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
Julie Kanter washko

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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
Emily Warner
843-876-8614
warnere@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

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

Date Added
October 24th, 2017
PRO Number
Pro00071740
Researcher
Julie Kanter washko

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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 18 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
Emily Warner
843-876-8614
warnere@musc.edu

Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Save

Date Added
August 30th, 2017
PRO Number
Pro00066053
Researcher
Julie Kanter washko

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Keywords
Transplant
Summary

This is a multi-center, longterm safety and efficacy follow-up study for subjects who have been treated with the LentiGlobin BB305 drug product on HGB-206 sickle cell gene therapy parent study. This study is observational only, and no investigational treatment will be given on this follow-up study. Participants for this study are requested to follow-up every six months for the first three years and then once every year for the remaining Year 4 through Year 13, totaling thirteen years in this longterm study. Study assessments will include exams, laboratory studies, imaging, ECG, surveys, and record review.

Institution
MUSC
Recruitment Contact
Brandi Day
843-792-3379
dayb@musc.edu

Disseminating NIH Evidence Based Sickle Cell Recommendations in North Carolina and South Carolina Save

Date Added
August 22nd, 2017
PRO Number
Pro00064785
Researcher
Julie Kanter washko

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Keywords
Blood Disorders
Summary

The purpose of this project is to utilize implementation science to evaluate patient and system outcomes associated with the dissemination of a toolbox of decision support tools designed based on the NHLBI evidence-based guidelines for treating individuals with sickle cell disease (SCD). These tools will be disseminated to primary care physicians and emergency department providers across NC and SC. The overarching goal is to implore provider education and the implementation of a co-management model of care to improve the efficiency and quality of healthcare for individuals with SCD. The co-management model of care suggests that individuals with SCD benefit from visiting both their primary care clinician and their SCD specialist twice per year.

Institution
MUSC
Recruitment Contact
Ashley Green
843-876-8652
greenan@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

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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

A Study to Compare Bone Marrow Transplantation to Standard Care in Adolescents and Young Adults with Severe Sickle Cell Disease Save

Date Added
December 6th, 2016
PRO Number
Pro00056206
Researcher
Julie Kanter washko

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Keywords
Blood Disorders, Transplant
Summary

This study is meant to compare transplant to standard care (regular care) for sickle cell patients. By comparing the health outcomes for patients who receive bone marrow transplant to those patients who receive standard of care, this study will be able to determine whether the two treatments are the same, or if one is better than the other.

Institution
MUSC
Recruitment Contact
Brandi Day
843-792-3379
dayb@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

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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

AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE SAFETY OF RIVIPANSEL (GMI-1070) IN THE TREATMENT OF ONE OR MORE VASO-OCCLUSIVE CRISES IN HOSPITALIZED SUBJECTS WITH SICKLE CELL DISEASE Save

Date Added
February 23rd, 2016
PRO Number
Pro00052467
Researcher
Julie Kanter washko

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Keywords
Blood Disorders
Summary

The main symptom of sickle cell disease is pain-and it is the main reason for which patients are admitted. This study introduces a new drug, Rivaipansel, which may shorten the duration of this pain (called a "crisis"). This is a phase 3 open label extension research study using the drug Rivipansel in sickle cell patients in the inpatient setting. Patients who consented and participated in the first part of the study (B5201002) may be consented to receive the drug Rivipansel during a subsequent pain crisis to test the efficacy and safety of repeated treatments for pain crises.

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

A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF RIVIPANSEL (GMI-1070) IN THE TREATMENT OF VASO-OCCLUSIVE CRISIS IN HOSPITALIZED SUBJECTS WITH SICKLE CELL DISEASE Save

Date Added
October 13th, 2015
PRO Number
Pro00046192
Researcher
Julie Kanter washko

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Keywords
Blood Disorders
Summary

Phase 3 research study using the drug Rivipansel in sickle cell patient in the inpatient setting to possible lessen their pain crisis.

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

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