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

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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

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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
Julie Kanter washko

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

A Randomized, Placebo-controlled, Phase 2 Study to Evaluate the Safety and Pharmacodynamics of Once-daily Oral IW-1701 in Patients with Stable Sickle Cell Disease Save

Date Added
March 13th, 2018
PRO Number
Pro00075374
Researcher
Julie Kanter washko

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Keywords
Blood Disorders
Summary

This study is for patient with Sickle Cell Disease ages 16 to 70. The purpose of this study is to find out if participants with sickle cell disease have a reduction in the symptoms of their disease. Participants can expected to be in this study for about 12 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 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 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

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

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