Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders

NCT05411835 | Completed Early Phase 1

• 1 other identifier: 23859
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency; Carnitine Palmitoyltransferase Deficiency 2; Very Long Chain Acyl Coa Dehydrogenase Deficiency; Trifunctional Protein Deficiency

Outcome Measures

Primary Outcomes (10)

• Adverse Events- incidence

  Number of Adverse events

  2 hours after product intake

• Adverse Events- type

  Description of Adverse events: categorical

  2 hours after product intake

• Adverse Events- severity

  Severity of adverse event: grade 1-5

  2 hours after product intake

• Adverse Events- causal relationship

  Yes/No related to product intake

  2 hours after product intake

• Abdominal discomfort

  visual analogue scale (VAS) 0-10

  2 hours after product intake

• Decreased appetite

  visual analogue scale (VAS) 0-10

  2 hours after product intake

• Gastric reflux

  visual analogue scale (VAS) 0-10

  2 hours after product intake

• Nausea

  visual analogue scale (VAS) 0-10

  2 hours after product intake

• Diarrhea

  visual analogue scale (VAS) 0-10

  2 hours after product intake

• Headache

  visual analogue scale (VAS) 0-10

  2 hours after product intake

Secondary Outcomes (9)

• Blood Ketones

  20 minutes after moderate intensity exercise

• Blood Creatine Kinase

  20 minutes after moderate intensity exercise

• Blood Glucose

  20 minutes after moderate intensity exercise

• Blood Lactate

  20 minutes after moderate intensity exercise

• Blood Pressure

  peak 40 minute exercise

Study Arms (2)

Nutrition Ketogenic Supplement

ACTIVE COMPARATOR

The Ketone pre-exercise beverage is a nutritional supplement. The supplement will be provided by Nestle as powered sachets to mix in water. It contains a mix of D-beta-hydroxybutyrate salts, flavors and stevia.

Dietary Supplement: Nutritional Ketone Supplement

Isocaloric Placebo Supplement

SHAM COMPARATOR

The maltodextrin pre-exercise beverage is a nutritional supplement. The supplement will be provided by Nestle as powered sachets to mix in water. It contains an isocaloric amount of maltodextrin, flavors and stevia similar to the ketone beverage.

Dietary Supplement: Isocaloric Placebo Supplement

Interventions

Nutritional Ketone Supplement DIETARY_SUPPLEMENT

Mix of sodium, calcium, and magnesium salts of D-beta-hydroxybutyrate with nicotinamide riboside chloride, flavors and stevia sweetener

Also known as: NKS

Nutrition Ketogenic Supplement

Isocaloric Placebo Supplement DIETARY_SUPPLEMENT

Maltodextrin with flavors and stevia sweetener

Isocaloric Placebo Supplement

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Oregon Health and Science University: lead

Study Sites (1)

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Trifunctional Protein Deficiency With Myopathy And Neuropathy; Carnitine palmitoyl transferase 2 deficiency; VLCAD deficiency

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Sachets of the ketone or maltodextrin powder are coded and packaged identically. The bionutrition unit will prepare the beverage and maintain the randomization code and the code for the ketone and maltodextrin products.
• Purpose: OTHER
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Model Details: 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.

Study Record Dates

• First Submitted: May 27, 2022
• First Posted: June 9, 2022
• Study Start: August 1, 2022
• Primary Completion: January 31, 2023
• Study Completion: January 22, 2024
• Last Updated: April 2, 2024

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF
• Time Frame: The published manuscript will be available approximately 6-9 months after the conclusion of the study.
• Access Criteria: not applicable. A reprint will be sent to participants.

Locations

US (1)