Cystic Fibrosis
Conditions
Brief summary
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
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
Interventions
DIETARY_SUPPLEMENTKetone Monoester
Ketone supplement will be taken by mouth at a dose of 15 mL twice per day
Sponsors
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
University of Alabama at Birmingham
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
All involved with blinded, except for the study pharmacist
Intervention model description
Two-way ANOVA with repeated measures on time
Eligibility
Sex/Gender
ALL
Age
18 Years to 89 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Pulmonary Function Testing | 5 days | FEV1.0 (L/sec) |
| Inflammatory Markers | 5 days | Interleukin 1Beta (pg/mL) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Subjective Respiratory Quality of Life | 5-7 days | The Cystic Fibrosis Questionnaire-Revised (CFQ-R) was used to examine multiple domains related to quality of life. An outcome of the study was to examine subjective ratings of respiratory function using the CFQ-R comparing the ketone treated (KME) vs. control (placebo-control) groups. The measurement range for the score is from 0 - 100 with higher values representing a better outcome. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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)
Ketone Monoester: Ketone supplement will be taken by mouth at a dose of 15 mL twice per day | 10 |
| Placebo A placebo is being manufactured by KetoneAid to match the taste and other characteristics of the ketone supplement for blinding purposes
Ketone Monoester: Ketone supplement will be taken by mouth at a dose of 15 mL twice per day | 5 |
| Total | 15 |
Baseline characteristics
| Characteristic | Ketone Monoester | Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 10 Participants | 5 Participants | 15 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 10 Participants | 4 Participants | 14 Participants |
| Region of Enrollment United States | 10 Participants | 5 Participants | 15 Participants |
| Sex: Female, Male Female | 4 Participants | 4 Participants | 8 Participants |
| Sex: Female, Male Male | 6 Participants | 1 Participants | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 10 | 0 / 5 |
| other Total, other adverse events | 0 / 10 | 0 / 5 |
| serious Total, serious adverse events | 0 / 10 | 0 / 5 |
Outcome results
Primary
Inflammatory Markers
Interleukin 1Beta (pg/mL)
Time frame: 5 days
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ketone Monoester | Inflammatory Markers | 0.3 pg/mL | Standard Deviation 0.1 |
| Placebo | Inflammatory Markers | 0.1 pg/mL | Standard Deviation 0.1 |
Primary
Pulmonary Function Testing
FEV1.0 (L/sec)
Time frame: 5 days
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ketone Monoester | Pulmonary Function Testing | 1.9 L/sec | Standard Deviation 0.9 |
| Placebo | Pulmonary Function Testing | 2.0 L/sec | Standard Deviation 1 |
Secondary
Subjective Respiratory Quality of Life
The Cystic Fibrosis Questionnaire-Revised (CFQ-R) was used to examine multiple domains related to quality of life. An outcome of the study was to examine subjective ratings of respiratory function using the CFQ-R comparing the ketone treated (KME) vs. control (placebo-control) groups. The measurement range for the score is from 0 - 100 with higher values representing a better outcome.
Time frame: 5-7 days
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ketone Monoester | Subjective Respiratory Quality of Life | 70.1 units on a scale | Standard Deviation 28.5 |
| Placebo | Subjective Respiratory Quality of Life | 60.3 units on a scale | Standard Deviation 26 |