Ketone Supplementation in Cystic Fibrosis

Ketone Monoester Supplementation in Cystic Fibrosis: A Pilot and Feasibility Study

This study will examine the effects of a novel ketone monester supplement on patients with cystic fibrosis experiencing an acute pulmonary exacerbation requiring hospitalization. Patients will undergo standard testing along with blood sampling to examine concentrations of inflammatory markers. Patients will then receive the ketone supplement for 5 days followed by post-testing to examine changes in pulmonary function and inflammatory markers

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Dietary supplement: Ketone Monoester

Detailed Description

Up to 25 hospitalized patients with cystic fibrosis will take part in this study at UAB. Patients will be randomly assigned to receive either an exogenous ketone or placebo supplement. Participants will take the ketone or placebo for 5 days during hospitalization. Participants will drink one 60 mL bottle twice daily of either the ketone or placebo. On day 1 and day 5, before the patient drinks the ketone (or placebo) the patient will be asked to fast overnight for 8 hours. Following administration of the first morning dose of ketones (or placebo) blood samples will be obtained from a finger stick before (time 0 min) and following (15, 30, 60, 90, and 120 min) after they drink the supplement. A commercially available ketone meter (Keto-Mojo, Napa, CA) that provides instant assessment of circulating ketone concentrations will be used to measure ketone concentrations in the blood. Medical history history will be obtained at hospitalization. The study team will also examine the body for any abnormal signs and symptoms. Sputum and blood will be collected prior to and following the ketone supplement or placebo. The hypothesis is that ketone supplementation will reduce inflammation via a well known inflammatory pathway to improve outcomes in patients with cystic fibrosis experiencing an acute pulmonary exacerbation

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Eric P Plaisance, PhD; Phone Number: 205-996-7909; Email: plaisep@uab.edu
• Study Contact Backup: Name: Kevin R Fontaine, PhD; Phone Number: 205-975-8397; Email: kfontai1@uab.edu

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 89 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Diagnosis of CF
• Age >19
• Colonization with P. Aeruginosa
• Acute pulmonary exacerbation requiring inpatient care

Exclusion Criteria:

• Concurrent or recent (within 28 d of enrollment) use of corticosteroids
• Inability to produce sputum
• Acute respiratory failure
• Chronic liver or renal disease
• Pregnancy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ketone Monoester; The ketone used is from KetoneAid and is commercially available as KE4 and will be consumed at 30 mL twice daily (15 g / dose)	Dietary supplement: Ketone Monoester; Ketone supplement will be taken by mouth at a dose of 15 mL twice per day
Placebo Comparator: Placebo; A placebo is being manufactured by KetoneAid to match the taste and other characteristics of the ketone supplement for blinding purposes	Dietary supplement: Ketone Monoester; Ketone supplement will be taken by mouth at a dose of 15 mL twice per day

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pulmonary Function Testing	FEV1.0 (L/sec)	5 days
Inflammatory markers	Interleukin 1Beta (ug/mL)	5 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of quality of life, respiratory symptoms, and patient outlook	A questionnaire will be used to assess quality of life and patient outlook 30 days following the intervention	5 days

Collaborators and Investigators

• Sponsor: University of Alabama at Birmingham
• Investigators: Principal Investigator: Eric P Plaisance, PhD, University of Alabama at Birmingham

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2021
• Primary Completion (Actual): April 30, 2023
• Study Completion (Actual): April 30, 2023

Study Registration Dates

• First Submitted: June 9, 2021
• First Submitted That Met QC Criteria: June 15, 2021
• First Posted (Actual): June 24, 2021

Study Record Updates

• Last Update Posted (Actual): June 6, 2023
• Last Update Submitted That Met QC Criteria: June 5, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: IRB-300007323

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No