An Open-label, Expanded Access Protocol for Pediatric Patients with Sickle Cell Disease Who Have No Alternative Treatment Options

Date Added
April 13th, 2021
PRO Number
Pro00107737
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

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.

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

A PHASE 3B, PROSPECTIVE, OPEN-LABEL, UNCONTROLLED, MULTICENTER STUDY ON LONG-TERM SAFETY AND EFFICACY OF rVWF IN PEDIATRIC AND ADULT SUBJECTS WITH SEVERE VON WILLEBRAND DISEASE (VWD)

Date Added
October 27th, 2020
PRO Number
Pro00099367
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

This study will evaluate if Food and Drug Administration (FDA) approved recombinant von Willebrand Factor (rVWF) is safe and effective with long term use in adult and pediatric/adolescent patients with von Willebrand Disease (VWD). Dosing is an optional prophylactic (preventative) dosing on a weekly basis, or on demand (OD) for bleeding episodes and in the management of surgical bleeding. This study may last up to 3 years. You will have clinic visits in 3 month intervals during this time.

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

Down syndrome biomarker project

Date Added
June 25th, 2020
PRO Number
Pro00090922
Researcher
Eric Hamlett

List of Studies


Keywords
Alzheimers, Blood Disorders, Brain, Children's Health, Dementia, Inflammation, Vascular
Summary

Alzheimer's disease and Epilepsy may affect over 80% of individuals that have Down syndrome by the age of 60. Biomarkers found in the blood can enhance our understanding of the earliest changes linked to disease and may enhance clinical detection and healthy aging for individuals with Down syndrome.

The purpose of this study is to discover early neurobiological processes underlying the transition from healthy aging to disease. Our research team has developed technology that allows detection of small changes in the brain that get transferred to the blood.

We are recruiting individuals that either have or do not have Down syndrome for this biomarker study. Participants should be between the ages of 6 months and 85 years old and may include mothers and siblings of a child with Down syndrome. Infants and children will require consent form a parental or legal guardian.

Each participant will provide a blood sample for research purposes. We will also gather some basic health information about senses, habits, exercise level and smoking/vaping exposures.

Institution
MUSC
Recruitment Contact
Eric Hamlett
919 357 7543
hamlette@musc.edu

Treatment of Drug-resistant Adult and Pediatric Primary Focal Segmental Glomerulosclerosis Using the Liposorber® LA-15 System

Date Added
November 26th, 2019
PRO Number
Pro00089025
Researcher
Milos Budisavljevic

List of Studies


Keywords
Blood Disorders, Kidney
Summary

A device called the "Liposorber LA-15 System" has been approved by the
United States Food and Drug Administration for treating kids with focal
segmental glomerulosclerosis (FSGS). The "Liposorber LA-15 System" can only be used if other treatment options, like drugs, don't work or can't be used,
but the kidneys are still working okay. It can also be used if the subject
has had a kidney transplant and the FSGS comes back after the
transplant. Although the Liposorber System can be used for FSGS, we
are not sure how well the Liposorber System works. So, we are doing this
study to find out how well the treatment works in adults.
In this research study, there will be up to 5 adults who have FSGS
enrolled at MUSC. Subjects will come back for up to 12 treatments over 9
weeks and then 5 visits to their study doctor over the next 2 years.

Institution
MUSC
Recruitment Contact
Linda Walker
843-792-6109
walkerlp@musc.edu

A phase 2, Multicenter, Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide, in Sequential, Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vaso-Occlusive Crisis

Date Added
August 14th, 2018
PRO Number
Pro00079784
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

This study will assess the appropriate dosing and evaluate the safety of crizanlizumab in pediatric sickle cell disease patients. The study is for male and female subjects between the ages of 6 months to 17 years old who have experienced at least one pain crisis within a 12 month period. The drug is given via an IV infusion in an outpatient setting and has the potential to reduce the amount of sickle cell pain crisis a participant may experience. Participants can expected to participant in this study for up to 2 years.

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

Treatment of Drug-resistant Adult and Pediatric Primary Focal Segmental Glomerulosclerosis Using the LIPOSORBER® LA-15 System

Date Added
January 10th, 2017
PRO Number
Pro00059528
Researcher
Katherine Twombley

List of Studies


Keywords
Blood Disorders, Kidney, Pediatrics
Summary

A device called the "Liposorber LA-15 System" has been approved by the United States Food and Drug Administration for treating kids with focal segmental
glomerulosclerosis (FSGS). The "Liposorber LA-15 System" can only be used if other treatment options, like drugs, don't work or can't be used, but the kidneys are still working okay. It can also be used if the subject has had a kidney transplant and the FSGS comes back after the transplant. Although the Liposorber System can be used for FSGS, we are not sure how well the Liposorber System works. So, we are doing this study to find out how well the treatment works.

In this research study, there will be up to 5 children who have FSGS enrolled at MUSC. Subjects will come back for up to 12 treatments over 9 weeks and then 5 visits to their study doctor over the next 2 years.

Institution
MUSC
Recruitment Contact
Katherine Twombley
843-792-8904
twombley@musc.edu



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