Trial Outcomes & Findings for Carnitine Consumption and Augmentation in Pulmonary Arterial Hypertension (NCT NCT04908397)

NCT ID: NCT04908397

Last Updated: 2025-09-30

Results Overview

Change in plasma Carnitine concentration from Visit 2 (Week 12) to Visit 4 (Week 14).

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 10 participants
• Primary outcome timeframe: 2 weeks of intervention
• Results posted on: 2025-09-30

Participant Flow

Single arm study

Participant milestones

Measure	Supplement Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Overall Study STARTED	10
Overall Study Visit 1	9
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Supplement Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Carnitine Consumption and Augmentation in Pulmonary Arterial Hypertension

Baseline characteristics by cohort

Measure	Supplement n=10 Participants Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	47.3 years STANDARD_DEVIATION 9.3 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Functional Class Functional Class I	1 Participants n=5 Participants
Functional Class Functional Class II	8 Participants n=5 Participants
Functional Class Functional Class III	0 Participants n=5 Participants
Functional Class Not evaluated	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 weeks of intervention

Change in plasma Carnitine concentration from Visit 2 (Week 12) to Visit 4 (Week 14).

Outcome measures

Measure	Supplement n=7 Participants Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Plasma Carnitine Concentration	44 uM Interval 37.3 to 48.7

SECONDARY outcome

Timeframe: 12 weeks

Population: Number of participants reporting carnitine supplement use

Quantify the prevalence of Carnitine supplement use of Carnitine in PAH patients by evaluating the number of study participants that report use of a daily carnitine supplement in baseline dietary reporting.

Outcome measures

Measure	Supplement n=7 Participants Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Prevalence of Carnitine Supplement Use Baseline	0 Participants
Prevalence of Carnitine Supplement Use 12 weeks	0 Participants

SECONDARY outcome

Timeframe: Reported week 1 (3 days leading up to visit 1) and week 12 (3 days leading up to visit 2).

Population: Number of subjects that ate a standard diet that included carnitine meat, fish and/or eggs, which provide Carnitine.

Measure oral ingestion of Carnitine in PAH patients by assessing food intake recorded by diary. Reports lacking meat, fish, and/or eggs are deemed "no ingestion by diet".

Outcome measures

Measure	Supplement n=7 Participants Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Carnitine Ingestion Use Through Food Week 1	7 Participants
Carnitine Ingestion Use Through Food Week 12	7 Participants

SECONDARY outcome

Timeframe: 14 weeks

Mean change in meters walked pre- and post- carnitine on 6mwt

Outcome measures

Measure	Supplement n=7 Participants Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Six-minute Walk	13.14286 meters Standard Deviation 17.9

SECONDARY outcome

Timeframe: 14 weeks

count of pre- and post-carnitine supplement and WHO functional class

Outcome measures

Measure	Supplement n=7 Participants Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
WHO Functional Class Pre-supplement · WHO functional class II	4 Participants
WHO Functional Class Pre-supplement · WHO functional class I	3 Participants
WHO Functional Class Post-supplement · WHO functional class I	2 Participants
WHO Functional Class Post-supplement · WHO functional class II	5 Participants

SECONDARY outcome

Timeframe: 2 weeks- between visit 2 and visit 4

Population: number of participants reporting side effects

Markers of tolerability of Carnitine supplement including presence of side effects, adverse events, and serious adverse events. Side effects to be reported beginning Visit 2, at carnitine start, and ended at Visit 4.

Outcome measures

Measure	Supplement n=7 Participants Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Patient Reported Side Effects	1 participants

SECONDARY outcome

Timeframe: 14 weeks

Correlation of change in plasma Carnitine with change in markers of RV function including TAPSE and RV fractional area change

Outcome measures

Measure	Supplement n=6 Participants Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Echocardiography Measurements of TAPSE and RV Fractional Area TAPSE- Spearman	0.543 correlation coefficient Interval -0.48 to 0.94
Echocardiography Measurements of TAPSE and RV Fractional Area RV fractional- Spearman	-0.086 correlation coefficient Interval -0.839 to 0.78
Echocardiography Measurements of TAPSE and RV Fractional Area TAPSE- Pearson	0.736 correlation coefficient Interval -0.187 to 0.969
Echocardiography Measurements of TAPSE and RV Fractional Area RV fractional area- Pearson	0.107 correlation coefficient Interval -0.772 to 0.845

SECONDARY outcome

Timeframe: 12 weeks- change from visit 1(week 1) to visit 2(week 12)

Change in plasma carnitine from visit 1 to visit 2

Outcome measures

Measure	Supplement n=9 Participants Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Stability of Plasma Carnitine	2.708 uM change Interval 0.249 to 7.154

Adverse Events

Supplement

Serious events: 1 serious events

Other events: 3 other events

Deaths: 1 deaths

Serious adverse events

Measure	Supplement n=10 participants at risk Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Cardiac disorders Chest pain	10.0% 1/10 • Number of events 1 • Participants were monitored for adverse events for a total of 16 weeks, from Visit 1 until 14 days following the last administration of study treatment.
Cardiac disorders Pericardial effusion	10.0% 1/10 • Number of events 1 • Participants were monitored for adverse events for a total of 16 weeks, from Visit 1 until 14 days following the last administration of study treatment.

Other adverse events

Measure	Supplement n=10 participants at risk Form: 500 mg L-carnitine tablet Dosage: Subjects 50-90kg: 3g/day Subjects \<50kg or \>90kg: 50mg/kg/day Frequency: twice a day for 2 weeks L-carnitine: supplement provided twice a day for 2 weeks
Musculoskeletal and connective tissue disorders Foot fracture	10.0% 1/10 • Number of events 1 • Participants were monitored for adverse events for a total of 16 weeks, from Visit 1 until 14 days following the last administration of study treatment.
Respiratory, thoracic and mediastinal disorders Respiratory symptoms	10.0% 1/10 • Number of events 1 • Participants were monitored for adverse events for a total of 16 weeks, from Visit 1 until 14 days following the last administration of study treatment.
General disorders Cough, dyspnea with exertion, fatigue	10.0% 1/10 • Number of events 1 • Participants were monitored for adverse events for a total of 16 weeks, from Visit 1 until 14 days following the last administration of study treatment.

Additional Information

Anna R. Hemnes

Vanderbilt University Medical Center

Phone: 615-343-8227

Email: anna.r.hemnes@vumc.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place