A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia

NCT ID: NCT02094417

Last Updated: 2024-04-30

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-04-14

Study Completion Date

2017-11-07

Brief Summary

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The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the Aplastic Anemia (AA) patients with immunosuppressive-therapy refractory thrombocytopenia and also to assess the pharmacokinetics of this product. Its efficacy and safety during the extension period beyond one year will also be evaluated.

Detailed Description

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Conditions

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Aplastic Anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AMG531 (Dose 1)

Group Type EXPERIMENTAL

AMG531

Intervention Type DRUG

Subcutaneous, weekly injection

AMG531 (Dose 2)

Group Type EXPERIMENTAL

AMG531

Intervention Type DRUG

Subcutaneous, weekly injection

AMG531 (Dose 3)

Group Type EXPERIMENTAL

AMG531

Intervention Type DRUG

Subcutaneous, weekly injection

AMG531 (Dose 4)

Group Type EXPERIMENTAL

AMG531

Intervention Type DRUG

Subcutaneous, weekly injection

Interventions

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AMG531

Subcutaneous, weekly injection

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Patient who is diagnosed as AA and refractory to immunosuppressive therapy
* Platelet ≤ 30,000/μL

Exclusion Criteria

* Concurrent active infection not adequately responding to appropriate therapy
* HIV positivity
* Bone marrow reticulin grade of \> 1
* Clinically significant cardiac disease
* Arterial or venous thrombosis within the last 1 year before enrollment
* Other cause of thrombocytopenia
* AA with hemolytic predominant paroxysmal nocturnal hemoglobinuria (PNH)
* Uncontrolled diabetes
* Receiving any agent used to treat AA, including antithymocyte globulin (ATG) or ATG + cyclosporine within 6 months before starting study treatment and/or cyclosporine or anabolic hormone within 6 weeks before starting the study treatment
* History of PEG-rHuMGDF, recombinant human thrombopoietin, AMG531, and other thrombopoietin (TPO)-receptor agonist
* Who plans to conduct hematopoietic stem cell transplantation within 1 year
Minimum Eligible Age

19 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Kyowa Kirin Korea Co., Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Korea, Republic of

Seoul, , South Korea

Site Status

Countries

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South Korea

References

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Jang JH, Mitani K, Tomiyama Y, Miyazaki K, Nagafuji K, Usuki K, Uoshima N, Fujisaki T, Kosugi H, Matsumura I, Sasaki K, Kizaki M, Sawa M, Hidaka M, Kobayashi N, Ichikawa S, Yonemura Y, Murotani K, Shimizu M, Matsuda A, Ozawa K, Nakao S, Lee JW. Predictive factors of romiplostim response in patients with refractory aplastic anemia: data from two clinical trials. Ann Hematol. 2025 Aug;104(8):4003-4011. doi: 10.1007/s00277-025-06337-7. Epub 2025 Jul 1.

Reference Type DERIVED
PMID: 40593138 (View on PubMed)

Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, Kim JA, Oh IH, Jang JH. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019 Nov;6(11):e562-e572. doi: 10.1016/S2352-3026(19)30153-X. Epub 2019 Aug 29.

Reference Type DERIVED
PMID: 31474546 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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531-KR001

Identifier Type: -

Identifier Source: org_study_id

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