Treat Autosomal Dominant Polycystic Kidney Disease With Oral Ketone Ester?

Study Overview
=================
Brief Summary
-----------------
The investigators are running a study to see if a special drink, called a ketone ester, can help people with a type of kidney disease named Autosomal Dominant Polycystic Kidney Disease or ADPKD for short. The investigators want to find out: If it's easy for patients to take this drink every day for about 2 months. If it's safe and doesn't cause any problems. If it makes a difference in the size and function of the kidneys. Who can join? People between 16 to 70 years old who have ADPKD. Those with a certain amount of kidney size and function. People who haven't been on specific diets or lost a lot of weight recently. Women who are not breastfeeding and are using birth control. People with a body weight that is not too low or too high. Who cannot join? People who've been on a high-fat diet or skipped meals for a while recently. Those with other health conditions like diabetes or certain metabolic issues. Anyone who has a problem with getting an MRI scan. If participants are in another medical study right now. The study will happen in two Belgian hospitals and is supported by the UZ Brussel's nephrology department. The investigators hope to include 20 people and start in November 2023.

Official Title
-----------------
Treat Autosomal Dominant Polycystic Kidney Disease With Oral Ketone Ester?

Conditions
-----------------
ADPKD

Intervention / Treatment
-----------------
* Dietary Supplement: Ketone ester


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: - ADPKD patients (diagnosis based on genetics or imaging) ≥ 16 and ≤ 70 years. Total Kidney Volume ≥ 600 mL. eGFR (CKD-EPI) ≥ 30 ml/min/1.73m2. Normal blood glucose, HbA1C and ketones. Women of childbearing potential who are non-lactating and using an effective form of birth control. Stable disease-modifying treatment for the last 2 months. Well controlled hypertension (stable antihypertensive medications for at least 2 months). BMI > 18 < 30. Written informed consent. Exclusion Criteria: Exposure to a ketogenic diet or intermittent fasting for more than 2 weeks within 6 months before inclusion. Significant weight-loss (> 10%) within 6 months before inclusion. Diabetes mellitus. Conditions prohibiting the use of a ketogenic diet (liver damage, pancreatic failure, pyruvate-carboxylase deficiency, defects in fatty acid oxidation/gluconeogenesis/ketolysis/neogenesis, hyperinsulinism), based on patient history. Diagnosis with any disorder of fatty acid metabolism based on patient history. Eating disorder. Alcohol abuse. Contraindication for MRI. Participation in other interventional trial.

Ages Eligible for Study
-----------------
Minimum Age: 16 Years
Maximum Age: 70 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Repeated measures design to register the changes before and after treatment. Non-randomized, non-blinded, multi center prospective interventional cohort trial.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ketone Ester<br>Up to 100g ketone ester daily for 56 days | Dietary Supplement: Ketone ester<br>* Up to 100g ketone ester daily for 56 days<br>* Other names: (R)-3-hydroxybutyl (R)-3-hydroxybutyrate (Ketone Ester);|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Observance | Objective observance (ketone body levels ≥0.8 mmol/l in ≥75% of blood measurements and ≥60% of maximal theoretic dose determined by used packages and self-report) | 56 days | 
| Feasibility | Patient reported feasibility, defined as a cohen d effect size of < 0.5 in the ADPKD impact scale.Both targets (observance and self-reported feasibility) must be met to reach the endpoint. | 56 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Total Kidney Volume | Change in Total kidney volume (in mL; assessed with MRI) baseline vs day 56 | 56 days | 
| Kidney Function | Change in estimated glomerular filtration rate (eGFR) according to CKD-EPI; baseline vs day 56 | 56 days | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
ketone ester