A Phase 1/2 randomized, double-blind, placebo-controlled, multicenter, ascending dose, safety and Pharmacokinetic/Pharmacodynamic PK/PD study of SHP655 (rADAMTS13) in sickle cell disease at baseline health and during acute vaso-occlusive crisis.

Date Added
August 25th, 2020
PRO Number
Pro00101087
Researcher
Shayla Bergmann

List of Studies

Keywords
Blood Disorders
Summary

Sickle cell disease (SCD) is a inherited disease that can cause sudden, severe pain. The management of this pain is accomplished through analgesic medications. This study for for male and female subjects between the ages of 18 and 65 years. This study will assess the appropriate dose and the evaluate the safety of SHP 655 in SCD patients at a baseline health state which is Part A of this study. The study medication is given by infusion as a single dose. SHP 655 is believed to increase blood flow and decrease blood cells from being trapped in low blood flow areas such as joints which can lead to tissue death.

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

INTEGRATION OF mHEALTH INTO THE CARE OF PATIENTS WITH SICKLE CELL DISEASE TO INCREASE HYDROXYUREA UTILIZATION – mESH study

Date Added
July 23rd, 2020
PRO Number
Pro00097832
Researcher
Cathy Melvin

List of Studies

Keywords
Blood Disorders, Stroke, Stroke Recovery
Summary

The purpose of this research project is to evaluate the effectiveness of a mobile health (mHealth) application in improving adherence to hydroxyurea therapy in patients with Sickle Cell Disease (SCD). Participants will be asked to install an application on their phone that will remind them to take their medication regularly. It notifies the participants when it is time to request a refill, it tracks their hydroxyurea use, and it gives them information on their medication. It also has resources that could be helpful for the ongoing care of their sickle cell disease. This is a 24-week project with 3 study visits. The first study visit will be at the beginning of the study period (enrollment or baseline visit), the second will be at approximately 12 weeks, and the third is at the end of the 24 weeks. At each visit, participants will complete a survey, share with us their experience with the application, and share with us where they refilled their medications. Some participants will also be asked to complete an interview at the end of their final study visit. Patient participants will receive a $25 Walmart Gift card at each of visits (including an additional $25 gift card for the optional interview) for their participation.

Institution
MUSC
Recruitment Contact
Sarah Bourne
8433452545
bourne@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

ATHN 9: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Severe Von Willebrand Disease (VWD)

Date Added
January 27th, 2020
PRO Number
Pro00092790
Researcher
Stephanie Ambrose

List of Studies

Keywords
Adolescents, Blood Disorders, Children's Health, Circulation, Dental, Genetics, Infant, Joint, Men's Health, Minorities, Muscle, Non-interventional, Nose, Obstetrics and Gynecology, Pain, Pediatrics, Periodontal Disease, Rare Diseases, Rheumatoid, Surgery, Women's Health
Summary

ATHN 9 is a natural history study to assess the safety of various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD.

Institution
Palmetto
Recruitment Contact
Shannon Cearley
803-434-4088
shannon.cearley@prismahealth.org

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 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Date Added
September 24th, 2019
PRO Number
Pro00091452
Researcher
Charles Greenberg

List of Studies

Keywords
Blood Disorders
Summary

The purpose of this study is to test the safety and effectiveness of a new study drug in adults who have warm antibody autoimmune hemolytic anemia (wAIHA). This study aims to determine if participants receiving the study drug, called fostamatinib, respond better than participants receiving placebo (an inactive substance) measured by an increase in red blood cell count. Participation in the study will be for a duration of at least 30 weeks with a total of approximately 15 visits to the study clinic.

Institution
MUSC
Recruitment Contact
Ormarie Vazquez Silva
843-792-7333
vazquezo@musc.edu

An Open-Label, Randomised, Active Controlled, Multi-Centre Phase 3 Study to Evaluate the Safety and Efficacy of Danaparoid vs Argatroban in Treatment of Subjects with Acute HIT (HITSOVA study)

Date Added
August 27th, 2019
PRO Number
Pro00091006
Researcher
Charles Greenberg

List of Studies

Keywords
Blood Disorders, Drug Studies
Summary

The purpose of this study is to evaluate the likely safety and effectiveness of danaparoid sodium versus argatroban for patients suffering from heparin-induced thrombocytopenia (HIT). Patients with HIT will have low blood platelet counts due to being given the medication heparin. Adult and pediatric participants who are determined to be eligible to participate will be assigned by chance to treatment with danaparoid sodium or argatroban. The study drugs are administered through intravenous therapy (IV). Study and safety assessments, including blood draws and compression ultrasounds, will be completed at study visits. Participants will be in this study for at least 45 days, but could receive treatment longer depending on HIT severity. You will be hospitalized while you are receiving study drug treatment for a minimum fo 14 days. You will have 4 study follow-up visits after they have been discharged from the hospital.

Institution
MUSC
Recruitment Contact
Reshil-Marie Dukes
843-792-0387
dukesre@musc.edu

SMYLS: A Self-Management Program for Youth Living with Sickle Cell Disease

Date Added
March 17th, 2019
PRO Number
Pro00084400
Researcher
Shannon Phillips

List of Studies

Keywords
Adolescents, Blood Disorders, Pain, Pediatrics
Summary

The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of their sickle cell disease. The intervention lasts 12 weeks with a 3-month follow up period, and uses a smartphone or a tablet. For more information, please contact Shannon at 843-792-9379.

Institution
MUSC
Recruitment Contact
Shannon Phillips
843-792-9379
phillipss@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

A Phase IIb Randomized, Double-Blind, Placebo-Controlled Multi-Center Study to Assess the Safety, Tolerability, and Efficacy of Riociguat in Patients with Sickle Cell Disease

Date Added
January 9th, 2018
PRO Number
Pro00074247
Researcher
Temeia Martin

List of Studies

Keywords
Blood Disorders
Summary

This study is for patients 18 years and older who have Sickle Cell Disease. The study will be evaluating the safety and tolerability of treatment with riociguat to potentially improvement in blood pressure, blood circulation, and disease symptoms. Participants can expect to be in this study for 20 weeks.

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



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