Clinical trials can be extremely beneficial in learning more about various diseases, as well as the safety, efficacy, and tolerability of potential treatments. According to a news release from global pharmaceutical company Vifor Pharma (“Vifor”), the first patient was enrolled in the Phase 2a SCD-202 clinical trial. During the trial, researchers will evaluate Vamifeport (VIT-2763) for patients with sickle cell disease (SCD).
Vamifeport (VIT-2763) is a ferroportin inhibitor, which is administered orally. Normally, ferroportin plays a role in iron transport and acquisition within the body. However, in patients with sickle cell disease, too much iron could enter into the bloodstream. Vamifeport stops this from occurring and, in turn, stops the formation of too much abnormal hemoglobin. Researchers believe this could prevent some of the inflammation and red blood cell destruction often seen in SCD. Outside of SCD, researchers are also evaluating Vamifeport as a potential therapy for patients with beta-thalassemia. So far, this therapy has received Orphan Drug designations in both Europe and the United States.
In the burgeoning SCD-202 trial, researchers will evaluate Vamifeport for adults with SCD. Altogether, 25 patients will enroll. The study aims to focus on whether the treatment reduces incidences of hemolysis (red blood cell destruction).
About Sickle Cell Disease (SCD)
Sickle cell disease (SCD) comprises of a group of inherited red blood cell disorders. Normally, hemoglobin helps carry oxygen throughout the body. In patients with SCD, gene mutations create abnormal hemoglobin. As a result, they develop malformed, sickle-shaped red blood cells which can cause blockages or restrict blood flow. SCD is inherited in an autosomal recessive pattern, meaning patients must receive one defective gene from each parent. Those of African American descent have a higher risk of developing SCD. Symptoms associated with SCD include:
- Anemia (low red blood cell count)
- Jaundice (yellowing of the skin, eyes, and mucous membranes)
- Pain crisis
- Swelling of the hands and feet
- Increased risk of infections
- Delayed growth
- Organ damage (including eyes, spleen, liver, kidneys, brain, heart, lungs, and more)