Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE)- Part 2 Save

Date Added
August 25th, 2018
PRO Number
Pro00080391
Researcher
Julie Kanter washko

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

This study is to better understand barriers to and resources for transcranial Doppler (TCD) screening in children with sickle cell anemia (SCA) ages 2 to 16 years. Surveys and interviews are being conducted with children with SCA and their parents/caregivers, healthcare providers, and other stakeholders to learn about their thoughts and experiences.

Institution
MUSC
Recruitment Contact
Joannie Hayes
843-876-8767
hayesj@musc.edu

The epidemiology of silent and overt strokes in adults with sickle cell disease: a prospective cohort study Save

Date Added
July 20th, 2018
PRO Number
Pro00080248
Researcher
Julie Kanter washko

List of Studies


Profiles_link
Keywords
Blood Disorders, Stroke
Summary

This is a prospective review study which will follow the routine care adult sickle cell patients will receive to be able to determine the risk of recurrent strokes. Participants will be followed for a minimum of 3.5 years.

Institution
MUSC
Recruitment Contact
Emily Warner
843-876-8614
warnere@musc.edu

A Phase 3, Randomized, Double-Blind, Placebo Controlled Study of Voxelotor (GBT440) in Pediatric Participants with Sickle Cell Disease and an Open-label Study in Infants with Sickle Cell Disease (HOPE Kids 2) Save

Date Added
May 8th, 2018
PRO Number
Pro00076380
Researcher
Julie Kanter washko

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

This study will assess the safety and efficacy of voxelotor with long term, daily oral dosing compared to placebo in pediatric participants (ages 9 months to 12 years old) with SCD as measured by improvement in anemia. Participants can expected to be in this study for about 52 weeks with at least 12 visits to the study center.

Institution
MUSC
Recruitment Contact
Joannie Hayes
843-876-8767
hayesj@musc.edu

A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study of IMR-687 in Adult Patients with Sickle Cell Anaemia (Homozygous HbSS or Sickle-?0 Thalassemia) Save

Date Added
January 9th, 2018
PRO Number
Pro00074278
Researcher
Julie Kanter washko

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

This is for patients aged 18 and older who have Sickle Cell Disease. The main purpose of this study is to find out how safe IMR-687 is for treating people with Sickle Cell, and whether it is has any unwanted effects. Participation in this study will last about 8 months.

Institution
MUSC
Recruitment Contact
Emily Warner
843-876-8614
warnere@musc.edu

A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease Save

Date Added
February 28th, 2017
PRO Number
Pro00063476
Researcher
Julie Kanter washko

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

GBT440 is an study drug that has been shown to increase oxygen in the blood. This study is recruiting patients with sickle cell disease to establish efficacy and safety of GBT440 as compared with placebo.

Institution
MUSC
Recruitment Contact
Emily Warner
843-876-8614
warnere@musc.edu

South Carolina Sickle Cell Disease Access to Care Pilot Program (SC) Save

Date Added
July 19th, 2016
PRO Number
Pro00054236
Researcher
Julie Kanter washko

List of Studies


Profiles_link
Keywords
Adolescents, Blood Disorders, Children's Health, Infant
Summary

The purpose of this research protocol is to collect information about individuals living with sickle cell disease to help improve the care of those patients. We hope to understand more about the disease itself, the best ways to treat the disease, and the best ways to help patients with sickle cell disease get care. The goal is to have clinical information about every person diagnosed with sickle cell disease in South Carolina so that we can better treat the disease and help develop new ways to improve outcomes.

Institution
MUSC
Recruitment Contact
Katherine Williams-Turner
843-876-0821
willkat@musc.edu

Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients with Sickle Cell Disease or Other Anemias Save

Date Added
June 23rd, 2015
PRO Number
Pro00045163
Researcher
Julie Kanter washko

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

Ferriprox is an iron chelator that is approved by the FDA of the USA in October of 2011 for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. LA38-EXT is an open-labeled extension trial of LA38-0411 where patients who completed LA38-0411 will be invited to enroll in the extension trial to determine the long-term safety and tolerability of Ferriprox in iron-overloaded patients with sickle cell disease or other anemias.

Institution
MUSC
Recruitment Contact
Emily Warner
843-876-8614
ierardie@musc.edu

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