Clinical Trials

Numerous clinical trials spanning Early Phase 1 through Phase 4 evaluate 5-aminolevulinic acid HCl for diagnostic and therapeutic applications, primarily targeting neuro-oncological disorders such as glioblastoma, anaplastic astrocytoma, and recurrent brain tumors, as well as Crohn disease, type 2 diabetes, and mitochondrial oxygenation measurement. Sponsored by academic institutions and pharmaceutical entities including medac GmbH, Dartmouth-Hitchcock Medical Center, and Hiroshima University, these studies encompass varied operational statuses. While key evaluations of fluorescence-guided tumor resection have been completed, other trials currently remain active, recruiting, or terminated.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06876038 NOT_YET_RECRUITING
Oral Cancer; Oral Cavity Cancer; Oral Leukoplakia; Oral Lichen Planus; Oral Squamous Cell Carcinoma
University of Massachusetts, Boston
 2026-09 PHASE2
NCT05590689 RECRUITING
Glioblastoma
Universität Münster
 2022-11-09 PHASE1; PHASE2
NCT02191488 ACTIVE_NOT_RECRUITING
Brain Tumors
David W. Roberts
 2014-07 PHASE1
NCT06948552 RECRUITING
Upper Tract Urothelial Carcinoma
Henry Ford Health System
 2026-08 PHASE2
NCT07225621 RECRUITING
Glioblastoma; GBM
Alpheus Medical, Inc.
 2026-01-28 PHASE2
NCT07038278 NOT_YET_RECRUITING
Soft Tissue Sarcoma (STS)
University of Colorado, Denver
 2026-09-01 EARLY_PHASE1
NCT05475522 SUSPENDED
Gliomas, Malignant; Metastases to Brain
Sklifosovsky Institute of Emergency Care
 2022-09-01
NCT04381806 SUSPENDED
Solid Tumor
Fox Chase Cancer Center
 2020-07-30 PHASE1
NCT03897491 TERMINATED
Glioblastoma
photonamic GmbH & Co. KG
 2021-09-03 PHASE2
NCT06665724 COMPLETED
Anaplastic Astrocytoma (AA); Anaplastic Oligodendroglioma (AO); Glioblastoma (GBM)
Universität Münster
 2025-01-27 PHASE1
NCT06160492 RECRUITING
5-aminolevulinic Acid; Fluorescence-guided Resection
Lee's Pharmaceutical Limited
 2025-04-14 PHASE3
NCT06679712 NOT_YET_RECRUITING
Lung Cancer - Non Small Cell; Lung Metastases From Any Primary
Taoyuan General Hospital
 2024-12-01
NCT04738162 COMPLETED
Brain Tumor, Pediatric
Universität Münster
 2020-09-25 PHASE2
NCT06417281 COMPLETED
5-aminolevulinic Acid; Malignant Glioma of Brain
Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.
 2024-06-19 PHASE3
NCT04469699 TERMINATED
Glioblastoma Multiforme, Adult
University Hospital Muenster
 2021-04-12 PHASE2
NCT05362409 COMPLETED
High Grade Glioma
Alpheus Medical, Inc.
 2022-06-29 PHASE1
NCT04845919 COMPLETED
Glioblastoma Multiforme
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
 2023-02-22 PHASE2
NCT01898416 RECRUITING
The Aim of the Study is to Evaluate the Efficacy of Photodynamic Therapy as an; Adjuvant Therapy on the Clinical Outcome of Patients With Desmoid Tumors After; Surgical Resection
michal roll
 2013-06 PHASE2
NCT05228483 UNKNOWN
Vulva Lichen Sclerosus
Xijing Hospital
 2021-11-23
NCT04164849 COMPLETED
Crohn Disease
University Hospital, Akershus
 2019-11-22 PHASE1; PHASE2
NCT03066843 COMPLETED
Actinic Keratoses
Northwestern University
 2017-03-01
NCT07536737 COMPLETED
Oral Lichen Planus
Medical University of Bialystok
 2021-09-30
NCT03109353 COMPLETED
Cutaneous T-Cell Lymphoma, Unspecified; Chronic Graft Versus Host Disease in Skin
St. Olavs Hospital
 2017-09-20 PHASE1; PHASE2
NCT05101798 TERMINATED
Neoplasm; Skull Base Neoplasm
Icahn School of Medicine at Mount Sinai
 2021-09-14 PHASE2
NCT04167982 UNKNOWN
Acne
Shanghai Dermatology Hospital
 2020-03-28
NCT07027670 COMPLETED
Cardiac Surgical Procedures; Coronary Artery Bypass; Aortic Valve Disease; Mitral Valve Disease
University of Oxford
 2021-02-01 PHASE2
NCT04542850 COMPLETED
SARS-CoV 2; COVID-19
Royal College of Surgeons in Ireland - Medical University of Bahrain
 2020-11-15
NCT04636242 UNKNOWN
C. Acnes; Phototherapy; Postoperative Infection; Arthroscopic Rotator Cuff Repair; Shoulder Surgery
Rothman Institute Orthopaedics
 2019-08-22 PHASE3
NCT01128218 COMPLETED
Brain Neoplasms
Southern Illinois University
 2011-03 PHASE1; PHASE2
NCT04319159 COMPLETED
Actinic Keratoses
Biofrontera Bioscience GmbH
 2020-03-05 PHASE1
NCT04631250 COMPLETED
Acne Vulgaris
Tel-Aviv Sourasky Medical Center
 2018-11-01
NCT04626661 Completed
Mitochondrial Oxygenation Measurement|Measurement Error|Healthy
Sanquin-LUMC J.J van Rood Center for Clinical Transfusion Research|Leiden University Medical Center
 2020-06-15 --
NCT04020653 WITHDRAWN
Malaria
Neopharma Japan Co., Ltd.
 2019-09-06 PHASE2
NCT04221126 COMPLETED
Normal Skin
Merete Haedersdal
 2019-04-29 PHASE4
NCT00752323 COMPLETED
Brain and Central Nervous System Tumors
Andrew Sloan, MD
 2009-12-08 PHASE2
NCT02685592 COMPLETED
Lentigo Maligna
Joint Authority for Päijät-Häme Social and Health Care
 2016-02 PHASE4
NCT01502280 COMPLETED
Glioma
St. Joseph's Hospital and Medical Center, Phoenix
 2010-11 PHASE3
NCT02644187 COMPLETED
Therapy, Photodynamic; Keratosis, Actinic; Pain
Vastra Gotaland Region
 2015-12
NCT02451579 COMPLETED
Actinic Keratoses
Goldman, Butterwick, Fitzpatrick and Groff
 2015-02
NCT01445691 WITHDRAWN
Benign Neoplasms, Brain; Brain Cancer; Brain Neoplasms, Benign; Brain Neoplasms, Malignant; Brain Tumor, Primary; Brain Tumor, Recurrent; Brain Tumors; Intracranial Neoplasms; Neoplasms, Brain; Neoplasms, Intracranial; Primary Brain Neoplasms; Primary Malignant Brain Neoplasms; Primary Malignant Brain Tumors; Gliomas; Glioblastoma
Constantinos Hadjipanayis
 2015-11 PHASE2
NCT02050243 UNKNOWN
Central Nervous System Tumor, Pediatric
Tel-Aviv Sourasky Medical Center
 2014-02 PHASE1; PHASE2
NCT01682811 COMPLETED
Neurofibromatoses
Harry T Whelan, MD
 2012-03-12 PHASE1
NCT01502605 TERMINATED
Astrocytoma; Glioma
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
 2012-09 PHASE1
NCT01491711 UNKNOWN
Superficial Basal Cell Carcinoma
Maastricht University Medical Center
 2013-08 PHASE4
NCT01310868 COMPLETED
Glioblastoma
University College, London
 2011-05 PHASE2
NCT01575275 TERMINATED
Adult Giant Cell Glioblastoma; Adult Glioblastoma; Adult Gliosarcoma; Recurrent Adult Brain Tumor
Michael Vogelbaum, MD, PhD
 2012-05 PHASE2
NCT01610778 COMPLETED
Type 2 Diabetes on Medication
Hiroshima University
 2012-05
NCT01380548 COMPLETED
Iron Deficiency Anemia
Hiroshima University
 2011-06
NCT01508741 COMPLETED
Insomnia; Nocturnal Awakening; Irritability; Coping Behavior; Stress
University of Hawaii
 2012-01
NCT00870779 COMPLETED
Brain Tumors
David W. Roberts
 2007-05 PHASE1
NCT00571974 COMPLETED
Leukoplakia; Erythroplakia
University of Arkansas
 2007-01 PHASE1; PHASE2
NCT01459393 COMPLETED
Actinic Keratosis
Barretos Cancer Hospital
 2010-11 PHASE3
NCT00977795 WITHDRAWN
Brain Neoplasms
Endeavor Health
 2009-09 PHASE1; PHASE2
NCT00663910 TERMINATED
Non-melanomatous Skin Cancer
The Cleveland Clinic
 2008-03 EARLY_PHASE1
NCT02799030 COMPLETED
Actinic Keratosis
Biofrontera Bioscience GmbH
 2006-10 PHASE2
NCT00241670 COMPLETED
Brain Cancer; Brain Tumors; Cancer of Brain; Primary Brain Tumors; Brain Tumor, Primary
medac GmbH
 1999-10 PHASE3
NCT02755142 COMPLETED
Brain Neoplasm
medac GmbH
 2000-02 PHASE1; PHASE2
NCT00241670 Completed
Brain Cancer|Brain Tumors|Cancer of Brain|Primary Brain Tumors|Brain Tumor Primary
medac GmbH
 1999-10 Phase 3

(data from https://clinicaltrials.gov, updated on 2026-03-19)

Check the 5-Aminolevulinic acid HCl product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

As a key metabolic intermediate in the heme biosynthesis pathway, 5-aminolevulinic acid HCl is converted intracellularly into the photosensitizer protoporphyrin IX, which generates cytotoxic reactive oxygen species upon light activation to trigger selective cellular destruction. This tumor-preferential accumulation and photosensitization facilitate intraoperative visualization and targeted photodynamic toxicity, directly supporting its clinical utilization for the fluorescence-guided resection of malignant brain tumors such as glioblastoma.

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.