Hydoxycarbamide and L-Carnitine Therapy in Sickle Cell Anemia

What Was Tested

In this phase 4 trial, researchers set out to test L-Carnitine for anemia.

It had four arms (groups): Hydra+L-Carnitine (experimental), Hydra only (active comparator), L-Carnitine only (active comparator) and Supportive measures (no intervention).

Hydroxycarbamide+ L-Carnitine+supportive treatment.

Hydroxycarbamide+ supportive treatment.

L-Carnitine+ supportive treatment.

The registry classes the interventions as drug.

More on the Plan

Sickle cell disease (SCD) is a common monogenic disorder affecting over 100,000 people in the United States alone, and millions more worldwide.

Lives of patients with SCD are characterized by frequent episodes of severe pain (vaso-occlusive events or "crises"); acute organ dysfunction, including a pneumonia-like syndrome termed acute chest syndrome, and strokes starting in childhood; and progressive multi-organ damage.

Not surprisingly, patients with SCD have very high health care utilization (over $1 billion/year in healthcare costs in the United States alone, and a median life-expectancy of only \~45-58 years, compared to the life expectancy of 78.2 years overall in the United States.

HU therapy is offered if the patient does not want to have (more) children, and is weighed against any severe impairment of liver or kidney function, or blood cytopenia.

Who Took Part

91 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

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

Examples of who could join: Patients with sickle cell disease. Not welling for pregnancy in females or to father a baby in males. Non-compliance to transfusion.

The full record lists 4 inclusion and 4 exclusion criteria; among the exclusions: \<18 years; hypersensitivity to hydroxycarbamide or l-carnitine; other chronic infection or inflammation.

How the Study Was Run

It was run as a non-randomized, parallel-group and open-label study.

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

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.

It started in 2017-01-10 with completion listed for 2020-06-10.

It ran at 1 site in Egypt.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Hematological(HR) (time frame: 2-3 months). Hematological response (time frame: 2-3 months).

It also tracked 1 secondary outcome, for example Frequency of painful episodes/ blood transfusions.

What This Means in Plain Language

Put simply, this is a phase 4, non-randomized trial of L-Carnitine in 91 people with anemia.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the L-Carnitine compound page, Anemia research.