What side effects are associated with this type of intervention?

The most common treatment for Polycystic Kidney Disease (PKD) that regulates water reabsorption is tolvaptan, a vasopressin V2-receptor antagonist. Known side effects of tolvaptan include liver toxicity, polyuria (frequent urination), polydipsia (excessive thirst), and potential electrolyte imbalances such as hypernatremia. Other treatments, like ACE inhibitors or ARBs, used to manage hypertension in PKD, can cause side effects such as hypotension, hyperkalemia, and renal function deterioration. It is crucial to monitor patients closely for these adverse effects to manage them effectively.

What prior FDA approvals exist?

Tolvaptan is an FDA-approved treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD). It is a vasopressin V2-receptor antagonist that helps regulate water reabsorption in the kidneys, thereby slowing the progression of kidney cyst growth and preserving kidney function. This mechanism is similar to the focus of the PB trial, which aims to manage water reabsorption in the kidneys.

What other types of research exist?

Promising novel alternatives to PB for Polycystic Kidney Disease (PKD) patients include: 1) Tolvaptan, a vasopressin V2 receptor antagonist, which has been shown to slow kidney growth and function decline in ADPKD patients. 2) Lixivaptan, another vasopressin V2 receptor antagonist, which has demonstrated efficacy in animal models of PKD. 3) Dapagliflozin, a sodium-glucose cotransporter 2 (SGLT2) inhibitor, which has shown potential benefits in animal models of PKD. These treatments offer different mechanisms of action and may provide additional options for managing PKD.

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Vasopressin Analog

PB for Nephrogenic Diabetes Insipidus (SerendipityPB1 Trial)

Phase 2

Recruiting

Led By Fouad Chebib, MD

Research Sponsored by Mayo Clinic

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, day 15, day 45, day 75

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a drug called PB to see if it can help people who urinate frequently due to certain kidney conditions or treatments. It aims to help the kidneys manage water better, so patients don't have to go to the bathroom as often.

Who is the study for?

This trial is for adults who can consent and have been diagnosed with nephrogenic diabetes insipidus (inherited or from lithium/tolvaptan use), ADPKD, or severe polyuria. They must have a GFR ≥ 30 ml/min, low morning urine concentration, and controlled blood pressure if hypertensive. Excluded are those with chronic diseases like heart failure or liver disease, urinary issues, recent gout attacks, uncontrolled gout/hyperuricemia, drug allergies to PB, or past hepatotoxicity from tolvaptan.

What is being tested?

The study tests the medication PB's effectiveness in reducing excessive urination in patients with inherited nephrogenic diabetes insipidus (NDI), NDI due to long-term treatment of ADPKD with tolvaptan, or NDI caused by previous lithium therapy.

What are the potential side effects?

Potential side effects of PB may include allergic reactions for those sensitive to it and possibly liver-related issues as seen in past cases with similar treatments. The exact side effects will be monitored throughout the trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, day 15, day 45, day 75

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, day 15, day 45, day 75

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, day 15, day 45, day 75 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in urine osmolality

Secondary study objectives

Change in urine output

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Polyuric subjects with Hereditary Nephrogenic Diabetes InsipidusExperimental Treatment1 Intervention

Polyuric subjects with hereditary nephrogenic diabetes insipidus with loss of function of arginine vasopressin receptor 2 (AVPR2) or aquaporin 2 (AQP2) will be treated with PB

Group II: Polyuric subjects with Autosomal Dominant Polycystic Kidney Disease treated with TolvaptanExperimental Treatment1 Intervention

Polyuric subjects with autosomal dominant polycystic kidney disease on chronic tolvaptan treatment will be treated with PB

Group III: Polyuric subject secondary to lithium administrationExperimental Treatment1 Intervention

Polyuric subject post lithium administration will receive PB

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Polycystic Kidney Disease (PKD) include ACE inhibitors and ARBs, which help manage hypertension and reduce proteinuria, thus slowing kidney damage. Tolvaptan, a vasopressin-2 receptor antagonist, directly targets cyst growth by reducing cyclic AMP levels, which are implicated in cyst formation and fluid secretion. Metformin, an AMPK activator, inhibits pathways that drive cyst expansion. These treatments are crucial for PKD patients as they address the underlying mechanisms of cyst growth and kidney function decline. The regulation of water reabsorption, as studied in the trial PB, is also significant because it can mitigate the polyuria associated with tolvaptan treatment, improving patient quality of life.

Autosomal dominant polycystic kidney disease and pioglitazone for its therapy: a comprehensive review with an emphasis on the molecular pathogenesis and pharmacological aspects.Pro: Tolvaptan delays the progression of autosomal dominant polycystic kidney disease.Can progression of autosomal dominant or autosomal recessive polycystic kidney disease be prevented?