Hemochromatosis Clinical Trials 2023

What Are Hemochromatosis Clinical Trials?

Hemochromatosis is a disorder that affects the body’s ability to absorb iron from food is impacted, causing it to take up too much iron and resulting in an iron overload. The excess iron begins building up in the organs, especially the heart, liver, and pancreas. Hemochromatosis leads to the onset of various disorders and diseases and can cause organ damage.

Hemochromatosis clinical trials are research studies involving patients suffering from the disorder. These studies allow health experts and scientists to enhance their understanding of the disorder, leading to improved diagnostic and treatment methods.

Why Is Hemochromatosis Being Studied Through Clinical Trials?

Hemochromatosis affects one in every three to five hundred individuals worldwide. It is more common in men but can affect people of all ages. Causes of development of the disorder include anemia, iron supplements, blood transfusions, hepatitis C infection, liver disease, fatty liver disease, and kidney dialysis. However, it is most commonly caused due to hereditary reasons, where parents pass the faulty gene to the children.

It is a severe condition requiring immediate diagnosis and treatment. Symptoms of the disorder include unintended weight loss, joint pain, weakness, fatigue, ED in men, and irregular periods in women. If undiagnosed and untreated, it can lead to heart failure, cirrhosis, enlarged liver, liver failure, heart failure, arthritis, liver cancer, and diabetes. About 2 out of every 10,000 patients of hemochromatosis lose their life due to symptoms and disorders caused by the condition.

Therefore, hemochromatosis is studied through clinical trials to develop improved treatment options to save and improve patients' lives. Health experts also seek to develop preventative treatments for those at high risk of developing the disease.

What Are the Types of Treatments Available for Hemochromatosis?

Unfortunately, there is no definite cure for hemochromatosis. However, various treatment options allow for lowering the amount of iron in the blood.

The most common treatment for hemochromatosis is phlebotomy, i.e., the removal of blood through veins. It is no different than donating blood and is generally considered painless. The treatment is performed weekly when the disorder is first diagnosed. After a year or so, it is only required up to four times a year. Patients with thin veins who cannot regularly undergo phlebotomy can be prescribed deferasirox. The drug is used for chelation therapy, i.e., it encourages the body to absorb excess iron in the urine and feces so it can be released from the body.

No matter the route for releasing excess iron from the body, the treatment also requires strict dietary changes. This includes avoiding alcohol, fortified foods, iron supplements, vitamin C supplements, and raw seafood. Patients are also advised to intake a healthy and balanced diet.

What Are Some Recent Breakthrough Clinical Trials for Hemochromatosis?

2023: La Jolla Pharmaceutical Company, USA, ran a clinical trial testing the LJPC-401 to lower iron uptake by adult hemochromatosis patients' bodies. The study involved 70 participants whose blood was measured for iron amounts regularly during the trial. The drug showed hopeful results, but further trials are needed.

2020: The Stanford School of Medicine, USA, ran a clinical trial testing the drug ebselen. The drug was found successful in halting iron from entering human heart cells. The trial team hopes to get it FDA-approved so that hemochromatosis patients who die of heart failure can be saved.

Who Some Key Opinion Leaders/Researchers Are in Hemochromatosis Clinical Trial Research?

The Hemochromatosis UK and the American Hemochromatosis Society are the leaders in hemochromatosis clinical trials and research.