Clinical trial NCT05411835: Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders
Official title: Safety and Tolerability of Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
Carnitine Palmitoyltransferase Deficiency 2
Very Long Chain Acyl Coa Dehydrogenase Deficiency
Trifunctional Protein Deficiency
Lead sponsor: Oregon Health and Science University
Sponsor class: OTHER
First posted: 2022-06-09
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: Subjects with either CPT2, TFP, LCHAD or VLCAD deficiency will be randomized to receive a ketone beverage (see composition in Appendix A) or an isocaloric maltodextrin control beverage (see composition in Appendix A) before moderate intensity exercise. Twenty minutes after consuming the pre-exercise beverage, subjects will complete a moderate intensity treadmill exercise test for 45 minutes. After a washout period of 48 hours, subjects will repeat the exercise. During the 2nd exercise test subjects will crossover to the beverage not consumed during the 1st exercise study.
Primary purpose: OTHER

Brief summary: The purpose of the study is to determine if an oral ketone beverage is safe and well-tolerated during moderate intensity exercise in participants with long-chain fatty acid oxidation disorders and if it will raise blood ketones to levels similar to that reported among normal healthy subjects.

Detailed description: Purpose: Subjects with long-chain fatty acid oxidation disorders (LC-FAOD) do not make ketones during fasting or with exercise. Ketones are an important alternative energy substrate during moderate exercise, sparing the oxidation of glucose and providing a source of ATP to the central nervous system and exercising muscle. Fatty acid oxidation in the liver is required to make ketones. Subjects with a LC-FAOD cannot generate ketones because of their block in fatty acid oxidation during exercise. Providing ketones in an oral ketone beverage may increase blood ketones with exercise to levels normally observed in humans.

Aim: To determine the safety and tolerability of an oral ketone beverage during moderate intensity exercise among subjects with a LC-FAOD compared to an isocaloric maltodextrin beverage, and to determine blood ketone concentrations.

Hypothesis: Oral consumption of a ketone beverage before moderate intensity exercise will be safe and well-tolerated, and will raise blood ketones among subjects with a LC-FAOD to concentrations similar to that reported in the literature among normal healthy subjects.

Eligibility criteria: Inclusion Criteria:

* confirmed diagnosis of VLCAD, LCHAD/TFP or CPT2 deficiency
* speak English
* willing to complete 2 moderate intensity exercise treadmills

Exclusion Criteria:

* subjects actively participating in another research study that prohibits their participation
* pregnant females
* subjects with diabetes or taking medications to treat diabetes

Primary outcomes:
measure: Adverse Events- incidence
timeFrame: 2 hours after product intake
measure: Adverse Events- type
timeFrame: 2 hours after product intake
measure: Adverse Events- severity
timeFrame: 2 hours after product intake
measure: Adverse Events- causal relationship
timeFrame: 2 hours after product intake
measure: Abdominal discomfort
timeFrame: 2 hours after product intake
measure: Decreased appetite
timeFrame: 2 hours after product intake
measure: Gastric reflux
timeFrame: 2 hours after product intake
measure: Nausea
timeFrame: 2 hours after product intake
measure: Diarrhea
timeFrame: 2 hours after product intake
measure: Headache
timeFrame: 2 hours after product intake
Secondary outcomes:
measure: Blood Ketones
timeFrame: 20 minutes after moderate intensity exercise
measure: Blood Creatine Kinase
timeFrame: 20 minutes after moderate intensity exercise
measure: Blood Glucose
timeFrame: 20 minutes after moderate intensity exercise
measure: Blood Lactate
timeFrame: 20 minutes after moderate intensity exercise
measure: Blood Pressure
timeFrame: peak 40 minute exercise
measure: Blood Pressure
timeFrame: peak 40 minute exercise
measure: Perceived exertion
timeFrame: peak 40 minute exercise
measure: Respiratory Exchange Ratio
timeFrame: peak 40 minute exercise
measure: Heart Rate
timeFrame: peak 40 minute exercise
Other outcomes:
Locations:
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
Study officials:
References: