Aberrations in Carnitine Homeostasis in Congenital Heart Disease With Increased Pulmonary Blood Flow

What Was Tested

In this phase 1 trial, researchers set out to test L-Carnitine.

To this end, a pilot clinical trial is proposed.

It had 1 arm (groups): IV L-carnitine (experimental).

The registry classes the interventions as drug.

More on the Plan

Serial blood samples will be obtained to determine free, total, and acylcarnitine levels, and plasma markers of mitochondrial function, oxidative stress, and bioavailable NO.

Adverse events will be sought, and clinical outcomes will be assessed.

Study design: The inclusion and exclusion criteria are as described in Aim 3A except only infants with VSD or AVSD will be enrolled (no TOF).

The safety profile of L-carnitine is outstanding, with no reports of toxicity from overdose reported113.

Who Took Part

It was open to people of any sex aged from 2 months up to 12 months.

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

Examples of who could join: Have unrestrictive VSD, AVSD. Are undergoing complete repair. Are between 2-12 months of age. Are corrected gestational age ≥34 weeks. Will have an indwelling arterial or venous line.

The full record lists 6 inclusion and 5 exclusion criteria; among the exclusions: have body weight \< 2.0 kg; pulmonary artery or vein abnormalities not being addressed surgically; suspected or proven in-born error of metabolism.

How the Study Was Run

The set-up was 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 2014-12 with completion listed for 2020-07.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Blood carnitine level (free, total, and acylcarnitine).

It also tracked 13 secondary outcomes, for example Bioavailable nitric oxide, Plasma levels of superoxide, Carnitine Palmityl Transporter-1 and -2 expression, Cardiopulmonary bypass, Echocardiographic measurements and Blood BNP level.

What This Means in Plain Language

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

It was withdrawn before enrolling anyone; no results are posted to the registry yet, so nothing here describes outcomes.

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