Trial Outcomes & Findings for Acute Effects of Medium Chain Triglyceride (MCT) Nutritional Ketosis on Parkinson's Disease (PD) Symptoms and Biomarkers (MCT-PD) (NCT NCT04584346)

Last Updated: 2022-09-13

Results Overview

Analysis of feasibility was determined by 3 co-primary endpoints: retention, adherence, and acceptability, measured at the end of week 3 (outpatient segment). After each co-primary endpoint was calculated, three benchmark criteria were used to determine feasibility. All criteria must be met for feasibility to be positive. Benchmark criteria for Retention was defined as a completion rate at study end (week 3) of \>80%, i.e., \>80% of participants must remain in the study at the 3 week time point.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

21 participants

Primary outcome timeframe

Week 3

Results posted on

2022-09-13

Participant Flow

Potential participants were recruited from the pool of participants seen in the NIH Parkinson's Clinic and patients with Parkinson's disease who have participated in other NINDS protocols. Recruitment occurred between January and October 2021.

Participants were consented and screened for eligibility. 21 participants were screened and 17 participants met eligibility criteria. One participant withdrew after screening but prior to randomization. Eligible participants were randomized in a blinded manner to receive either a Ketogenic diet supplemented with MCT oil or a Standard American Diet x 6 days while an inpatient. This was followed by both groups receiving a Ketogenic Diet supplemented with MCT oil at home x 2 weeks (unblinded).

Participant milestones

Participant milestones
Measure	Ketogenic Diet Participants consumed a ketogenic diet supplemented with MCT oil x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.	Standard American Diet Participants consumed a standard American diet x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.
Phase 1 - Inpatient (Blinded) STARTED	7	9
Phase 1 - Inpatient (Blinded) COMPLETED	7	9
Phase 1 - Inpatient (Blinded) NOT COMPLETED	0	0
Phase 2 - At Home (Unblinded) STARTED	7	9
Phase 2 - At Home (Unblinded) COMPLETED	7	8
Phase 2 - At Home (Unblinded) NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Ketogenic Diet Participants consumed a ketogenic diet supplemented with MCT oil x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.	Standard American Diet Participants consumed a standard American diet x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.
Phase 2 - At Home (Unblinded) Withdrawal by Subject	0	1

Baseline Characteristics

Acute Effects of Medium Chain Triglyceride (MCT) Nutritional Ketosis on Parkinson's Disease (PD) Symptoms and Biomarkers (MCT-PD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ketogenic Diet n=7 Participants Participants consumed a ketogenic diet supplemented with MCT oil x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.	Standard American Diet n=9 Participants Participants consumed a standard American diet x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.	Total n=16 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Age, Categorical >=65 years	5 Participants n=5 Participants	6 Participants n=7 Participants	11 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	5 Participants n=7 Participants	7 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	4 Participants n=7 Participants	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=5 Participants	7 Participants n=7 Participants	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants	7 Participants n=7 Participants	13 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 3

Population: Number of participants remaining in the study at week 3.

Outcome measures

Outcome measures
Measure	All Participants n=16 Participants Participants consumed a ketogenic diet supplemented with MCT oil (at home) for two weeks.	Standard American Diet Participants consumed a standard American diet x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.
Feasibiilty of Ketogenic Diet - Retention (Co-primary Endpoint)	15 Participants	—

PRIMARY outcome

Timeframe: Week 3

Population: Average net carbohydrate intake across all participants during the 2 week outpatient period.

Analysis of feasibility was determined by 3 co-primary endpoints: retention, adherence, and acceptability, measured at the end of week 3 (outpatient segment). After each co-primary endpoint was calculated, three benchmark criteria were used to determine feasibility. All criteria must be met for feasibility to be positive. Benchmark criteria for Adherence was defined as a mean net carbohydrate intake of \</=10% during the 2 week outpatient period. Mean net carbohydrate intake was determined using the following calculation: (total carbohydrates minus total dietary fiber) x 4 divided by total calories.

Outcome measures

Outcome measures
Measure	All Participants n=15 Participants Participants consumed a ketogenic diet supplemented with MCT oil (at home) for two weeks.	Standard American Diet Participants consumed a standard American diet x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.
Feasibility of Ketogenic Diet - Adherence (Co-primary Endpoint)	9.7 percentage of diet Standard Deviation 3.2	—

PRIMARY outcome

Timeframe: Week 3

Population: Average score on the Likert scale for diet acceptability across all participants at week 3.

Analysis of feasibility was determined by 3 co-primary endpoints: retention, adherence, and acceptability, measured at the end of week 3 (outpatient segment). After each co-primary endpoint was calculated, three benchmark criteria were used to determine feasibility. All criteria must be met for feasibility to be positive. Acceptability was defined via an exit survey (at end of study week 3) using a 4-point Likert scale to indicate how likely the participant would continue the diet on at least an intermittent basis in the future with 1 representing "Very likely" and 4 representing "Very unlikely". The benchmark criteria for Acceptability was defined as at least 2 out of 4 on the Likert scale.

Outcome measures

Outcome measures
Measure	All Participants n=15 Participants Participants consumed a ketogenic diet supplemented with MCT oil (at home) for two weeks.	Standard American Diet Participants consumed a standard American diet x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.
Feasibility of Ketogenic Diet - Acceptability (Co-primary Endpoint)	2.26 score on a scale Standard Deviation 0.96	—

SECONDARY outcome

Timeframe: Day 7

The Timed Up and Go (TUG) test is a simple test used to assess a person's mobility. The TUG measures the time required to perform a sequence of activities, i.e.,sit-to-stand transfer, straight walking, turning, and walk-to-sit transfer. The TUG is administered at baseline, and each day during the inpatient visit. The results represent a comparison of the group mean score at the end of admission (day 7) for the two cohorts, i.e., patients receiving a Ketogenic Diet and patients receiving a Standard American Diet. A time of greater than 13.5 seconds may suggest a greater risk of falls.

Outcome measures

Outcome measures
Measure	All Participants n=7 Participants Participants consumed a ketogenic diet supplemented with MCT oil (at home) for two weeks.	Standard American Diet n=9 Participants Participants consumed a standard American diet x 6 days (inpatient) during phase 1, followed by a 2 week ketogenic diet supplemented with MCT oil (at home) during phase 2.
Timed Up and Go (TUG)	8.4 Seconds Standard Deviation 1.2	9.1 Seconds Standard Deviation 2.3

Adverse Events

Ketogenic Diet - Phase 1

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Standard American Diet - Phase 1

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Ketogenic Diet - Phase 2

Serious events: 1 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Ketogenic Diet - Phase 1 n=7 participants at risk Participants consumed a ketogenic diet supplemented with MCT oil x 6 days (inpatient) during phase 1.	Standard American Diet - Phase 1 n=9 participants at risk Participants consumed a standard American diet x 6 days (inpatient) during phase 1.	Ketogenic Diet - Phase 2 n=15 participants at risk Participants consumed a ketogenic diet supplemented with MCT oil x for 2 weeks (home) during phase 2.
Blood and lymphatic system disorders Pulmonary Embolus	0.00% 0/7 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	6.7% 1/15 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.

Other adverse events

Additional Information

Dr. Debra Ehrlich

National Institutes of Health

Phone: 301-443-7888

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Ketogenic Diet - Phase 1 n=7 participants at risk Participants consumed a ketogenic diet supplemented with MCT oil x 6 days (inpatient) during phase 1.	Standard American Diet - Phase 1 n=9 participants at risk Participants consumed a standard American diet x 6 days (inpatient) during phase 1.	Ketogenic Diet - Phase 2 n=15 participants at risk Participants consumed a ketogenic diet supplemented with MCT oil x for 2 weeks (home) during phase 2.
Musculoskeletal and connective tissue disorders Lower back pain	14.3% 1/7 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/15 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Musculoskeletal and connective tissue disorders Generalized muscle ache with headache	14.3% 1/7 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/15 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Gastrointestinal disorders Indegestion with acid reflux	28.6% 2/7 • Number of events 2 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	11.1% 1/9 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/15 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Injury, poisoning and procedural complications Headache s/p hit head	14.3% 1/7 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/15 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Nervous system disorders Headache	0.00% 0/7 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	11.1% 1/9 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	6.7% 1/15 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Gastrointestinal disorders Constipation	14.3% 1/7 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	13.3% 2/15 • Number of events 2 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Gastrointestinal disorders Increased salivation	14.3% 1/7 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/15 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Metabolism and nutrition disorders Fatigue	14.3% 1/7 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	11.1% 1/9 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	33.3% 5/15 • Number of events 5 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	14.3% 1/7 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	6.7% 1/15 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Cardiac disorders Hypotension	0.00% 0/7 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	11.1% 1/9 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/15 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Nervous system disorders Dyskinesia (increased from baseline)	0.00% 0/7 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	22.2% 2/9 • Number of events 2 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/15 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Gastrointestinal disorders Diarrhea	0.00% 0/7 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	11.1% 1/9 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	6.7% 1/15 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Musculoskeletal and connective tissue disorders Flexing and External rotation of toes	0.00% 0/7 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	6.7% 1/15 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Metabolism and nutrition disorders Malaise	0.00% 0/7 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	6.7% 1/15 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.
Metabolism and nutrition disorders Nausea	0.00% 0/7 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	0.00% 0/9 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.	6.7% 1/15 • Number of events 1 • Adverse events were collected with start dates occurring any time after informed consent was obtained until 7 days (for non-serious AE) or 30 days (for SAEs) after the last day of study participation.