A Phase 2/3 Study of AMG531 in Patients with Aplastic Anemia

Phase 2

Completed

• Conditions: Aplastic Anemia

• Registration Number: JPRN-jRCT2080224673
• Lead Sponsor: Kyowa Kirin Co., Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-05-09
• Study Completion: 2022-05-10
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: completed
• Sex: All
• Target Recruitment: 14

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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified