sickle-cell-disease Clinical Trials
A listing of sickle-cell-disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 152 clinical trials
Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing …
Dietary Intake and Dietary Behaviors in Adults With Sickle Cell Disease
Background Sickle Cell Disease (SCD) causes blood cells form a crescent shape. It is caused by a genetic mutation in the hemoglobin gene. People with SCD are at increased risk for illnesses like stroke, chronic pain, and heart problems, as well as decreased overall health and well-being. Researchers want to …
A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
Sickle Cell Disease (SCD) impairs oxygen transport to tissue and causes endothelial injury. Thus, therapeutic interventions aim to improve both, but there is an unmet need for biomarkers to determine when intervention is necessary and evaluate the effectiveness of the chosen intervention in individual patients. This study proposes to monitor …
Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation
The purpose of this research study is to document and understand the effects of hydroxyurea exposure for women with SCD and their babies, during both gestation and lactation.
Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle Cell Disease
The goal of this observational study is to help us understand more about the best ways to help individuals living with Sickle Cell Disease (SCD) get the best care. The main question it aims to answer is: How to find individuals unaffiliated from SCD specialist care use three distinct pathways? …
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD). The objective is to assess the safety profile and clinical effectiveness of Xromi® under routine clinical conditions. The …
Study to Understand the Genetic Risk of Developing an Immune Response After Blood Transfusions Among Individuals With Sickle Cell Disease
The purpose of this research study is to look at genes and determine how they interact with each other to find changes that could explain why some people's immune systems may respond to blood transfusions. This response is called an alloimmune response. We strongly believe that when someone has an …
Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease
This study will examine sleep disordered breathing and sleep quality in participants (ages 12-18) diagnosed with sickle cell disease of any genotype. We will utilize remote peripheral arterial tonometry (PAT) and questionnaires to evaluate difficulties with sleep. PAT assessments will occur remotely in the homes of participants. Neurocognitive, behavioral, and …
Serial Assessment of Fertility Experiences
The SAFE study is a long-term research project that watches people with sickle cell anemia (SCA) over time. The main goal is to see how a medicine called hydroxyurea affects their growth, puberty, and ability to have children. A second goal is to see how hydroxyurea affects pregnancy outcomes, by …
Lung Ultrasound in Pediatric Acute Chest Syndrome
Sickle-cell disease is a common disease with serious complications, in particular acute chest syndrome (ACS), which can be life threatening. The pathophysiology of ACS is poorly understood, but alveolar hypoventilation appears to play an important role. Pulmonary ultrasound is increasingly used in pediatrics to diagnose ACS. The management of ACS …
