Table of Contents
- Clinical trials overview
- Heart failure studies
- Lung cancer study
- Eye disease study
- Advanced solid tumor study
- Main outcomes measured
- Who may take part
Clinical trials overview
The trial data shows that Acetazolamide is being studied in several different research settings, not just one disease area.[1][2][3][4][5][6]
These studies include interventional trials, which means the researchers give a treatment and then measure what happens.[1][2][3][4][5][6]
The main trial phases in the data are Phase 1, Phase 1/2, and Phase 3.[1][2][3][4][5][6]
Heart failure studies
Several trials are studying Acetazolamide in people with acute heart failure, especially those with volume overload, which means the body has too much fluid.[1][4]
In NCT06166654, the goal is to find the most effective diuretic treatment strategy for patients with acute decompensated heart failure who have volume overload and may have diuretic resistance.[1]
This is a Phase 3 study with 939 planned participants, and the main outcome is days alive and outside hospital until day 30, which measures how much time patients spend alive and not admitted to the hospital during the first month.[1]
Another Phase 3 study, 2024-513799-16-00, is also in acute heart failure and looks at Acetazolamide together with SGLT2i treatment, with 24-hour sodium excretion as the main outcome.[4]
That outcome means the study checks how much sodium leaves the body in urine during one day, which helps show whether fluid removal is improving.[4]
A third Phase 3 study, 2024-519565-21-01, is in chronic congestive heart failure and compares standard diuretic therapy with combination therapy that includes Acetazolamide and free-dose metolazone.[5]
This study is looking at differences in short- to medium-term rehospitalizations, meaning how often patients need to return to the hospital after treatment.[5]
Lung cancer study
NCT03467360 is a Phase 1 study in small cell lung carcinoma, which is a fast-growing type of lung cancer.[2]
The trial is suspended and includes 27 participants.[2]
Its main purpose is to find the Maximum Tolerated Dose and Recommended Dose of acetazolamide when used with radiotherapy and other cancer treatments.[2]
The primary endpoint is based on dose-limiting toxicities, which are side effects that may stop a dose from being increased, and these are measured during treatment and for up to 6 months afterward.[2]
The study includes two groups: one with localised disease treated with radiochemotherapy and one with extensive disease treated with radio-immunotherapy.[2]
Eye disease study
NCT04850118 is a Phase 3 study in people with X-linked retinitis pigmentosa caused by RPGR mutations.[3]
This trial was withdrawn and planned to enroll 65 participants.[3]
In this study, Acetazolamide was one of several medicines used around a single subretinal injection of AGTC-501, which is a treatment given under the retina.[3]
The main efficacy endpoint in Europe is the change in mean sensitivity across the whole grid on MAIA microperimetry at Month 12, which is an eye test that measures how well the retina responds to light in different areas.[3]
Advanced solid tumor study
NCT05706129 is a Phase 1/2 study in people with unresectable locally advanced or metastatic solid tumors, meaning tumors that cannot be removed by surgery or have spread to other parts of the body.[6]
The study is authorised and has a planned enrollment of 300 participants across several tumor types, including clear cell renal cell carcinoma, pancreatic ductal adenocarcinoma, colorectal cancer, urothelial carcinoma, indeterminate renal mass, head and neck cancer, triple negative breast cancer, and squamous non-small cell lung cancer.[6]
Acetazolamide appears in this study as part of the imaging-related procedure alongside [68Ga]Ga-DPI-4452 and [177Lu]Lu-DPI-4452.[6]
The trial measures safety, dose-limiting toxicities, imaging results, objective response rate, and PET/CT findings such as radiotracer uptake and tumor-to-background ratios.[6]
Main outcomes measured
The trial data shows that Acetazolamide studies are not all using the same endpoint, because each trial is designed for a different medical question.[1][2][3][4][5][6]
Hospital-free days are used in acute heart failure to see how long patients stay alive and out of the hospital after treatment.[1]
Sodium excretion is used to measure how much sodium is removed in urine in one day, which helps show fluid-loss effect.[4]
Rehospitalization is used in chronic heart failure to see whether combination treatment lowers return visits to the hospital.[5]
Safety and tolerability are central in the early cancer and imaging studies, where researchers watch for side effects and how well the treatment is accepted.[2][6]
Visual function is measured in the eye study with MAIA microperimetry to see whether the retina’s light response changes over time.[3]
Tumor response and PET imaging findings are used in the advanced solid tumor study to see whether the tracer reaches the tumors and whether the tumors respond.[6]
Who may take part
Each study has its own entry rules, so participation depends on the disease being studied and the trial design.[1][2][3][4][5][6]
In the heart failure studies, participants are patients with acute or chronic heart failure and fluid overload, while the lung cancer study includes people with small cell lung carcinoma.[1][2][4][5]
The eye study is for participants with X-linked retinitis pigmentosa caused by RPGR mutations, and the tumor imaging study is for people with unresectable locally advanced or metastatic solid tumors.[3][6]
Across the trial data, Acetazolamide is being studied both alone and as part of combination treatment plans, depending on the condition and the trial goal.[1][2][4][5][6]
