1. A 5-year, Prospective, Non-Interventional, Multicenter Registry in Sickle Cell Disease Patients
A long-term observational study, to collect data on sickle cell disease patients in the United States in order to enhance the understanding of the disease patterns, current transfusion practices, treatments and outcomes in sickle cell disease.
2. A Phase III, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia (Emmaus Medical Inc., Protocol GLUSCC09-01)
Sponsor: Emmaus Medical, Inc.
A study to evaluate the efficacy of oral L-glutamine, as a therapy for sickle cell anemia and sickle bo-thalassemia as evaluated by the number of occurrences of sickle cell crises.
3. Toward a Less Toxic Yet Highly Effective Conditioning Regimen in Allogeneic Hematopoietic Stem Cell Transplantation for Children and Adolescents with Severe Sickle Cell Disease: a Pilot Study
Sponsor: The Friends Research Fund of Children’s Healthcare of Atlanta
A HLA matched related hematopoietic stem cell transplant study for children with sickle cell disease, using a reduced conditioning regimen pre-transplant in order to reduce neurological complications.
4. PFAST: Patent Foramen Ovale and Stroke
Sponsor: Doris Duke Charitable Foundation (DDCF)
A multicenter controlled study to compare the prevalence of patent foramen ovale (PFO) or other potential intracardiac shunts detectible by transthoracic echocardiography (TTE) by conventional 2-D, color Doppler, and agitated saline-contrast studies in children with sickle cell disease and stroke to a control group of patients without sickle cell disease or stroke.
5. A Pilot Study of an Immunosuppressive and Myeloablative Preparative Regimen for Allogeneic Unrelated Donor Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease
Sponsor: Nationwide Children’s Hospital
A pilot study to determine the safety and feasibility of the preparative regimen for hematopoietic stem cell transplantation using a novel immunosuppressive regimen for unrelated donors.
Silent Infarct Transfusion Study (SIT):
A six year study involving 25 sites in the US, Canada, Engaland, and France, which determined that blood transfusions could prevent silent strokes in at-risk patients with sickle cell disease.
Stroke Prevention in Sickle Cell Disease (STOP) Study:
Approximately 3,000 children with sickle cell disease from 14 centers were screened with transcranial Doppler (TCD). A total of 130 were randomized to receive either standard supportive care or periodic blood transfusions if they were found to be at high risk of stroke on the basis of elevated cerebral blood flow as measured by TCD screening tests. The study led to a Clinical Alert, issued by the NHLBI, to recommend screening and consideration of treatment in children with sickle cell disease and 2 to 16 years of age who are at risk based on transcranial Doppler ultrasonography, and who have not had stroke
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