Vaso-occulsive crisis is a complication of Sickle cell disease in which the red blood cells (RBC) change shape, causing congestion within the blood vessels that leads to pain and tissue damage.
The study medication FT-4202, an oral tablet, is believed to reduce the rate of sickle cell polymerization and improve RBC membrane function, thereby reduction sickling of RBCs and their hemolysis (breakdown of red cells) that causes vascular obstruction and anemia.
This study will consist of a 52-week, randomized (volunteers are selected by chance to receive study either study medication or placebo) , placebo controlled (a placebo is a look-alike pill that contains no active medication). There will be 17 study visits.
The study is followed by a 52-week open label extension study in which all participants will receive study medication. There will be 11 study visits.
This study is intended to provide open-label, early access to voxelotor for pediatric patients with Sickle Cell Disease between the ages of 4 to 11 years old who have no alternative treatment options. Voxelotor is approved by the Food and Drug Administration in patients ages 12 and up with Sickle Cell Disease. Voxelotor has not been approved by the Food and Drug Administration for patients less than 12 years of age. Participants will follow their standard of care schedule and receive supply of medication every 3 months during study period.
Iron deficiency is the most common type of nutritional deficiency in the world and is unique in that it affects both developing and developed countries. The most common complication of iron deficiency is anemia (a low level of iron in the red blood cells). Although patients with anemia may not have any symptoms, many patients with anemia do have problems such as fatigue, tiredness, shortness of breath and/or pale skin.
Study participants will undergo some evaluations in addition to their regular anemia work up. These include the following:
2)Iron absorption test consisting of 3 blood samples over the span of 3 hours after having drank a liquid iron solution.
3) stool and urine sample collection.
4) food diary to monitor iron dietary intake.
5)If an upper endoscopy is also part of the participant's standard of care workup, the study team will ask the endoscopist to take an additional biopsy sample to test for one of the proteins that is responsible for taking up iron from your food into the intestine.
Participation in this study will last over 2 visits lasting 1 to 4 hours each. The two visits should fall within the span of 1 month of each other.
Risks associated with this study include side effects of oral iron supplement ingestion. This oral iron may have a metallic taste. In some patients, it could even cause nausea or vomit, abdominal gas or abdominal discomfort. We also ask to draw blood and blood withdrawal may have side effects including bruising, pain, bleeding or rarely infection at the puncture site. Confidentiality breach is also a risk.
There are no direct benefits to the participant. However, this study will help advance diagnosis and clinical assessments of iron deficiency anemia.
The alternative is to not participate in the study and continue regular iron deficiency anemia work up exclusively with the treating physician and medical team.