Table of Contents

• Trials overview
• Who participated
• Conditions studied
• Trial phases and study goals
• Main endpoints measured
• Key trial details
• What these trials mean for patients

Trials overview

The studies of Aminolevulinic Acid Hydrochloride in the source data are interventional clinical trials, which means researchers gave the study treatment and then measured what happened.^{[1][2][3][4][5][6][7][8]}

These trials were mostly early studies, with phases ranging from Phase 1 to Phase 2, and one combined Phase 1/2 study.^{[1][2][3][4][5][6][7][8]}

Who participated

The trials included several different patient groups, such as children and adolescents with brain tumors, adults with glioblastoma, people with ovarian cancer, people with colorectal cancer, and people with malignant pleural mesothelioma.^{[1][2][3][4][5][6][7][8]}

One study also included healthy subjects, meaning people without colorectal cancer, to compare test results after a single oral dose.^[8]

Some studies had very specific age limits, such as patients 18 to 75 years old in the glioblastoma photodynamic therapy study.^[3]

Conditions studied

The main conditions studied were different types of brain tumors, recurrent glioblastoma, newly diagnosed glioblastoma, high-grade glioma, ovarian cancer, colorectal carcinoma, and malignant pleural mesothelioma.^{[1][2][3][4][5][6][7][8]}

Several trials focused on brain tumors, especially glioblastoma, which is a fast-growing brain cancer.^[2][3][4][5]

Trial phases and study goals

The Phase 2 studies mainly looked at safety, tolerability, and whether the treatment approach could be used in a practical way during surgery or imaging.^{[1][4][5][6][7][8]}

The Phase 1 study in recurrent glioblastoma focused on finding the maximum tolerated dose, which is the highest dose that can be given without too many serious side effects.^[2]

The Phase 1/2 study in glioblastoma looked at safety, feasibility, and the dose of light to use in the planned trial.^[3]

Main endpoints measured

Common endpoints were adverse events, which are health problems seen during the study, and serious side effects graded by standard cancer safety systems.^{[1][2][4][5][6][8]}

Some trials measured dose-limiting toxicity, meaning a side effect that is serious enough to stop dose increases or limit treatment.^[2][3]

Other studies measured whether the treatment could help find tumor tissue that was not seen with normal light or palpation, which means feeling the tissue by hand during surgery.^[7]

Key trial details

NCT04738162 studied children and adolescents with unifocal, contrast-enhancing intra-axial brain tumors and measured severe adverse events during and after fluorescence-guided resections.^[1]

2024-517651-12-00 studied repeat dosing with radiodynamic therapy in first recurrence of glioblastoma and measured the maximum tolerated dose, toxicological safety, neurological safety, dermatological safety, and new adverse events.^[2]

NCT05736406 studied intraoperative photodynamic therapy in adults 18 to 75 years old with grade IV glioblastoma and measured the maximum tolerated dose based on dose-limiting toxicity.^[3]

2023-509238-20-00 studied sonodynamic therapy in newly diagnosed high-grade glioma and measured adverse events, lab tests, and physical and neurological examinations over 28 days.^[4]

NCT03897491 studied stereotactic interstitial photodynamic therapy in adults with newly diagnosed supratentorial IDH wild-type glioblastoma and measured treatment-emergent adverse events within two weeks.^[5]

NCT04400539 studied intrapleural photodynamic therapy followed by nivolumab in malignant pleural mesothelioma and measured how many patients could complete the full treatment without unexpected grade 3 or worse toxicity.^[6]

2024-510614-33-00 studied intraoperative imaging in ovarian cancer and measured whether at least one 5-ALA positive tumor spot was also confirmed by tissue testing.^[7]

2023-508668-31-00 studied oral use in first-occurrence colorectal cancer and healthy subjects and measured adverse drug reactions and treatment stop rates, plus urine porphyrin levels for diagnostic value.^[8]

What these trials mean for patients

Overall, the trial data show that Aminolevulinic Acid Hydrochloride is being tested in several cancer settings, mostly to see whether it can be used safely and whether it helps during surgery or image-guided treatment.^{[1][2][3][4][5][6][7][8]}

The studies are still early in development, so the main goal is not to prove long-term benefit, but to learn how the treatment behaves in different patient groups and treatment settings.^{[1][2][3][4][5][6][7][8]}