MedPath

Short Term Induction of Ketosis in PKD

Not Applicable
Completed
Conditions
ADPKD
Interventions
Other: Ketogenic diet
Other: Fasting
Registration Number
NCT04472624
Lead Sponsor
University of Cologne
Brief Summary

Recently, it has been shown that ketose-inducing dietary interventions slow disease progression in animal models of polycystic kidney disease (PKD), even when the state of ketosis is only induced for a short period of time. The present study aims to investigate the effects of short term ketosis on total kidney volume (TKV) (and other parameters) in 10 ADPKD-patients with rapidly progressive disease.

Detailed Description

Recently, dietary models inducing ketosis have been shown to inhibit disease progression in animal models of PKD. Those beneficial dietary models included time-restricted feeding (TRF) without caloric reduction, ad libitum administered ketogenic diet (KD) and acute short-term fasting in mouse, rat and feline models of PKD.

In a PKD rat model, TRF without caloric reduction resulted in a strong inhibition of mTOR signaling, proliferation and fibrosis in the affected kidneys. The adminstration of an ad libitum fed KD led to similar results. In rat, mouse and feline models of PKD, acute fasting led to a significant reduction of cyst volume. Therefore, cystic cells seem to be metabolically inflexible and exhibit an altered metabolism characterized by increased glycolysis and, amongst others, defective fatty acid oxidation, similar to the Warburg effect in cancer. (Torres, Kruger et al. 2019)

While those beneficial observations were made in mouse, rat and feline models of PKD, the effects of a ketogenic diet in human ADPKD patients have not been investigated yet, even though the adminstration of ketogenic diets is used as a treatment for epilepsy in children since the 1920s and fasting is one of the oldest medical procedures.

Therefore, the aim of the present study is to investigate the effects of a short-period ketonic state in 10 ADPKD patients with fast progressive disease.

10 ADPKD-patients (aged 18-60 years, CKD G 1-3a) will be enrolled after giving informed consent. These 10 subjects will go through four trial-related visits. During these visits, physical examinations will be performed, blood will be drawn, urine will be collected and ketone body measurements in breath, blood and urine will be carried out. Each study visit includes an abdominal MRI-scan. Between visit 1 and visit 2, patients will eat their regular carbohydrate-rich diet. After visit 2, a ketonic state will be induced in those patients. Patients can choose whether the ketonic state will be induced by acute fasting for 72 hours (under sufficient water consumption and salt substitution) or by eating a KD for 14 days. Study visit 3 will take place within 72 hours after finishing the dietary intervention. After study visit 3, patients will restart eating their regular diet. Study visit 4 will provide follow-up data 3-6 weeks after the dietary intervention.

After study completion, the relative difference of TKV measured by MRI-based volumetry of the kidneys immediately before and immediately after the ketonic state, will be compared to TKV growth/decline while eating a regular carbohydrate-rich diet.

In general, patients are counselled in our outpatient department as part of "The German AD(H)PKD Registry" (more than 700 patients enrolled). As from those patients, clinical data, laboratory data and imaging studies are present, identification and recruitment of patients with fast rapidly progressing disease will be facilitated. Also, the investigators are closely liaised with the German self-help group PKDCure (PKD Familiaere Zystennieren e.V.), which is dedicated to ADPKD-linked research. This will facilitate recruitment of patients even more.

Data obtained from the study visits will be collected. The parameters listed below constitute the core data set, additional parameters can be included if considered essential.

Registered patients will be provided with diaries for the documentation of feeling of hunger, problems and discomforts as well as acetoacetate concentrations in breath. Patients are also provided with a portable breath-analyzer to measure acetoacetate concentrations in breath in between study visits. Study diaries are collected on Visit 4.

Data capture will be performed at each study visit after enrollment.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
10
Inclusion Criteria
  • Male and female ADPKD-patients (diagnosed by genetics / typical MRI / ultrasound)

  • With evidence of fast progression, at least one of the following criteria

    1. Mayo Class 1C-1E
    2. Truncating PKD1-mutation
    3. Hypertension < 35 years
    4. Urological complications < 35 years
    5. ≥ 1 first class or second class family member with need of renal replacement therapy < 60 years
    6. eGFR loss > 2,5 ml/min/1,73m2 per year
    7. PROPKD-Score > 6
  • Age ≥ 18 and ≤ 60 years

  • CKD stage 1-3a according to eGFR

  • Signed written informed consent

Exclusion Criteria
  • Currently under tolvaptan
  • BMI < 18 or > 35
  • Diabetes mellitus (Type I, Type II, MODY, LADA)
  • Active alcoholism
  • Vegan or vegetarian lifestyle
  • Inability to sign or understand written informed consent
  • Anamnestically known circumstances which forbid the induction of a ketonic state by ketogenic diet or acute fasting (Liver damage (AST/ALT > 3x upper limit of normal, alkaline phosphatase > 6x upper limit of normal , Bilirubin ≥ 3 mg/dl), Diabetes mellitus, Pyruvate carboxylase deficiency, defects of gluconeogenesis, defects of ketolysis / ketogenesis, hyperinsulinism, defects of fatty acid oxidation )
  • Allergies or food intolerance against components of a ketogenic diet
  • Eating disorders (Anorexia nervosa / Bulimia)
  • Participation in a weight loss program (e.g. Optifast) or intake of medication to promote weight loss within the last six months
  • Ketogenic Diet > 1 month within the last 12 months
  • Chronic renal replacement therapy
  • Previous history of kidney transplantation
  • Uncontrolled local or systemic infection (according to clinical assessment)
  • Simultaneous participation in other interventional studies
  • Pregnant or breastfeeding women
  • Persons who are dependent of or employed by the study investigators
  • Contraindications against MRI examinations (stents, pacemakers or other metal inserts, claustrophobia)
  • Persons living in an establishment by court order or official instructions

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Diet armKetogenic dietParticipants will be using ketogenic diet for 14 days
Fasting armFastingParticipants will be fasting during 72 hours
Primary Outcome Measures
NameTimeMethod
Relative difference of TKV immediately before and after the ketonic stateVisit 2: 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2

MRI-based kidney volumetry at study visit 2 and study visit 3

Secondary Outcome Measures
NameTimeMethod
Absolute and relative difference of TKVBaseline visit (V1) and final study visit (V4) after at maximum 95 days

assessed by MRI-based volumetry at all study visits

Absolute and relative difference of height-adjusted total kidney volume (htTKV)Baseline visit (V1) and final study visit (V4) after at maximum 95 days

assessed by MRI-based volumetry at all study visits

Absolute and relative difference of total liver volume (TLV)Baseline visit (V1) and final study visit (V4) after at maximum 95 days

assessed by MRI-based volumetry at all study visits

Absolute and relative difference of cystic burden of kidneys and liverBaseline visit (V1) and final study visit (V4) after at maximum 95 days

assessed by MRI at all study visits

Absolute and relative change of Renal Function PanelBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement of creatinine, urea and uric acid in blood and urine samples (measurement unit mg/dl)

Absolute and relative change of electrolytes and mineralsBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement of electrolyte and minerals (sodium, natrium, potassium, magnesium, chloride, phosphate) in blood (measurement unit mmol/L) and urine samples (measurement unit mmol/24h)

Absolute and relative change of albuminBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement in blood and urine samples

Absolute and relative change of glucoseBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement in blood and urine samples

Absolute and relative change of Hepatic Function PanelBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement of Alanine aminotransferase(ALT), Aspartate Aminotransferase (ASP), Alkaline Phosphatase (ALP), gamma-glutamyl transferase (GGT) and Lactate Dehydrogenase (LD) in blood samples (measurement unit U/L)

Absolute and relative change of bilirubinBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement in blood samples

Absolute and relative change of lipid panelBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement of high density lipoprotein (HDL), low density lipoprotein (LDL), total cholesterol, triglycerides and lipoprotein(a) in blood samples

Absolute and relative change of inflammatory parametersBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement of c-reactive protein (CRP) and high sensitivity c-reactive protein (hsCRP) in blood samples ( measurement unit mg/L)

Absolute and relative change in blood countBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement of white blood cells, red blood cells, platelets in blood samples (measurement unit 10\^9/L )

Absolute and relative change of hemoglobinBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement in blood samples

Absolute and relative change of hematokritBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement in blood samples

Absolute and relative change of betahydroxybutyrate levelBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement of betahydroxybutyrate level in blood samples for determination of ketosis

Absolute and relative change in blood gas analysisBaseline visit (V1), study visit 2 (V2): 2-4 Weeks after enrolment; Visit 3: 3 - 21 days after Visit 2, and final study visit (V4) after at maximum 95 days

Measurement with blood gas analyzer for determination of ketosis

Analysis of the ketonic state with betahydroxybutyrate levelBaseline visit (V1), during 14 days of ketogenic diet or 3 days fasting and final study visit (V4) after at maximum 95 days

Betahydroxybutyrate measurement from blood samples of the fingertip by patients at home

Analysis of the ketonic state with ketonuriaBaseline visit (V1), during 14 days of ketogenic diet or 3 days fasting and final study visit (V4) after at maximum 95 days

Ketonuria measured with urine stix by patients at home

Analysis of the ketonic state with acetoacetate in breathBaseline visit (V1), during 14 days of ketogenic diet or 3 days fasting and final study visit (V4) after at maximum 95 days

determination of acetoacetate in breath by patients at home

Absolute and relative change of heightBaseline visit (V1), V2 (2-4 Weeks after enrolment); Visit 3 (3 - 21 days after Visit 2 ) and final study visit (V4) after at maximum 95 days

Determination of height

Absolute and relative change of weightBaseline visit (V1), V2 (2-4 Weeks after enrolment); Visit 3 (3 - 21 days after Visit 2 ) and final study visit (V4) after at maximum 95 days

Determination of weight

Absolute and relative change of waist circumferenceBaseline visit (V1), V2 (2-4 Weeks after enrolment); Visit 3 (3 - 21 days after Visit 2 ) and final study visit (V4) after at maximum 95 days

Determination of waist circumference

Relative change of calorimetryBaseline visit (V1), V2 (2-4 Weeks after enrolment); Visit 3 (3 - 21 days after Visit 2 ) and final study visit (V4) after at maximum 95 days

Could be determined instead of anthropometric parameters

Changes in BioimpedanceBaseline visit (V1), V2 (2-4 Weeks after enrolment); Visit 3 (3 - 21 days after Visit 2 ) and final study visit (V4) after at maximum 95 days

Bioimpedance measurements on each visit

Feeling of hunger, discomforts and problemsIn between baseline visit (V1) and final study visit (V4) after at maximum 95 days

measured and documented at home in a study dietary in between study visits

Acetoacetate-concentrationsIn between baseline visit (V1) and final study visit (V4) after at maximum 95 days

measured and documented at home in a study dietary in between study visits

Trial Locations

Locations (1)

Department II of Internal Medicine, University Hospital Cologne

🇩🇪

Cologne, Germany

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