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Sickle Cell Thalassemia clinical trials

View clinical trials related to Sickle Cell Thalassemia.

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NCT ID: NCT05675436 Enrolling by invitation - Sickle Cell Anemia Clinical Trials

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

Start date: May 9, 2024
Phase:
Study type: Observational

Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen; this in turn can injure organs including the heart, lungs, and kidneys. SCD can lead to serious illness and death. Treatments such as bone marrow transplants and gene therapies can cure SCD, but they are not widely available. Current drug treatments for SCD are not always effective. This natural history study will examine how a study drug (mitapivat) affects red blood cells in people with SCD. Objective: To learn how mitapivat affects red blood cells in people with SCD. Eligibility: People with SCD who are enrolled in the parent study, NIH protocol IRB001565-H. Design: Procedures for this study will be done during visits already scheduled for the parent study. Participants will have additional blood drawn during study visits. The additional amount will be about 3.5 teaspoons. Participants will undergo a test called near infrared spectroscopy (NIRS) up to 9 times. Probes will be placed on their skin. A blood pressure cuff will be placed on their arm. The cuff will be filled with air for up to 5 minutes and then released. Participants may be asked to breathe at a certain rate or to hold their breath during these measurements. NIRS measures oxygen levels, blood flow, and the makeup of skin and muscle. Researchers will draw additional information for this study from participants medical records.

NCT ID: NCT05414045 Recruiting - Cancer Clinical Trials

Autologous Testicular Tissue Transplantation

Start date: July 29, 2020
Phase: N/A
Study type: Interventional

Freezing testicular tissue of prepubertal boys is a method for preserving spermatogonial stem cells (SSCs) in case of imminent gonadotoxic treatment during childhood. In case of total azoospermia in adulthood and presence of a childwish, the investigators intend to perform the first in men autologous testicular tissue transplantation to restore fertility.

NCT ID: NCT04579926 Completed - Sickle Cell Disease Clinical Trials

PINPOINT: Gaming Technology for SCD Pain

Pinpoint II
Start date: September 19, 2018
Phase: N/A
Study type: Interventional

Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet programs to assist SCD adolescents with better identification and communication about pain are lacking. Research shows that interactive gaming technology can enhance adolescents' learning, and can be especially effective in delivering health-related messages and tools to improve self-care. Pinpoint is an interactive gaming app that will be tested in a Phase II project to determine whether the app assists SCD teens with improving their communication and identification skills for pain self-report.

NCT ID: NCT03376893 Recruiting - Stroke Clinical Trials

Epidemiology of Silent and Overt Strokes in Sickle Cell Disease

ESCD
Start date: June 2, 2017
Phase:
Study type: Observational

Sickle Cell Disease (SCD) is a rare disease occurring in an estimated 100,000 individuals, often poor and underserved, in the US. Silent and overt strokes contribute significantly to morbidity in adults with SCD, resulting in functional impairment, challenges with school and job performance, and premature death. Five NIH-funded randomized controlled trials have identified therapies to prevent silent and overt strokes in children with SCD, including monthly blood transfusion therapy (for preventing initial and recurrent strokes) and hydroxyurea (for preventing initial strokes). Despite the observation that at least 99% of children with SCD in high-income countries reach adulthood, and approximately 60% of adults will experience one or more strokes (~50% with silent strokes and ~10% with overt strokes), no stroke trials have established therapeutic approaches for adults with SCD. For adults with SCD, inadequate evidence-based guidelines exist for secondary stroke prevention strategies. Applying stroke prevention strategies in children may not be effective for stroke prevention in adults with SCD, particularly given the high rate of co-morbidities. Identifying subgroups of adults with SCD and higher incidence coupled with the contribution of established stroke risk factors in the general population (smoking, diabetes, obesity, renal disease) will provide the requisite data required for the first-ever phase III clinical trials focused on secondary stroke prevention in adults.

NCT ID: NCT00736060 Completed - Sickle Cell Anemia Clinical Trials

Clinical and Laboratory Characteristics of Sickle Cell Anemia Patients Admitted With Fever

Start date: May 2008
Phase: N/A
Study type: Observational

This study will summarized the clinical and laboratory data and the outcome of all the patients suffering from Sickle Cell Anemia (Including Sickle cell thalassemia) admitted to the pediatric ward.

NCT ID: NCT00512564 Completed - Sickle Cell Anemia Clinical Trials

Clinical and Laboratory Assessment of Iron Overload in Sickle Cell Anemia and Sickle Cell Thalassemia

Start date: September 2008
Phase: N/A
Study type: Observational

Iron overload is well study in Thalassemia patients and it's not only related to blood transfusions, since intestinal iron absorption is also increased in those patients. Sickle cell patients didn't developed significant clinical symptoms and signs of iron overload in spite frequent transfusions. The purpose of this study is to assess the iron overload in Sickle cell anemia and Sickle cell Thalassemia patients using clinical parameters and laboratory studies including Non Transferrin Binding Iron, Labile Iron and Hepcidin, in order to determine the cardiac and liver iron.

NCT ID: NCT00512226 Completed - Sickle Cell Anemia Clinical Trials

Iron Overload Assesment in Sickle Cell Anemia and Sickle Cell Thalassemia

Start date: September 2007
Phase: N/A
Study type: Interventional

Iron overload is well study in Thalassemia patients and it's not only related to blood transfusions, since intestinal iron absorption is also increased in those patients. Sickle cell patients didn't develope significant clinical symptoms and signs of iron overload in spite frequent transfusions. The purpouse of this study is to assess the iron overload in Sickle cell anemia and Sickle cell Thalassemia patients using clinical parameters and cardiac T2*MRI in order to determine the cardiac and liver iron.

NCT ID: NCT00480974 Completed - Sickle Cell Anemia Clinical Trials

Long Term Follow up in Sickle Cell Patients Treated by Hydroxyurea

Start date: May 2007
Phase: N/A
Study type: Observational

Hydroxyurea was found to be a good treatment in adult patients with sickle cell anemia with significant decrease in the frequency of vaso-occlusive crises and other crises related to SCA. Several studies were published with relative short term follow up in pediatric and young adult age. The purpose of this study is to assess the long term follow up in a group of patients that initiated Hydroxyurea treatment in childhood.