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Research

Dr. Kanter visiting patient

Our Research Team

Emily Warner: Sickle Cell Research Program Manager
Cheryl Alston: Research Program Coordinator
Brandi Day: Research Program Coordinator 
Karen Hawkins: Research Nurse
Joannie Hayes: Research Program Coordinator
Gary Link: (SC)2 Human Service Coordinator
Sarah Moderhack: (SC)² Data Coordinator
Jkeonye Moss: Sickle Cell State Coordinator

Clinical Trials

For more information or to see if you are eligible for a clinical trial today contact Emily Warner at 843-876-8614 or sicklestrong@musc.edu.

  • CALYPSO trial:  Phase II randomized study to evaluate treatment compliance, efficacy and safety of an improved Deferasirox formulation (granules) in pediatric patients with sickle cell patients and iron overload
  • FIRST trial:  Phase III randomized study to evaluate the use of a new iron chelator (Ferriprox) for patient ages 2 years and older with sickle cell disease and iron overload.
  • LA38-EXT trial: Is the open labeled extension of FIRST which guarantees subjects who participated in FIRST will receive Ferriprox to treat their iron overload.
  • HOPE trial: Phase III randomized study to assess the efficacy of a new drug, GBT440 in adolescents and adults with sickle cell disease as measured by improvement in anemia.
  • HGB206 trial:  Phase I gene therapy and autologous transplant study with Lentiglobin BB305 Lentiviral Vector in subjects with severe sickle cell disease.
  • LTF-303: The Longterm Follow up of Sickle Cell Gene Therapy trial HGB206. Subjects enrolled in the HGB206 trial will be followed for an additional 13 years.
  • RESET trial:  Phase III randomized study for children and adults to evaluate the use of a new IV medication (Rivipansel) to decrease pain and hospitalization in patients 6 years and older with sickle cell disease.
  • RESET-EXT trial:  Is the open labeled extension of RESET which guarantees subjects who participated in RESET will be able to receive the study drug during future hospitalizations.
  • STRIDE:  A bone marrow transplant study to evaluate outcomes in patients with sickle cell disease undergoing bone marrow transplant compared to standard of care.
  • STAR:  Multi-Center Retrospective Registry of Children with Sickle Cell Disease following Hematopoietic Cell Transplantation: A Sickle Transplant Alliance for Research (STAR) Project.
  • SCOT trial:  Phase 2 randomized study evaluating the safety, effectiveness and to find the optimal dose of a new drug called SC411 in pediatric patients ages 5 to 17. SC 411 may help prevent sickle cell crises, decrease inflammation and continuous breakdown of red blood cells.
  • IMARA: Phase II randomized study to evaluate the safety of a new drug IMR-687 in patients 18 and older with sickle cell disease. IMR-687 is thought to increase the production of HgbF and help reduce the complications of sickle cell disease.
  • SEG101- Phase II open-labled study to evaluate the efficacy of crizablizumab in patients 18 and older with sickle cell disease. Crizablizumab is a new IV medication thought to help reduce the rate of pain crisis.
  • STERIO- Phase II randomized study to assess the safety, tolerability and efficacy of Riociguat in patients 18 and older with sickle cell disease. Riociguat is an FDA approved drug in the treatment of pulmonary hypertension and thought to help reduce the frequency of sickle cell related painful crisis.

Departmental & Interdisciplinary Research Resources

South Carolina Clinical and Translational Research Institute (SCTR)
The Medical University of South Carolina established the South Carolina Clinical and Translational Research Institute(SCTR) in 2006 in response to the NIH Clinical and Translational Science Award (CTSA) Program, aimed at transforming approaches to research and discovery implementation. The main thrust of the CTSA Program is to catalyze the development of interdisciplinary research initiatives to accelerate the translation of discoveries into improved therapies and clinical practice while breaking down programmatic boundaries. MUSC received NIH funding for the SCTR in July 2009; the present application requests renewed support for the next five year period. Multiple cores currently operate under the SCTR umbrella, including:

  • Biomedical Informatics
  • Community Engagement
  • Biostatistics & Epidemiology
  • Novel Clinical & Translational Methodologies
  • Regulatory Knowledge and Support
  • Research Ethics
  • SUCCESS Center & Research Toolkit
  • Training and Education
  • Translational Technologies & Resources