View clinical trials related to Sickle Cell Disease.
Filter by:This study is design to assess the effects of an increase in nutritional intake on the bone mineral density of children with sickle cell disease, for 12 months.
The objective of this Expanded Access Protocol (EAP) is to provide voxelotor for the treatment of sickle cell disease (SCD): The study GBT440-041 is the EAP for pediatric patients aged 6 months to 11 years who have no alternative treatment options and are ineligible to participate in a clinical trial of voxelotor and the study C5341057 is the EAP for adults/adolescents 12 years of age and older for patients who cannot satisfactorily be treated with an authorized medicinal product
As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed. ESCORT-HU Extension study aims at evaluating the long-term safety of Siklos® focusing on some questions regarding its safety when used in current practice in adults and paediatric patients treated with Siklos® and followed for up to 5 years. The study will focus on the following concerns : occurrence and incidence of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®.
Vitamin D deficiency may be under-diagnosed in sickle cell disease French children. Therefore, the investigator need an epidemiologic study about the prevalence of vitamin D deficiency in this specific population. There are not specific guidelines neither testing nor treatment. The investigator propose to test vitamin D status in all children with sickle cell disease who are consulting their referring haematologist doctor or in the emergency ward.
The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). To meet this objective, the researchers will conduct a prospective cohort study to determine the longitudinal relationship between HU adherence and health-related quality of life (HRQOL) overtime among AYA with SCD. The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.
The primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized controlled trial to test the feasibility and acceptability of MED-Go app in adolescents and young adults (AYA) with sickle cell disease (SCD). The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.
L-glutamine has been approved in the US to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients 5 years of age and older. The purpose of this single-center, open-label, phase 4 study is to evaluate the pharmacokinetic characteristics and safety of L-glutamine in patients with SCD.
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among adolescents and young adults with sickle cell disease (SCD). In this proposed study, we intend to test the functionality of a PROs-toolbox feature, which will be integrated into our existing smartphone application platform (SCD-app), over a 24-week period in a cohort of SCD patients and their caregivers.
The objective of this project is to determine the prevalence of hypertension, hyperlipidemia and hyperglycemia in the pediatric population with sickle cell disease who are obese in Mississippi compared to those pediatric patients with sickle cell disease who are not overweight/obese. The pediatric hematology department at the University of Mississippi Medical Center (UMMC) has a relatively large population of patients with sickle cell disease who are overweight and obese. This is a paradoxical trend since high-energy expenditure of the body to produce new red blood cells usually results in underweight to normal weight patients. From our previous chart review, the investigators found our pediatric patients with sickle cell disease to have similar rates of overweight and obesity to that of state and national levels. The metrics our team will measure include: blood pressure, blood cholesterol levels and blood glucose levels. The investigators expect to find higher rates of hypertension, high cholesterol and high glucose levels in the overweight and obese patients with SCD compared to that of underweight and normal weight. Our ultimate goal for follow up projects will be to determine the baseline risk of hypertension, hyperlipidemia and hyperglycemia in this population so we can then determine effective, sustainable interventions for weight and the co-morbidities that come with increasing weight status. Our goal would also be to educate the patient and families on these interventions and provide them with resources, which could lead to an overall improvement in health and patients quality of life.
This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality of life (HRQOL) most important to them, (2) frequency at which they are willing to complete them, and (3) other procedures related to the use, uptake and effect of the HU-Go app as a tool to improve hydroxyurea (HU) adherence. Second, this study seeks to utilize novel modern mobile technology using a multi-functional personalized platform to improve adherence to HU and measure HRQOL in youth with SCD, using NIH-endorsed PROMIS® measures, based on a conceptual model with predefined behavioral targets and mediators. Third, we plan to assess HRQOL changes and identify modifiable behavioral strategies that could serve as surrogates or predictors for HU adherence. This real-time feedback might empower self-directed changes in behavior that could improve adherence to HU.