View clinical trials related to Sickle Cell Disease.
Filter by:Sickle cell disease (SCD) refers to a group of hemoglobinopathies that include mutations in the gene encoding the beta subunit of hemoglobin. Within the umbrella of SCD, many subgroups exist, namely sickle cell anemia (SCA), hemoglobin SC disease (HbSC), and hemoglobin sickle-beta-thalassemia (beta-thalassemia positive or beta-thalassemia negative). Several other minor variants within the group of SCDs also, albeit not as common as the varieties mentioned above. It is essential to mention the sickle cell trait (HbAS), which carries a heterozygous mutation and seldom presents clinical signs or symptoms. Sickle cell anemia is the most common form of SCD
CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy.
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnosis for single-gene disorders (SGD-NIPD). This can be performed from 9 weeks of amenorrhea and offers an early, safe and accurate definitive diagnosis without the miscarriage risk associated with invasive procedures. One of the major difficulties is distinguishing fetal genotype in the high background of maternal cfDNA, which leads to several technical and analytical challenges. Besides, unlike noninvasive prenatal testing for aneuploidy, NIPD for monogenic diseases represent a smaller market opportunity, and many cases must be provided on a bespoke, patient- or disease-specific basis. As a result, implementation of SGD-NIPD remained sparse, with most testing being delivered in a research setting. The present project aims to take advantage of the unique French collaborative network to make SGD-NIPD possible for theoretically any monogenic disorder and any family.
Background: Sickle cell disease (SCD) is a genetic disorder where red blood cells, that carry oxygen, are stiff and become stuck in small blood vessels. As a result, affected patients can experience severe pain and serious organ damage. SCD can be cured with a hematopoietic cell transplant (HCT), that is, when they receive blood stem cells from a family donor. But HCT can also have serious side effects, especially in people with organ damage. Researchers want to find ways to make HCT safer for everyone. Objective: To test a new combination of drugs (briquilimab, abatacept, and alemtuzumab), used along with radiation, in people undergoing HCT for SCD. Eligibility: People aged 16 and older with SCD. They must be eligible for HCT and have a family member who is a good donor match. Donors must be aged 4 and older. Design: Participants with SCD will be screened. They will have blood tests and tests of organs including their heart and lung function. Donors will have blood drawn. Participants with SCD will have a tube inserted into a blood vessel in their chest (intravenously). This line will remain in place up to 2 months; it will be used to draw blood and administer the donor cells and other medications. Briquilimab will be administered intravenously 1 time, along with other drugs used to prepare for HCT. Participants will receive abatacept 6 times, from just before they receive their donor cells until 6 months after. Participants will undergo radiation therapy and take other drugs that are standard for HCT. Most HCT recipients remain in the hospital for about 30 days after HCT. Follow-up visits will continue for 5 years....
Sickle cell disease is a painful inherited disorder that affects approximately 100,000 people in the United States, and more than half of these individuals develop chronic or persistent pain that is often severe and disabling. The factors that predict whether an individual with sickle cell disease will develop severe, disabling pain are unclear. The goal of this project is to identify the factors that predict severe pain outcomes in individuals living with sickle cell disease in order to improve pain management strategies and guide future studies of non-opioid therapies for treatment of their pain. Participants who agree to enroll in this study will be asked to participate in a virtual and then an in-person study visit for their full initial study assessment. They will answer survey questions during the virtual visit, and will be asked to complete several types of standard testing to understand how their body handles pain during the in-person visit. After completing the virtual and in-person sessions, participants will receive text or electronic medical record messages with brief survey (will take less than 8 minutes to complete) on their pain experiences every three months until the study is completed (or up to 48 months for people who are enrolled at the beginning of the study).
This study develops and tests the feasibility and acceptability of an adapted intervention, Integrative Strong Body and Mind Training (I-STRONG), in adolescents with pain from sickle cell disease.
The Sickle Cell Children's Exercise Study (SuCCESs) will explore the feasibility and effects of a moderate intensity strengthening, balance, speed, and agility intervention program in children with sickle cell disease.
Psychological distress (anxiety and depression) is common in and experienced differently by people living with long-term health conditions (LTCs). Being able to measure whether psychological distress is related to living with a LTC would allow researchers and clinicians to provide interventions specifically tailored to the challenges of living with a LTC and therefore provide the most appropriate support for these patients. Such a measure would also be useful in research to identify the presence of illness-related distress in different patient groups. This project will therefore create a new measure of illness-related distress that has applications for both research and clinical practice. This will involve the psychometric validation of the new illness-related distress measure to test how valid and reliable the measure is. The aim of the project is to provide initial validation of the Illness Related Distress Scale in a community sample, recruited through online platforms. The objective of the study is to gather initial validity and reliability data for the scale.
The purpose of this study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms.
Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels. There have been numerous studies examining the clinical impact of voxelotor in sickle cell disease (SCD) patients, but there are few published reports on the effects of treatment on physical function in patients with SCD. The hypothesis to be tested is that anemic SCD patients will have improvements in performance after 6 months of voxelotor treatment.