Clinical Trials

Numerous clinical trials evaluate L-ornithine and its derivatives across Early Phase 1 to Phase 4 protocols for conditions including acute liver failure, postmenopausal hypertension, hirsutism, African trypanosomiasis, and advanced malignancies such as non-small cell lung cancer and glioblastoma. Supported by academic institutions, medical centers, and pharmaceutical sponsors, these registered studies demonstrate a wide operational spectrum ranging from actively recruiting and completed to suspended, terminated, or withdrawn.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06465199 RECRUITING
Atypical Teratoid/Rhabdoid Tumor; Embryonal Tumor With Multilayered Rosettes; Ewing Sarcoma; Diffuse Intrinsic Pontine Glioma; Osteosarcoma; Neuroblastoma; DIPG Brain Tumor
Milton S. Hershey Medical Center
 2026-05-13 PHASE1; PHASE2
NCT07321912 RECRUITING
Osteosarcoma; Ewing Sarcoma
Milton S. Hershey Medical Center
 2026-06-05 PHASE2
NCT07468136 RECRUITING
Anaplastic Oligodendroglioma; Astrocytoma, IDH-Mutant, Grade 3; Astrocytoma, IDH-Mutant, Grade 4; Diffuse Astrocytoma; Glioblastoma, IDH-Wildtype; Malignant Glioma
Mayo Clinic
 2026-06-23 PHASE1; PHASE2
NCT06219174 SUSPENDED
Non Small Cell Lung Cancer; Lung Cancer
H. Lee Moffitt Cancer Center and Research Institute
 2024-08-07 PHASE1; PHASE2
NCT04301843 ACTIVE_NOT_RECRUITING
Neuroblastoma
Giselle Sholler
 2020-09-25 PHASE2
NCT04696029 RECRUITING
Medulloblastoma
Giselle Sholler
 2021-03-29 PHASE2
NCT02679144 ACTIVE_NOT_RECRUITING
Neuroblastoma
Giselle Sholler
 2016-02 PHASE2
NCT01349881 ACTIVE_NOT_RECRUITING
Colorectal Neoplasms
SWOG Cancer Research Network
 2013-08-23 PHASE3
NCT07117630 RECRUITING
Breast Cancer
Fudan University
 2025-09-25 PHASE2
NCT05594563 ACTIVE_NOT_RECRUITING
Type 1 Diabetes
Emily K. Sims
 2023-03-14 PHASE2
NCT05717153 RECRUITING
Diffuse Glioma; Malignant Glioma
Mayo Clinic
 2023-10-01 EARLY_PHASE1
NCT06059118 RECRUITING
Prostate Cancer
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
 2023-10-04 PHASE2
NCT05879367 RECRUITING
Glioblastoma, IDH-wildtype; Glioblastoma; Glioblastoma Multiforme; Glioblastoma IDH (Isocitrate Dehydrogenase) Wildtype; GBM; Astrocytoma; Astrocytoma, IDH-Mutant
Orbus Therapeutics, Inc.
 2023-07-24 PHASE1
NCT06976424 RECRUITING
Hirsuitism
Khyber Teaching Hospital
 2025-07-15 PHASE4
NCT06455267 UNKNOWN
Mild to Severe Crohns Disease
The Third Xiangya Hospital of Central South University
 2024-04-01
NCT07037394 COMPLETED
Overt Hepatic Encephalopathy
Qurratul Ain Jamil
 2024-12-02 PHASE4
NCT04128462 WITHDRAWN
Hepatic Encephalopathy
Mallinckrodt
 2021-11 PHASE3
NCT03794349 ACTIVE_NOT_RECRUITING
High Risk Neuroblastoma; Recurrent Neuroblastoma; Refractory Neuroblastoma
Children's Oncology Group
 2019-07-08 PHASE2
NCT05500508 TERMINATED
Cancer; Solid Tumor; Solid Carcinoma; Advanced Cancer; DIPG Brain Tumor; Ovary Cancer; Breast Cancer; Papillary Thyroid Cancer; Head and Neck Cancer; Gastric Cancer; Nsclc; Mesotheliomas Pleural; Mesothelioma Peritoneum; Esophageal Cancer; Diffuse Midline Glioma, H3 K27M-Mutant; Endometrial Cancer; Cervical Cancer; Melanoma; Colorectal Cancer; Glioma, Malignant
Aminex Therapeutics, Inc.
 2022-11-29 PHASE1; PHASE2
NCT05539027 COMPLETED
Hepatic Encephalopathy
Ain Shams University
 2022-09-20 PHASE4
NCT05778461 UNKNOWN
Liver Failure, Acute
Institute of Liver and Biliary Sciences, India
 2023-03-15
NCT05737030 ACTIVE_NOT_RECRUITING
Cirrhosis
Medical University of Graz
 2023-02-06 PHASE4
NCT05920213 UNKNOWN
Hepatic Encephalopathy Stage 2; Hepatic Encephalopathy Stage 3; Hepatic Encephalopathy Stage 4
Jinnah Postgraduate Medical Centre
 2023-10 PHASE4
NCT02030964 COMPLETED
Neuroblastoma
New Approaches to Neuroblastoma Therapy Consortium
 2014-01-16 PHASE1
NCT06025383 Recruiting
Menopause|Hypertension
Texas Tech University
 2023-07-20 Not Applicable
NCT04403282 TERMINATED
Pseudofolliculitis Barbae
Walter Reed National Military Medical Center
 2020-09-27 PHASE4
NCT02796261 UNKNOWN
Anaplastic Astrocytoma; Recurrent Anaplastic Astrocytoma
Orbus Therapeutics, Inc.
 2016-07 PHASE3
NCT03536728 COMPLETED
Cancer; Solid Tumor; Solid Carcinoma; Advanced Cancer
Aminex Therapeutics, Inc.
 2018-06-12 PHASE1
NCT02794428 COMPLETED
Gastric Cancer; Gastric Intestinal Metaplasia
Vanderbilt-Ingram Cancer Center
 2016-09-19 PHASE2
NCT03712280 COMPLETED
Hepatic Cirrhosis; Hepatic Encephalopathy (HE)
Mallinckrodt
 2018-12-01 PHASE2
NCT02384889 COMPLETED
Type 1 Diabetes
Indiana University
 2015-04 PHASE1
NCT01483144 COMPLETED
Familial Adenomatous Polyposis
Cancer Prevention Pharmaceuticals, Inc.
 2013-10 PHASE3
NCT05042024 COMPLETED
Periodontitis
Ukrainian Medical Stomatological Academy
 2017-10-20 PHASE3
NCT05227781 Completed
Hypertension|Menopause
Texas Tech University
 2018-05-24 Not Applicable
NCT02395666 COMPLETED
Neuroblastoma
Giselle Sholler
 2015-03-05 PHASE2
NCT01722578 COMPLETED
Cirrhosis of Liver; Hepatic Encephalopathy
Dayanand Medical College and Hospital
 2013-12 PHASE4
NCT01685827 COMPLETED
Human African Trypanosomiasis (HAT); Sleeping Sickness
Drugs for Neglected Diseases
 2012-10 PHASE2; PHASE3
NCT00983580 COMPLETED
Adenomatous Polyp
National Cancer Institute (NCI)
 2009-08-20 PHASE2
NCT02158182 COMPLETED
Hepatic Encephalopathy
Hospital General de Mexico
 2014-07 PHASE4
NCT01636128 WITHDRAWN
Focus of Study: Drug Response Biomarkers, Chemoprevention, Neoplasms
Cancer Prevention Pharmaceuticals, Inc.
 2014-03 PHASE2
NCT01847651 COMPLETED
Hepatic Encephalopathy; Minimal Hepatic Encephalopathy; Cirrhosis
Imperial College London
 2013-08 PHASE4
NCT01817894 COMPLETED
Hirsutism
Bispebjerg Hospital
 2012-01 PHASE4
NCT01245816 WITHDRAWN
Familial Adenomatous Polyposis
Cancer Prevention Pharmaceuticals, Inc.
 2011-03 PHASE3
NCT00006101 COMPLETED
Prostate Cancer
Thomas E. Ahlering
 1998-10 PHASE2
NCT01440829 COMPLETED
Decompensated Cirrhosis; Portal Hypertension; Bleeding Varices; Refractory Ascites
Air Force Military Medical University, China
 2011-12 PHASE2; PHASE3
NCT01041755 COMPLETED
Hepatic Encephalopathy
Centro Regional para el Estudio de las Enfermedades Digestivas
 2009-11 PHASE4
NCT00906880 COMPLETED
Human African Trypanosomiasis
Drugs for Neglected Diseases
 2009-04 PHASE4
NCT00740142 COMPLETED
Hepatic Encephalopathy
Mahidol University
 2008-09 PHASE4
NCT00896831 UNKNOWN
Hepatic Encephalopathy
Hospital de Clinicas de Porto Alegre
 2008-11 PHASE4
NCT00601640 COMPLETED
Other Benign Neoplasm of Skin, Unspecified
University of Arizona
 2007-01 PHASE2
NCT00033371 COMPLETED
Colorectal Cancer; Familial Adenomatous Polyposis
National Cancer Institute (NCI)
 2001-12-13 PHASE2
NCT00118365 COMPLETED
Precancerous Condition
National Cancer Institute (NCI)
 1998-07 PHASE3
NCT00146627 COMPLETED
Trypanosomiasis, African
Drugs for Neglected Diseases
 PHASE3
NCT00204789 COMPLETED
Post-solid Organ Transplant; Skin Neoplasms
University of Wisconsin, Madison
 2003-07 PHASE2
NCT00006079 COMPLETED
Cervical Cancer; Precancerous Condition
M.D. Anderson Cancer Center
 1998-06-19 PHASE2
NCT00086736 COMPLETED
Prostate Cancer
University of Alabama at Birmingham
 2001-11 PHASE2
NCT00021294 COMPLETED
Non-melanomatous Skin Cancer; Precancerous/Nonmalignant Condition
University of Arizona
 2001-05 PHASE2
NCT00003076 COMPLETED
Esophageal Cancer
University of Michigan Rogel Cancer Center
 1995-10 PHASE2
NCT00005884 COMPLETED
Non-melanomatous Skin Cancer
University of Wisconsin, Madison
 PHASE3
NCT00152048 COMPLETED
Hirsutism
Allergan
 2004-11-30 PHASE4
NCT00470314 UNKNOWN
Acute Liver Failure
All India Institute of Medical Sciences
 2005-01 PHASE2
NCT00330148 TERMINATED
Trypanosomiasis, African
Epicentre
 2001-03 PHASE3
NCT00004386 TERMINATED
Ornithine Transcarbamylase Deficiency Disease
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
 1995-10 PHASE1
NCT00305591 COMPLETED
Cirrhosis; Hepatic Encephalopathy
Imperial College London
 2006-03
NCT00004498 TERMINATED
Ornithine Transcarbamylase Deficiency Disease
University of Pennsylvania
 1998-07 PHASE1
NCT00489658 TERMINATED
Trypanosomiasis, African
Epicentre
 2002-10 PHASE2; PHASE3
NCT00003814 COMPLETED
Bladder Cancer
National Cancer Institute (NCI)
 1999-02 PHASE3
NCT00433368 COMPLETED
Hepatic Encephalopathy
Aga Khan University
 2003-10 PHASE3

(data from https://clinicaltrials.gov, updated on 2026-09-03)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

L-Ornithine functions as an essential substrate within the hepatic urea cycle, combining with carbamoyl phosphate via ornithine transcarbamylase to drive the conversion of neurotoxic ammonia into excretable urea. This enhancement of nitrogen clearance and cellular energy efficiency mitigates hyperammonemic metabolic stress, demonstrating direct therapeutic relevance in managing hepatic dysfunctions such as acute liver failure investigated in clinical trials.

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.