A Phase 3b, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Treatment Effect of Voxelotor on Neurocognitive Function in Pediatric Participants 8 to < 18 Years of Age with Sickle Cell Disease

Date Added
May 10th, 2022
PRO Number
Pro00118230
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

It has been established that individual's with Sickle cell disease (SCD) have an increased risk of mental changes (cognitive impairment) related to neurological damage such as a stroke (cerebral vascular accident) or decreased vascular blood flow in the brain decreases oxygenation (hypoxia) in the blood stream related to decreased blood counts and severe anemia.

Voxelotor is an oral medication taken once a day, that was approved by the Food and Drug administration (FDA) in 2019 for patients with SCD ages 12 years and older to improve patients' hemoglobin and the delivery of oxygen to the brain.

This study is designed to assess patient neurocognitive (ability to concentrate, remember things, process information, learn, speak, and understand) functions by having subjects answer questionnaires at the week 1 and end of treatment visits. The study will have 5 visits and last approximately 4 1/2 months,

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

A Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Depemokimab in Adults with Hypereosinophilic Syndrome (HES)

Date Added
May 3rd, 2022
PRO Number
Pro00119532
Researcher
Kelli Williams

List of Studies


Keywords
Blood Disorders, Rare Diseases
Summary

The purpose of this study is to see if taking depemokimab is safe and effective in treating Hypereosinophilic syndrome (HES) in adults (≥18 years) with uncontrolled HES receiving standard of care (SoC) therapy. The study will last approximately 52 weeks and is a placebo-controlled, double blind, multicentre study.

Institution
MUSC
Recruitment Contact
Natalie Naylon
843-792-5824
naylon@musc.edu

A Phase 1, Multi-Center, Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CSL889 in Adult Patients with Stable Sickle Cell Disease

Date Added
November 9th, 2021
PRO Number
Pro00112906
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders, Rare Diseases
Summary

This study is testing an IV infusion medication to treat painful pain episodes, called vaso-occulsive crises, in participants with Sickle Cell Disease. Participants will have a screening visit followed by a 24-hour inpatient hospitalization visit to receive the IV medication and then 8 follow up visits. The purpose of this study is to learn more about how a new potential drug for treating SCD symptoms affects the body and how the body processes it.

Institution
MUSC
Recruitment Contact
Linda Wozniak
843-876-8651
wozniakl@musc.edu

An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE)

Date Added
July 13th, 2021
PRO Number
Pro00107924
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

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.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

Malabsorption as a Cause of Iron Deficiency Anemia

Date Added
November 17th, 2020
PRO Number
Pro00096495
Researcher
Sara Hatoum

List of Studies

Keywords
Blood Disorders, Nutrition
Summary

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:
1) Questionnaires;
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.

Institution
MUSC
Recruitment Contact
Halim Bou Daher
(843) 792-0650
boudaher@musc.edu