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Effect of MitoQ on Platelet Function and Reactive Oxygen Species (ROS) Generation in Patients With Sickle Cell Anemia
MitoQ is commercially available as a dietary supplement and it has been tested as a potential drug in other diseases, but it has never been tested in patients …
Assessing Molecular Mechanisms and Effects of Music Therapy in Youth With Sickle Cell Disease Using Single-cell RNA-sequencing
The goal of this clinical trial is to evaluate whether a 4-week music therapy (MT) intervention can reduce chronic pain and improve psychosocial outcomes in yo…
An Open-label, Multi-centre, Rollover Study to Characterise Long-term Safety and Efficacy of Etavopivat in Adults, Adolescents and Children Who Have Sickle Cell Disease or Thalassaemia and Have Completed a Treatment Period in an Etavopivat Study
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are…
An Evaluation of the Relationship Between Abnormal Myocardial Perfusion and Diastolic Dysfunction in Sickle Cell Disease Using PET (Stress-Rest) Myocardial Perfusion Imaging (MYPERS)
There is limited information on what causes injury to the heart in individuals with Sickle Cell Disease (SCD). Researchers in this study want to see if decreas…
Double-blind, Randomized, Placebo Controlled Study to Evaluate Optimal Timing of Ketamine Initiation
The goal of this clinical trial is to learn if the use of early ketamine decreases the chance of admission to the hospital in patients with sickle cell disease…
Early Identification and Intervention Of Developmental Delay Among Infants And Toddlers With Sickle Cell Disease Using the Sickle Cell Caregiver Collaboration for Child Development (SCCCD) Intervention
Sickle cell disease affects 100,000 people and 2,000 newborns each year; 50% of these children have a developmental deficit (\>2 SD) before the age of 3. Early…
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…
Low-Dose Tocilizumab for Acute Chest Syndrome in Sickle Cell Disease
The investigators are evaluating the role of a low dose of tocilizumab in treating acute chest syndrome in patients with sickle cell disease. Tocilizumab inhib…
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment o…
RCT of Stress Management and Mindfulness Intervention for Patients With Sickle Cell Disease
The purpose of this research is to determine whether a new stress management and mindfulness program can improve quality of life in individuals with sickle cel…
An Adaptive Design Basket Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic materia…
Let's Get REAL: A Pilot Trial of a Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasi…