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

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

List of Studies


Profiles_link
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

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

Date Added
August 25th, 2018
PRO Number
Pro00080391
Researcher
Shannon Hudson

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

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 Save

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

List of Studies

Silhouette
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 Randomised, Double-Blind, Parallel-Group, Multicentre, Phase III Study to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease (HESTIA3) Save

Date Added
July 24th, 2018
PRO Number
Pro00078866
Researcher
Shayla Bergmann

List of Studies

Silhouette
Keywords
Blood Disorders
Summary

This study will assess the effect of ticagrelor versus placebo in reducing the rate of pain crisis in sickle cell disease patients. The study is for male and female subjects between the ages of 2 to 17 who have experienced at least two pain crisis within a 12 month period. The drug is given by mouth in an outpatient setting and consists of 13 visits over an 18 month period.

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

An Open Label Extension Study of GBT440 Administered Orally to Patients with Sickle Cell Disease Who Have Participated in GBT440 Clinical Trials Save

Date Added
April 10th, 2018
PRO Number
Pro00076264
Researcher
Temeia Martin

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

This open label extension study is being conducted to assess the long-term safety and efficacy of GBT440 by providing participants from GBT440-031 continued access to treatment with GBT440 after completion of their participation in GBT440-031 and prior to the product potentially being available commercially. All participants enrolled into this study will receive GBT440.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@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 IIb Randomized, Double-Blind, Placebo-Controlled Multi-Center Study to Assess the Safety, Tolerability, and Efficacy of Riociguat in Patients with Sickle Cell Disease Save

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

List of Studies


Profiles_link
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

A phase 2, Multicenter, Open-Label Study to Assess PK/PD of SEG101 (crizanlizumab), with or without Hydroxyurea/Hydroxycarbamide, in Sickle Cell Patients with Vaso-Occlusive Crisis Save

Date Added
October 24th, 2017
PRO Number
Pro00071740
Researcher
Sarah Mennito

List of Studies


Profiles_link
Keywords
Blood Disorders
Summary

This study will assess the efficacy of crizanlizumab in sickle cell disease patients. The study is for male and female subjects between the ages of 16 to 70 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 consists of 52 visits.

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

A Family-Centered Self-Management Program for Young Children with Sickle Cell Disease: Phase II Save

Date Added
September 7th, 2017
PRO Number
Pro00068250
Researcher
Shannon Hudson

List of Studies


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

This study tests an intervention to help children with sickle cell disease ages 0-7 years and their families. The intervention is available on a smartphone or tablet, and includes an app for keeping track of symptoms and messaging with a health care provider. For more information, please contact Shannon at 843-792-9379.

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

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