Clinical Trials

Numerous clinical trials across Phase I, Phase II, and Phase III evaluate homoharringtonine protocols for acute myeloid leukemia, acute lymphoblastic leukemia, hematologic tumors, prostate cancer, and hereditary hemorrhagic telangiectasia with associated epistaxis. Supported by academic medical centers and industry sponsors, these studies encompass various recruitment statuses, including recruiting, active not recruiting, enrolling by invitation, completed, and terminated.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06855810 RECRUITING
Acute Lymphoblastic Leukemia; Childhood Leukemia, Acute Lymphoblastic; T Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma
Institute of Hematology & Blood Diseases Hospital, China
2025-03-11 PHASE2; PHASE3
NCT07651176 ENROLLING_BY_INVITATION
Acute Myeloid Leukemia; Lisaftoclax
First Affiliated Hospital of Zhejiang University
2026-03-01 PHASE2; PHASE3
NCT07651163 ENROLLING_BY_INVITATION
Acute Myeloid Leukemia (AML); Lisaftoclax
First Affiliated Hospital of Zhejiang University
2026-03-01 PHASE2; PHASE3
NCT07111598 RECRUITING
Hereditary Haemorrhagic Telangiectasia
Hospices Civils de Lyon
2025-09-24
NCT06221683 RECRUITING
AML, Childhood; Acute Myeloid Leukemia
Children's Hospital of Soochow University
2024-01-01 PHASE2
NCT04501120 RECRUITING
Relapsed/Refractory Acute Myeloid Leukaemia; Myeloid Malignancy
Ascentage Pharma Group Inc.
2020-09-28 PHASE1; PHASE2
NCT07643636 NOT_YET_RECRUITING
AML (Acute Myeloid Leukemia)
Dongguan People's Hospital
2026-06-30 PHASE2
NCT07743671 NOT_YET_RECRUITING
Hereditary Hemorrhagic Telangiectasia
Vaderis Therapeutics AG
2026-09 PHASE3
NCT07131059 RECRUITING
AML, Adult
Institute of Hematology & Blood Diseases Hospital, China
2024-05-11
NCT07091006 NOT_YET_RECRUITING
Relapse Leukemia
Ruijin Hospital
2025-08-01 PHASE2
NCT07507825 NOT_YET_RECRUITING
Acute Myeloid Leukemia (AML)
First People's Hospital of Hangzhou
2026-03-31 PHASE2
NCT07469046 NOT_YET_RECRUITING
Acute Myeloid Leukemia (AML); Elderly Patients (60-75 Years)
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
2026-04-10 PHASE3
NCT07505160 NOT_YET_RECRUITING
Acute Myeloid Leukemia (AML)
Second Affiliated Hospital, Zhejiang University, School of Medicine
2026-03 PHASE2
NCT06265545 RECRUITING
AML; Refractory; Relapsed
Institute of Hematology & Blood Diseases Hospital, China
2024-02-22
NCT06659640 ACTIVE_NOT_RECRUITING
Hereditary Hemorrhagic Telangiectasia
Alnylam Pharmaceuticals
2024-11-07 PHASE1; PHASE2
NCT06328179 RECRUITING
Acute Myeloid Leukemia
Huai'an First People's Hospital
2022-05-24
NCT06744556 ACTIVE_NOT_RECRUITING
AML
Institute of Hematology & Blood Diseases Hospital, China
2025-01-21 PHASE2
NCT07516704 NOT_YET_RECRUITING
Prostate Cancer
Zhongda Hospital
2026-04-01 EARLY_PHASE1
NCT07601425 RECRUITING
Hereditary Hemorrhagic Telangiectasia (HHT); Osler Weber Rendu Disease
Atavistik Bio, Inc
2026-07-27 PHASE1; PHASE2
NCT07163910 RECRUITING
Prostate Cancer
baotai Liang
2025-04-20 PHASE2
NCT07159906 NOT_YET_RECRUITING
Diffuse Large B-Cell Lymphoma; Relapsed Non-Hodgkin Lymphoma; Refractory Non-Hodgkin Lymphoma
First Affiliated Hospital of Zhejiang University
2025-09-08 PHASE1; PHASE2
NCT05406362 ACTIVE_NOT_RECRUITING
Hereditary Hemorrhagic Telangiectasia (HHT)
Vaderis Therapeutics AG
2022-07-18 PHASE1; PHASE2
NCT07517510 ENROLLING_BY_INVITATION
Newly Diagnosed Mixed Phenotype Acute Leukemia (MPAL)
Guangdong Second Provincial General Hospital
2026-04-01 PHASE2
NCT04976036 RECRUITING
Telangiectasia, Hereditary Hemorrhagic
Dr. Romain Lazor
2022-05-05 PHASE2
NCT06834321 RECRUITING
Prostate CA
baotai Liang
2025-03-19 PHASE2
NCT06483906 RECRUITING
Acute Myeloid Leukemia
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
2024-07-02 PHASE2
NCT06742463 RECRUITING
Acute T Lymphpblastic Leukemia/Lymphoma
First Affiliated Hospital of Zhejiang University
2024-12 PHASE2
NCT06810791 RECRUITING
Newly Diagnosed Acute Myeloid Leukemia With High Risk
Nanfang Hospital, Southern Medical University
2025-01-01 PHASE3
NCT06361329 RECRUITING
ETP-ALL
First Affiliated Hospital of Zhejiang University
2024-04-03 PHASE3
NCT04404881 ACTIVE_NOT_RECRUITING
Hereditary Hemorrhagic Telangiectasia
Hanny Al-Samkari, MD
2020-11-23 PHASE2
NCT03850730 UNKNOWN
Hereditary Hemorrhagic Telangiectasia; Epistaxis
Cure HHT
2023-07-01 PHASE1; PHASE2
NCT05906914 COMPLETED
Acute Myeloid Leukemia, Adult
First Affiliated Hospital of Zhejiang University
2022-10-26 PHASE2
NCT05805098 RECRUITING
Acute Myeloid Leukemia
The First Affiliated Hospital of Soochow University
2023-03-01 PHASE2; PHASE3
NCT05941585 COMPLETED
Acute Myeloid Leukemia
Institute of Hematology & Blood Diseases Hospital, China
2023-08-08
NCT05726110 RECRUITING
Relapsed or Refractory Acute Myeloid Leukemia
Shanxi Bethune Hospital
2023-01-29 PHASE3
NCT05269849 COMPLETED
Hereditary Hemorrhagic Telangiectasia; Nosebleeds; Epistaxis
Unity Health Toronto
2022-03-16 PHASE2
NCT04126681 COMPLETED
Chronic Myeloid Leukemia, Chronic Phase
Ascentage Pharma Group Inc.
2019-10-21 PHASE2
NCT05805072 UNKNOWN
Relapsed/Refractory AML
The First Affiliated Hospital of Soochow University
2023-05-01
NCT04824924 UNKNOWN
Leukemia, Myeloid, Acute
Shanghai Jiao Tong University School of Medicine
2021-03 PHASE2
NCT04646356 COMPLETED
Hereditary Hemorrhagic Telangiectasia; Epistaxis Nosebleed
Unity Health Toronto
2020-10-20 PHASE2
NCT06259292 Recruiting
Hereditary Hemorrhagic Telangiectasia|Arteriovenous Malformations|Telangiectasia|Epistaxis|GastroIntestinal Bleeding|Cerebral Arteriovenous Malformations|Vascular Malformation
Cure HHT|Augusta University|The Cleveland Clinic|Mayo Clinic|Massachusetts General Hospital|Columbia University|Oregon Health and Science University|University of California Los Angeles|University of California San Francisco|University of Colorado Denver|University of North Carolina Chapel Hill|University of Pennsylvania|University of Texas|University of Utah|Washington University School of Medicine|Yale University|Health Resources and Services Administration (HRSA)|Children''s Hospital of Philadelphia
2023-11-13 --
NCT05513131 UNKNOWN
Secondary Acute Myeloid Leukemia
The First Affiliated Hospital with Nanjing Medical University
2021-09-30 PHASE2
NCT05954481 Recruiting
Hereditary Hemorrhagic Telangiectasia|Rendu Osler Disease
Hospices Civils de Lyon
2023-08-03 Not Applicable
NCT06266624 ACTIVE_NOT_RECRUITING
HHT
University Hospital, Essen
2020-09-25
NCT03850964 Recruiting
Hereditary Hemorrhagic Telangiectasia|Epistaxis|Anemia|Nosebleed|HHT
Cure HHT
2023-05-08 Phase 2|Phase 3
NCT03954782 COMPLETED
Telangiectasia, Hereditary Hemorrhagic; Rendu Osler Disease
Hospices Civils de Lyon
2020-06-22 PHASE2
NCT04505995 UNKNOWN
JMML
Air Force Military Medical University, China
2020-01-01
NCT05457361 UNKNOWN
Venetoclax Combined With Azacitidine Plus Homoharringtonine; Venetoclax Combined With Azacitidine
Nanfang Hospital, Southern Medical University
2022-03-01 PHASE3
NCT04248595 UNKNOWN
Acute Myeloid Leukemia
Ge Zheng
2019-12-01 PHASE2
NCT03978364 UNKNOWN
Myelodysplastic Syndromes,Acute Myeloid Leukemia
Shandong Provincial Hospital
2019-06-01 PHASE3
NCT04976036 Recruiting
Telangiectasia Hereditary Hemorrhagic
Dr. Romain Lazor|Boehringer Ingelheim|Centre Hospitalier Universitaire Vaudois
2022-05-05 Phase 2
NCT04083911 UNKNOWN
Acute Myeloid Leukemia; Induction Chemotherapy
The First Affiliated Hospital of Soochow University
2019-04-01 PHASE3
NCT04108052 COMPLETED
Telangiectasia, Hereditary Hemorrhagic; Rendu Osler Disease
Hospices Civils de Lyon
2019-11-28
NCT06266624 Active not recruiting
HHT
University Hospital Essen
2020-09-25 Not Applicable
NCT03227263 COMPLETED
Rendu Osler Disease; Telangiectasia, Hereditary Hemorrhagic
Hospices Civils de Lyon
2017-09-28 PHASE3
NCT02389959 COMPLETED
HHT; Hereditary Hemorrhagic Telangiectasia; Epistaxis; Nose Bleeds; Nasal Bleeding
Stanford University
2014-08-04 PHASE4
NCT03453255 UNKNOWN
Chemotherapy
Chinese PLA General Hospital
2018-01-01 PHASE1; PHASE2
NCT03910244 Completed
Telangiectasia Hereditary Hemorrhagic
The Cleveland Clinic|RTI International
2019-10-17 Phase 2
NCT02638012 COMPLETED
Hereditary Hemorrhagic Telangiectasia (HHT); Epistaxis
Unity Health Toronto
2015-12
NCT01752049 COMPLETED
Hereditary Hemorrhagic Telangiectasia
Unity Health Toronto
2013-05 PHASE1; PHASE2
NCT03152019 COMPLETED
Hemorrhagic Hereditary Telangiectasia (HHT)
Hospices Civils de Lyon
2017-05-22 PHASE2
NCT02484716 COMPLETED
Telangiectasia, Hereditary Hemorrhagic; Osler Rendu Disease
Hospices Civils de Lyon
2015-06 PHASE2
NCT02106520 TERMINATED
Hereditary Hemorrhagic Telangiectasia; Epistaxis
Hospices Civils de Lyon
2014-04 PHASE2; PHASE3
NCT02464644 COMPLETED
Hereditary Hemorrhagic Telangiectasia (HHT)
Imperial College London
2012-04
NCT01485224 COMPLETED
Hereditary Hemorrhagic Telangiectasia; Epistaxis
Fondazione IRCCS Policlinico San Matteo di Pavia
2011-11 PHASE2
NCT01408030 COMPLETED
Telangiectasia, Hereditary Hemorrhagic; Epistaxis
James Gossage
2011-08 PHASE2
NCT01397695 COMPLETED
Hereditary Hemorrhagic Telangiectasia (HHT)
University of California, San Diego
2009-06 PHASE2
NCT01402531 COMPLETED
Hereditary Hemorrhagic Telangiectasia (HHT)
University of California, San Diego
2010-07-22 PHASE2
NCT01406639 WITHDRAWN
Hereditary Hemorrhagic Telangiectasia (HHT); Nosebleeds
University of California, San Diego
2011-07 PHASE1
NCT00375219 COMPLETED
Chronic Myeloid Leukemia
Teva Branded Pharmaceutical Products R&D, Inc.
2006-09-20 PHASE2
NCT00462943 COMPLETED
Chronic Myeloid Leukemia
Teva Branded Pharmaceutical Products R&D, Inc.
2007-03-07 PHASE2
NCT00843440 COMPLETED
Hemorrhagic Hereditary Telangiectasia
Hospices Civils de Lyon
2009-03 PHASE2
NCT00675350 COMPLETED
Hematologic Tumors
ChemGenex Pharmaceuticals
2008-04 PHASE1
NCT00114959 TERMINATED
Myeloid Leukemia, Chronic; Myeloid Leukemia, Chronic, Accelerated-Phase; Blast Phase; Myeloid Leukemia, Chronic, Chronic-Phase
ChemGenex Pharmaceuticals
2005-10 PHASE2
NCT00006364 COMPLETED
Childhood Chronic Myelogenous Leukemia; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Chronic Phase Chronic Myelogenous Leukemia; Relapsing Chronic Myelogenous Leukemia
National Cancer Institute (NCI)
1999-11 PHASE2
NCT00003694 COMPLETED
Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Chronic Phase Chronic Myelogenous Leukemia
National Cancer Institute (NCI)
1999-03 PHASE2
NCT00003239 COMPLETED
Leukemia
M.D. Anderson Cancer Center
1998-03 PHASE2
NCT00002574 COMPLETED
Leukemia
National Cancer Institute (NCI)
1994-09 PHASE2
NCT00004933 TERMINATED
Leukemia
Alliance for Clinical Trials in Oncology
2000-01 PHASE3
NCT00030355 WITHDRAWN
Leukemia
National Cancer Institute (NCI)
PHASE1; PHASE2

(data from https://clinicaltrials.gov, updated on 2025-08-26)

Check the Homoharringtonine (HHT) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Homoharringtonine interacts directly with the ribosomal A-site to block the initial elongation step of protein translation while reversibly inhibiting IL-6-induced STAT3 phosphorylation at Tyrosine 705, thereby downregulating anti-apoptotic protein expression and triggering cell death. This robust blockade of protein synthesis and survival pathways suppresses cancer cell proliferation, demonstrating clear clinical relevance in the treatment of hematologic malignancies such as acute myeloid leukemia.

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>).

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