Carnitine Consumption and Augmentation in Pulmonary Arterial Hypertension

What Was Tested

The trial (phase 1) is built around L-Carnitine.

Investigators propose three aims in humans to test this mechanistic hypothesis, 1) Measure the oral consumption of carnitine in human PAH.

This aim will use food diaries and carnitine supplement use questionnaires in PAH patients to test the hypothesis that carnitine supplementation is uncommon in PAH and food consumption is adequate.

It had 1 arm (groups): Supplement (experimental).

The registry classes the interventions as dietary supplement.

More on the Plan

No RV-specific therapies are available, in part because the underlying mechanisms of RV failure are poorly understood.

Interventions against metabolic dysfunction in PAH may protect against RV failure.

Investigators in the PH research group have identified abnormalities in fatty acid (FA) metabolism in PAH that overlap considerably with disorders of carnitine deficiency.

Carnitine links to an acyl group, which is required to transport FAs across the mitochondrial membrane to undergo beta-oxidation, the predominate source of ATP production in the human heart.

Who Took Part

Enrollment: 10 actual participants took part.

It was open to people of any sex aged from 18 years up to 85 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Adults aged 18 or older. Diagnosed with idiopathic, heritable, simple congenital heart defect, or drug- or toxin-associated pulmonary arterial hypertension (PAH) according to World Health Organization consensus recommendations. WHO Functional Class II-IV.

The full record lists 6 inclusion and 8 exclusion criteria; among the exclusions: drug and toxin associated pah patients with active drug use; prior diagnosis of cirrhosis; known allergy to l-carnitine supplements.

How the Study Was Run

Design: non-randomized, single-group and open-label.

Everyone knew when L-Carnitine was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2021-09-29 with completion listed for 2023-06-01.

It ran at 1 site in United States.

Its stated primary purpose is other.

What Was Measured

Researchers chose this as the main measure: Plasma Carnitine Concentration (time frame: 2 weeks of intervention).

It also tracked 7 secondary outcomes, for example Prevalence of Carnitine Supplement Use, Carnitine Ingestion Use Through Food, Six-minute Walk, WHO Functional Class, Patient Reported Side Effects and Echocardiography Measurements of TAPSE and RV Fractional Area.

What This Means in Plain Language

Put simply, this is a phase 1, non-randomized trial of L-Carnitine in 10 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the L-Carnitine compound page, all human trials.