Clinical Trials

Numerous clinical trials, spanning Phase I through Phase IV, evaluate avatrombopag across indications including chemotherapy-induced thrombocytopenia, primary immune thrombocytopenia, refractory aplastic anemia, and liver failure accompanied by low platelet counts. Sponsored by academic research centers and commercial entities, these international protocols span recruitment statuses of actively recruiting, completed, unknown, and withdrawn. Active Phase II studies focus on chemotherapy-induced thrombocytopenia in gastrointestinal neoplasms alongside refractory aplastic anemia.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07278908 NOT_YET_RECRUITING
ITP - Immune Thrombocytopenia
Institute of Hematology & Blood Diseases Hospital, China
 2025-12 PHASE2; PHASE3
NCT07786922 NOT_YET_RECRUITING
Aplastic Anemia
Swedish Orphan Biovitrum
 2026-07-31 PHASE2; PHASE3
NCT07051915 NOT_YET_RECRUITING
Thrombocytopenia Purpura
Ostfold Hospital Trust
 2025-09-15 PHASE3
NCT07133659 RECRUITING
ITP - Immune Thrombocytopenia
Al-Mustansiriyah University
 2025-06-08 PHASE4
NCT05772546 COMPLETED
Gastrointestinal Cancer; Gastrointestinal Neoplasms; Chemotherapy-Induced Thrombocytopenia
Hanny Al-Samkari, MD
 2023-11-01 PHASE2
NCT06281327 RECRUITING
Immune Thrombocytopenia; Treatment
Institute of Hematology & Blood Diseases Hospital, China
 2024-03-01 PHASE2
NCT05518331 UNKNOWN
Refractory Aplastic Anemia
Institute of Hematology & Blood Diseases Hospital, China
 2022-06-01
NCT05996393 COMPLETED
Aplastic Anemia
Peking Union Medical College Hospital
 2023-09-15 PHASE4
NCT05218226 UNKNOWN
Chemotherapy-induced Thrombocytopenia; Avatrombopag
Institute of Hematology & Blood Diseases Hospital, China
 2022-02-10 PHASE2
NCT06254287 UNKNOWN
Anemia, Aplastic
Institute of Hematology & Blood Diseases Hospital, China
 2024-02-01 PHASE2
NCT06202625 RECRUITING
Thrombocytopenia; Stem Cell Transplant Complications
Peking University People's Hospital
 2024-05-13 PHASE2; PHASE3
NCT06001567 UNKNOWN
Thrombocytopenia; Hepatocellular Carcinoma
Second Affiliated Hospital of Guangzhou Medical University
 2023-08-14 PHASE2
NCT06004752 UNKNOWN
Aplastic Anemia
Peking Union Medical College Hospital
 2023-08-17 PHASE2
NCT04993885 RECRUITING
Immune Thrombocytopenia; Autoantibodies; Evan Syndrome; Connective Tissue Diseases
Institute of Hematology & Blood Diseases Hospital, China
 2021-09-01 PHASE2
NCT05046327 WITHDRAWN
Immune Thrombocytopenia
Sobi, Inc.
 2021-10-14 PHASE3
NCT05382013 UNKNOWN
Thrombocytopenia; Hepatitis B, Chronic; Acute-On-Chronic Liver Failure
Third Affiliated Hospital, Sun Yat-Sen University
 2022-04-27 PHASE4
NCT05369208 COMPLETED
Immune Thrombocytopenia
Sobi, Inc.
 2022-06-15 PHASE3
NCT04638829 COMPLETED
Immune Thrombocytopenia
Sobi, Inc.
 2021-03-15 PHASE4
NCT05433922 UNKNOWN
Severe Aplastic Anemia
Institute of Hematology & Blood Diseases Hospital, China
 2022-06-01
NCT05571332 UNKNOWN
Aplastic Anemia
Institute of Hematology & Blood Diseases Hospital, China
 2022-06-28
NCT05720234 UNKNOWN
Severe Aplastic Anemia
Institute of Hematology & Blood Diseases Hospital, China
 2022-11-10 PHASE2
NCT04516967 COMPLETED
Immune Thrombocytopenia
Sobi, Inc.
 2021-03-05 PHASE3
NCT05772546 Recruiting
Gastrointestinal Cancer|Gastrointestinal Neoplasms|Chemotherapy-Induced Thrombocytopenia
Hanny Al-Samkari MD|Swedish Orphan Biovitrum|Massachusetts General Hospital
 2023-11-01 Phase 2
NCT04931849 WITHDRAWN
Thrombocytopenia
University of Rochester
 2022-05-06 EARLY_PHASE1
NCT04915287 UNKNOWN
Thrombocytopenia; Drugs; Chronic Liver Disease
Chinese PLA General Hospital
 2021-06-06 PHASE4
NCT04797182 UNKNOWN
Thrombocytopenia; Cytarabine Causing Adverse Effects in Therapeutic Use; Lymphoma
Sun Yat-sen University
 2021-04 PHASE2
NCT04906083 UNKNOWN
Liver Failure; Thrombocytopenia; Decompensated Cirrhosis
Tongji Hospital
 2021-02-01 PHASE4
NCT05143892 UNKNOWN
Platelet Disorder
The First Affiliated Hospital of Soochow University
 2021-12-01 PHASE2
NCT04312789 WITHDRAWN
Platelet Disorder
Ayman Saad
 2022-12 PHASE2
NCT04728789 UNKNOWN
Aplastic Anemia; Drug Effect
Peking Union Medical College Hospital
 2021-02-01 PHASE2; PHASE3
NCT04312789 Withdrawn
Platelet Disorder
Ayman Saad|Sobi Inc.|Ohio State University Comprehensive Cancer Center
 2022-12 Phase 2
NCT05518331 Recruiting
Refractory Aplastic Anemia
Institute of Hematology & Blood Diseases Hospital China|Peking University People''s Hospital|Xiyuan Hospital of China Academy of Chinese Medical Sciences|Second Hospital of Shanxi Medical University|First Affiliated Hospital of Harbin Medical University|The First Hospital of Hebei Medical University
 2022-06-01 Not Applicable
NCT05218226 Recruiting
Chemotherapy-induced Thrombocytopenia|Avatrombopag
Institute of Hematology & Blood Diseases Hospital China|Tianjin Medical University Cancer Institute and Hospital|Tianjin Medical University Second Hospital|Tianjin Third Central Hospital|Tianjin People''s Hospital|Henan Cancer Hospital|The Second Affiliated Hospital of Kunming Medical University
 2022-02-10 Phase 2
NCT04896528 UNKNOWN
Thrombocytopenia; Avatrombopag
Peking Union Medical College Hospital
 2021-06-06 PHASE2
NCT04609891 COMPLETED
Thrombocytopenia
Anhui Provincial Cancer Hospital
 2020-05-26
NCT04949009 Unknown status
Primary Immune Thrombocytopenia
Shandong University
 2021-03-01 --
NCT04437953 WITHDRAWN
Thrombocytopenia; Cancer; Liver Diseases
Memorial Sloan Kettering Cancer Center
 2020-05-27 PHASE2
NCT03471078 COMPLETED
Chemotherapy-induced Thrombocytopenia
Sobi, Inc.
 2018-10-12 PHASE3
NCT04609891 Completed
Thrombocytopenia
Anhui Provincial Cancer Hospital
 2020-05-26 Not Applicable
NCT03326843 TERMINATED
Thrombocytopenia
Sobi, Inc.
 2018-03-26 PHASE3
NCT01976104 COMPLETED
Thrombocytopenia Associated With Liver Disease
Eisai Inc.
 2013-11 PHASE3
NCT01972529 COMPLETED
Thrombocytopenia Associated With Liver Disease
Eisai Inc.
 2014-02 PHASE3
NCT02809768 COMPLETED
Healthy Participants
Eisai Inc.
 2016-04 PHASE1
NCT02227693 COMPLETED
Thrombocytopenia Associated With Chronic Liver Disease
Eisai Co., Ltd.
 2014-07 PHASE2
NCT02039076 COMPLETED
Thrombocytopenia
Eisai Inc.
 2013-12 PHASE1
NCT01355289 COMPLETED
Thrombocytopenia
Eisai Inc.
 2011-11 PHASE2
NCT01438840 COMPLETED
Chronic Thrombocytopenia; Immune Thrombocytopenia
Eisai Inc.
 2012-02-16 PHASE3
NCT01433978 TERMINATED
Idiopathic Thrombocytopenic Purpura
Eisai Inc.
 2012-03-26 PHASE3
NCT01759394 COMPLETED
Healthy Volunteers
Eisai Inc.
 2012-10 PHASE1
NCT00914927 COMPLETED
Thrombocytopenia Related to Chronic Liver Disease
Eisai Inc.
 2009-05 PHASE2
NCT00625443 COMPLETED
Idiopathic Thrombocytopenic Purpura
Eisai Inc.
 2007-05 PHASE2
NCT00441090 COMPLETED
Chronic Idiopathic Thrombocytopenic Purpura; Purpura, Thrombocytopenic, Idiopathic
Eisai Inc.
 2007-02 PHASE2

(data from https://clinicaltrials.gov, updated on 2025-12-12)

Check the Avatrombopag product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Avatrombopag selectively binds to and activates the transmembrane domain of the human thrombopoietin receptor (c-Mpl), triggering intracellular signal transduction pathways that stimulate megakaryocyte proliferation and differentiation without inducing intrinsic platelet activation. This target-specific expansion of megakaryocytes elevates circulating platelet counts, providing therapeutic efficacy in clinical indications such as chemotherapy-induced thrombocytopenia, primary immune thrombocytopenia, and liver disease-associated thrombocytopenia.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.