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

NCT02094417 | Completed Phase 2 Results

• 1 other identifier: 531-KR001
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Aplastic Anemia

Keywords

Anemia, Aplastic; Thrombopoietin; Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• Proportion of Subjects Achieving a Platelet Response at Week 9

  The proportion of subjects achieving a platelet response at Week 9, and two-sided 95% confidence interval will be calculated. Platelet response is defined as 1) achieving absolute platelet increase of ≥ 20x10\^9/L above baseline or 2) increase to ≥ 10x10\^9/L and by at least 100% from baseline.

  At week 9

Secondary Outcomes (6)

• Proportion of Subjects Achieving a Platelet Response

  Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156

• Proportion of Subjects Achieving Platelet Transfusion Independency

  Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156

• Proportion of Subjects Achieving Erythroid Response and/or Neutrophil Response

  Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156

• Duration of Platelet Response and Time to Platelet Response

  Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156

• Proportion of Subjects Achieving Tri-lineage Responses

  Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 157

Study Arms (4)

AMG531 (Dose 1)

EXPERIMENTAL

Drug: AMG531

AMG531 (Dose 2)

EXPERIMENTAL

Drug: AMG531

AMG531 (Dose 3)

EXPERIMENTAL

Drug: AMG531

AMG531 (Dose 4)

EXPERIMENTAL

Drug: AMG531

Interventions

AMG531 DRUG

Subcutaneous, weekly injection

AMG531 (Dose 1); AMG531 (Dose 2); AMG531 (Dose 3); AMG531 (Dose 4)

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Kyowa Kirin Korea Co., Ltd.: lead

Study Sites (1)

Korea, Republic of

Seoul, South Korea

Location

Related Publications (2)

• 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.

  PMID: 40593138 DERIVED

• 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.

  PMID: 31474546 DERIVED

MeSH Terms

Conditions

Anemia, Aplastic; Jacobs syndrome; Thrombocytopenia

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Blood Platelet Disorders; Cytopenia

Results Point of Contact

• Title: Clinical Development Team, Clinical Development Department
• Organization: Kyowa Kirin Korea Co. Ltd.,

kyungmo.lee.m6@kyowakirin.com

82221912918

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 18, 2014
• First Posted: March 21, 2014
• Study Start: April 14, 2014
• Primary Completion: April 24, 2017
• Study Completion: November 7, 2017
• Last Updated: April 30, 2024
• Results First Posted: February 15, 2021

Record last verified: 2024-04

Locations

KR (1)