Propionyl-L-Carnitine Hydrochloride in Patients With Mild Ulcerative Colitis; Efficacy, Safety and Tolerability Study

What Was Tested

In this phase 3 trial, researchers set out to test L-Carnitine.

It had two arms (groups): Propionyl-L-Carnitine (experimental) and Placebo (placebo comparator).

Modified release tablets containing inert substances.

The registry classes the interventions as drug.

More on the Plan

Although it seems clear that an abnormal function of the colonic epithelium is the central problem causing inflammation and the unusual immunological response to the normal gut flora in inflammatory bowel disease (IBD), the actual causes of these dysfunctions are still unknown.

Short Chain Fatty Acids (SCFA) are the main fuel of the colonic epithelium, and are normally produced by the bacterial flora by fermentation of the complex carbohydrates forming non soluble fibers usually introduced with everyday diet.

As far as the UC pathology is concerned, the effects of ST 261, given orally or intrarectally, were investigated at different dosages, in preclinical experimentation, either after a single trinitrobenzene sulphonic acid (TNBS) administration (acute colitis) or after repeated TNBS administrations (reactivated colitis).

Who Took Part

Enrollment: 150 actual participants took part.

It was open to people of any sex aged from 18 years up to 75 years.

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

Examples of who could join: Have read the Information for the Patient and signed the Informed Consent Form. Diagnosis of active ulcerative colitis since at least 4 weeks as confirmed endoscopically and histologically. Disease Activity Index comprised between 3 and 6, inclusive (mild ulcerative colitis), with rectal bleeding sub-score of at least 1. Stable background oral aminosalicylates (mesalazine, balsalazide, olsalazine) or sulfasalazine standard therapy for greater than or equal to 4 weeks prior to screening assessments.

The full record lists 5 inclusion and 18 exclusion criteria; among the exclusions: crohn's disease and indeterminate colitis; current or previous (in the last 10 days preceding the screening) use of systemic corticosteroids; use of systemic antibiotics in the last 10 days preceding the screening.

How the Study Was Run

The set-up was randomized, parallel-group and quadruple-blind.

Random assignment meant chance, not the researchers, decided who got L-Carnitine and who got the comparison, which limits bias.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Neither the participants nor the staff measuring them knew who was on L-Carnitine, so expectations could not easily colour the readings.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2012-04 with completion listed for 2013-10.

It ran at 58 sites in Germany (11), Hungary (10), France (9), Poland (9), Spain (7) and Austria (4).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Proportion of clinical/endoscopic remissions (time frame: End of treatment (week 8)).

It also tracked 9 secondary outcomes, for example Change from baseline in Rectal bleeding evaluation, Change from baseline in stool frequency evaluation, Histological response to the treatment, Change from baseline in C-reactive protein (CRP) and Fibrinogen, Haematology and Electrocardiogram.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of L-Carnitine in 150 people.

It ended early (terminated); no results are posted to the registry yet, so nothing here describes outcomes.

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