PB for Nephrogenic Diabetes Insipidus

(SerendipityPB1 Trial)

This trial tests a medication called PB to determine its effectiveness in managing frequent urination in individuals with certain kidney-related conditions. It aims to improve life for those with inherited or lithium-induced nephrogenic diabetes insipidus, which causes excessive urination, or those with autosomal dominant polycystic kidney disease treated with tolvaptan. Individuals with one of these conditions who experience frequent urination might be suitable for this trial. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important medical advancements.

The trial protocol does not specify if you need to stop taking your current medications. However, if you have high blood pressure, it must be controlled with medication for at least 30 days before starting the trial.

Research is investigating the safety and effectiveness of PB for treating conditions like nephrogenic diabetes insipidus and polyuria, which involves frequent urination due to other health issues. Although detailed information about PB remains limited, the study's early phase suggests it has shown promise for safety in initial tests. Early-phase studies typically assess whether a treatment is generally safe and free from major side effects.

Researchers are excited about PB for nephrogenic diabetes insipidus because it offers a new approach compared to standard treatments like thiazide diuretics and nonsteroidal anti-inflammatory drugs. PB is unique as it specifically targets the mechanisms causing excessive urination in different patient groups, including those with lithium-induced polyuria, hereditary nephrogenic diabetes insipidus, and autosomal dominant polycystic kidney disease. Unlike current treatments that generally aim to reduce urine output indirectly, PB may more precisely address the underlying causes, such as loss of function in specific receptors or channels like AVPR2 or AQP2. This targeted approach could lead to improved effectiveness and fewer side effects, making it a promising option for patients who have limited choices today.

Research has shown that PB may help treat nephrogenic diabetes insipidus (NDI), a condition causing frequent urination. In this trial, participants with hereditary NDI, NDI due to lithium treatment, or autosomal dominant polycystic kidney disease (ADPKD) treated with tolvaptan will receive PB. Studies have found PB effective in reducing frequent urination, particularly in inherited cases. Early findings suggest that PB could ease daily life by reducing bathroom visits, offering a promising option for those facing these challenging conditions.⁶⁷⁸⁹¹⁰