key Record Dates
Last Update Posted: May 26, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Kyowa Kirin Co., Ltd.:
aplastic anemia
romiplostim
Additional relevant MeSH terms:
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Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Failure Disorders
Bone Marrow Diseases