Clinical Trials

Numerous clinical trials evaluate eflornithine across diverse medical indications, including solid tumors, glioblastoma, medulloblastoma, non-melanoma skin cancer, type 1 diabetes, hirsutism, and African trypanosomiasis. Spanning Phases 1 through 4, these studies encompass various recruitment statuses, including actively recruiting, active not recruiting, completed, and terminated protocols. Investigations are sponsored by a broad mix of academic medical centers, non-profit organizations such as the Juvenile Diabetes Research Foundation, and biopharmaceutical entities including Aminex Therapeutics, Orbus Therapeutics, and Allergan.

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
NCT02559778 RECRUITING
Neuroblastoma
Giselle Sholler
 2015-09 PHASE2
NCT06892678 RECRUITING
Osteosarcoma Recurrent; Ewing's Tumor Recurrent
Montefiore Medical Center
 2025-04-07 PHASE1; PHASE2
NCT04301843 ACTIVE_NOT_RECRUITING
Neuroblastoma
Giselle Sholler
 2020-09-25 PHASE2
NCT04696029 RECRUITING
Medulloblastoma
Giselle Sholler
 2021-03-29 PHASE2
NCT07287917 RECRUITING
Melanoma (Skin Cancer); HER2-low Hormone Receptor Positive Breast Cancer
Aminex Therapeutics, Inc.
 2026-01-26 PHASE1; 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
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
NCT04091022 COMPLETED
Non-melanoma Skin Cancer
University of Alabama at Birmingham
 2021-01-18 PHASE2
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
NCT06957834 COMPLETED
Melasma
National Skin Centre
 2023-06-06 PHASE2
NCT02139397 COMPLETED
Neuroblastoma Recurrent
Giselle Sholler
 2014-06-06 PHASE1; PHASE2
NCT02030964 COMPLETED
Neuroblastoma
New Approaches to Neuroblastoma Therapy Consortium
 2014-01-16 PHASE1
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
NCT05594563 Recruiting
Type 1 Diabetes
Emily K. Sims|Juvenile Diabetes Research Foundation|Cancer Prevention Pharmaceuticals Inc.|Indiana University
 2023-03-14 Phase 2
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
NCT05500508 Recruiting
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 Phase 1|Phase 2
NCT04696029 Recruiting
Medulloblastoma
Giselle Sholler|Milton S. Hershey Medical Center
 2021-03-29 Phase 2
NCT02384889 COMPLETED
Type 1 Diabetes
Indiana University
 2015-04 PHASE1
NCT01483144 COMPLETED
Familial Adenomatous Polyposis
Cancer Prevention Pharmaceuticals, Inc.
 2013-10 PHASE3
NCT03536728 Completed
Cancer|Solid Tumor|Solid Carcinoma|Advanced Cancer
Aminex Therapeutics Inc.
 2018-06-12 Phase 1
NCT02395666 COMPLETED
Neuroblastoma
Giselle Sholler
 2015-03-05 PHASE2
NCT02636569 Active not recruiting
Non-melanoma Skin Cancer
University of Alabama at Birmingham
 2017-10-25 Phase 2
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
NCT01636128 WITHDRAWN
Focus of Study: Drug Response Biomarkers, Chemoprevention, Neoplasms
Cancer Prevention Pharmaceuticals, Inc.
 2014-03 PHASE2
NCT01059071 COMPLETED
Neuroblastoma
Giselle Sholler
 2010-02 PHASE1
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
NCT00906880 COMPLETED
Human African Trypanosomiasis
Drugs for Neglected Diseases
 2009-04 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
NCT00330148 TERMINATED
Trypanosomiasis, African
Epicentre
 2001-03 PHASE3
NCT00176995 COMPLETED
Pseudofolliculitis Barbae
Hordinsky, Maria K., MD
 1999-03 PHASE2
NCT00489658 TERMINATED
Trypanosomiasis, African
Epicentre
 2002-10 PHASE2; PHASE3
NCT00003814 COMPLETED
Bladder Cancer
National Cancer Institute (NCI)
 1999-02 PHASE3

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

Check the Eflornithine (DFMO) Hydrochloride Hydrate product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Eflornithine hydrochloride hydrate selectively and irreversibly binds to and inhibits ornithine decarboxylase, thereby blocking rate-limiting polyamine biosynthesis, suppressing angiogenesis, and impeding cellular proliferation. This severe depletion of essential intracellular polyamines disrupts rapid cell division, providing the mechanistic rationale for its clinical evaluation in oncology, precancerous dermatological conditions, and autoimmune disorders such as type 1 diabetes.

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.