Ongoing Clinical Trials for Dent’s Disease
There is currently 1 ongoing clinical trial exploring new treatment options for Dent’s disease, a rare genetic kidney disorder. This trial is investigating the use of Alpelisib, a medication that may help improve kidney function in patients with Dent disease type 2. The study is being conducted in Italy and focuses specifically on male patients aged 18 and older with genetically confirmed Dent 2 disease.
Clinical trial locations
Study on the Effectiveness and Safety of Alpelisib for Patients with Dent 2 Disease
This clinical trial is examining whether Alpelisib, a medication also known by its code name BYL719, can help improve kidney function in patients with Dent disease type 2. The study specifically aims to test whether this treatment can enhance the kidney’s ability to absorb certain proteins and substances that are typically lost in the urine of patients with this condition.
Main inclusion criteria: To participate in this study, you must be at least 18 years old and have genetically confirmed Dent 2 disease. Your kidney function needs to be at a certain level, measured by an eGFR of at least 50 ml/min/1.73 m². The eGFR, or estimated Glomerular Filtration Rate, is a test that shows how well your kidneys are filtering waste from your blood. Only male patients can join this study because Dent 2 disease is linked to the X chromosome and primarily affects males. You must also be willing to follow the study requirements and sign an Informed Consent Form that explains the study details.
Main exclusion criteria: Female patients cannot participate in this trial. Additionally, individuals considered part of a vulnerable population—meaning people who might have difficulty giving informed consent or who are at higher risk of harm—are not eligible to join.
Focus and goal: The trial follows a specific treatment plan over four weeks. During the first week, participants take 50 mg of Alpelisib orally each day in tablet form. For the following three weeks, the dose is increased to 150 mg per day. Throughout the treatment period, researchers use a special imaging technique with a substance called 99mTc-labeled DMSA to measure how well the kidneys are functioning. The main goal is to see if Alpelisib improves the kidney’s ability to absorb proteins and other substances. Researchers will also monitor changes in urine composition and symptoms related to Fanconi syndrome, a condition that affects kidney function.
Investigational drug: Alpelisib is the medication being tested in this study. It works by inhibiting a specific enzyme involved in cell growth and metabolism. This Phase II trial is exploring whether Alpelisib can help manage the kidney problems associated with Dent 2 disease by improving the kidneys’ ability to reabsorb important proteins that would otherwise be lost in urine. The medication is taken orally as film-coated tablets.
Summary
Currently, there is one active clinical trial available for patients with Dent’s disease, specifically focusing on Dent disease type 2. This trial is taking place in Italy and represents an important step in researching potential treatments for this rare genetic kidney disorder. The study is limited to male patients due to the X-linked genetic nature of the condition. The trial is investigating Alpelisib, a PI3K inhibitor, which represents a novel approach to treating the kidney dysfunction associated with this disease. While only one trial is currently available, it offers hope for patients seeking new treatment options that may help preserve kidney function and reduce protein loss in urine.
