Long-chain 3-hydroxyacyl-CoA Dehydrogenase DeficiencyEarly Phase 1NCT05411835
Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders
A Early Phase 1 study testing Nutritional Ketone Supplement in people with Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency and Carnitine Palmitoyltransferase Deficiency 2, run by Oregon Health and Science University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Carnitine Palmitoyltransferase Deficiency 2, Very Long Chain Acyl Coa Dehydrogenase Deficiency
- What is being tested?
- Nutritional Ketone Supplement
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2022to 22 Jan 2024
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- confirmed diagnosis of VLCAD, LCHAD/TFP or CPT2 deficiency
- speak English
- willing to complete 2 moderate intensity exercise treadmills
Who cannot join
- subjects actively participating in another research study that prohibits their participation
- pregnant females
- subjects with diabetes or taking medications to treat diabetes
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health & Science University
Portland, Oregon, 97239
About this study
Official title: Safety and Tolerability of Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders
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.
Read the 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.
- Conditions
- Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Carnitine Palmitoyltransferase Deficiency 2, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Trifunctional Protein Deficiency
- Being tested
- Nutritional Ketone Supplement, Isocaloric Placebo Supplement
- Medical subject headings
- Trifunctional Protein Deficiency With Myopathy And Neuropathy, Carnitine palmitoyl transferase 2 deficiency, VLCAD deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nutritional Ketone SupplementDietary supplement
Also called NKS
Mix of sodium, calcium, and magnesium salts of D-beta-hydroxybutyrate with nicotinamide riboside chloride, flavors and stevia sweetener
Isocaloric Placebo SupplementDietary supplement
Maltodextrin with flavors and stevia sweetener
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Dietary Supplement: Isocaloric Placebo Supplement |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events- incidence
Number of Adverse events
Measured: 2 hours after product intake
Adverse Events- type
Description of Adverse events: categorical
Measured: 2 hours after product intake
Adverse Events- severity
Severity of adverse event: grade 1-5
Measured: 2 hours after product intake
Adverse Events- causal relationship
Yes/No related to product intake
Measured: 2 hours after product intake
Abdominal discomfort
visual analogue scale (VAS) 0-10
Measured: 2 hours after product intake
Decreased appetite
visual analogue scale (VAS) 0-10
Measured: 2 hours after product intake
Gastric reflux
visual analogue scale (VAS) 0-10
Measured: 2 hours after product intake
Nausea
visual analogue scale (VAS) 0-10
Measured: 2 hours after product intake
Diarrhea
visual analogue scale (VAS) 0-10
Measured: 2 hours after product intake
Headache
visual analogue scale (VAS) 0-10
Measured: 2 hours after product intake
Secondary outcomes (9)
Blood Ketones
plasma beta-hydroxybutyrate concentration
Measured: 20 minutes after moderate intensity exercise
Blood Creatine Kinase
plasma CK concentration
Measured: 20 minutes after moderate intensity exercise
Blood Glucose
serum glucose
Measured: 20 minutes after moderate intensity exercise
Blood Lactate
serum lactate concentration
Measured: 20 minutes after moderate intensity exercise
Blood Pressure
diastolic BP
Measured: peak 40 minute exercise
Blood Pressure
systolic BP
Measured: peak 40 minute exercise
Perceived exertion
Borg scale 1-20
Measured: peak 40 minute exercise
Respiratory Exchange Ratio
VCO2/VO2 0.7 - 1.0
Measured: peak 40 minute exercise
Heart Rate
beats per minute
Measured: peak 40 minute exercise
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2022
- Study start1 Aug 2022
- Primary completion31 Jan 2023
- Study completion22 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Long-chain 3-hydroxyacyl-CoA Dehydrogenase DeficiencyStudies of the same condition, open and under way.
- More trials run by Oregon Health and Science UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- 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.
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oregon Health and Science University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Melanie B Gillingham, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05411835
- Sponsor's protocol ID
- 23859
- Status last verified
- Oct 2022
- First submitted
- 27 May 2022
- Last updated
- 2 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Participants will be provided with creatine kinase lab test and body composition measurement. The results of their creatine kinase lab test will be placed in their medical record. Results of the study will be written in a published manuscript and summarized in a cover letter to be sent via mail or email to the participant after publication of the data.
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Tolerability of Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders
- ClinicalTrials.gov ID
- NCT05411835
- Acronym / sponsor ID
- 23859
- Conditions
- Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Carnitine Palmitoyltransferase Deficiency 2, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Trifunctional Protein Deficiency
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Study start
- 1 Aug 2022 (actual)
- Primary completion
- 31 Jan 2023 (actual)
- Study completion
- 22 Jan 2024 (actual)
- First posted
- 9 Jun 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Nutrition Ketogenic Supplement | Active comparator | Dietary Supplement: Nutritional Ketone SupplementThe 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. |
| Isocaloric Placebo Supplement | Sham comparator | Dietary Supplement: Isocaloric Placebo SupplementThe 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- confirmed diagnosis of VLCAD, LCHAD/TFP or CPT2 deficiency
- speak English
- willing to complete 2 moderate intensity exercise treadmills
Key exclusion criteria (3 listed)
- subjects actively participating in another research study that prohibits their participation
- pregnant females
- subjects with diabetes or taking medications to treat diabetes
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nutritional Ketone SupplementAlso: NKSMix of sodium, calcium, and magnesium salts of D-beta-hydroxybutyrate with nicotinamide riboside chloride, flavors and stevia sweetener | Dietary supplement | Nutrition Ketogenic Supplement |
| Isocaloric Placebo SupplementMaltodextrin with flavors and stevia sweetener | Dietary supplement | Isocaloric Placebo Supplement |
Primary outcome
Adverse Events- incidence
Time frame: 2 hours after product intake
Number of Adverse events
Adverse Events- type
Time frame: 2 hours after product intake
Description of Adverse events: categorical
Adverse Events- severity
Time frame: 2 hours after product intake
Severity of adverse event: grade 1-5
Adverse Events- causal relationship
Time frame: 2 hours after product intake
Yes/No related to product intake
Abdominal discomfort
Time frame: 2 hours after product intake
visual analogue scale (VAS) 0-10
Decreased appetite
Time frame: 2 hours after product intake
visual analogue scale (VAS) 0-10
Gastric reflux
Time frame: 2 hours after product intake
visual analogue scale (VAS) 0-10
Nausea
Time frame: 2 hours after product intake
visual analogue scale (VAS) 0-10
Diarrhea
Time frame: 2 hours after product intake
visual analogue scale (VAS) 0-10
Headache
Time frame: 2 hours after product intake
visual analogue scale (VAS) 0-10
9 secondary outcomes
Blood Ketones
Time frame: 20 minutes after moderate intensity exercise
Blood Creatine Kinase
Time frame: 20 minutes after moderate intensity exercise
Blood Glucose
Time frame: 20 minutes after moderate intensity exercise
Blood Lactate
Time frame: 20 minutes after moderate intensity exercise
Blood Pressure
Time frame: peak 40 minute exercise
Blood Pressure
Time frame: peak 40 minute exercise
Perceived exertion
Time frame: peak 40 minute exercise
Respiratory Exchange Ratio
Time frame: peak 40 minute exercise
Heart Rate
Time frame: peak 40 minute exercise
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Apr 2024.
- Individual participant dataSharing planned: Yes. Participants will be provided with creatine kinase lab test and body composition measurement. The results of their creatine kinase lab test will be placed in their medical record. Results of the study will be written in a published manuscript and summarized in a cover letter to be sent via mail or email to the participant after publication of the data.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Nutritional Ketone Supplement in people with Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency and Carnitine Palmitoyltransferase Deficiency 2, run by Oregon Health and Science University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events- incidence (time frame: 2 hours after product intake). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Apr 2024.