View clinical trials related to Sickle Cell Disease.
Filter by:This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use. PRIMARY OBJECTIVE - To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events (AEs). SECONDARY OBJECTIVES - To characterize the efficacy of a single dose (Part A) or two doses (Part B) of motixafortide for hematopoietic stem cell (HSC) mobilization and apheresis collection in participants with SCD as determined by the yield of CD34+ cells (CD34+ cells/kg). - To measure the mobilization effects of single-day (Part A) or daily dosing (Part B) dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34+ counts - To recommend a phase 2 dosing strategy based on safety, efficacy, and mobilization effects
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.
This study is being conducted to evaluate the safety and effect of starting daily use of low dose (100 mg) aspirin in pregnant women with sickle cell disease, who are being followed in two county hospitals in Angola, in the first trimester versus the second trimester of the gestational period.
This study is an open-label, non-randomized, single-dose Phase 1/2 trial involving around 85 adult and pediatric participants aged between 2 and 50 years with sickle cell disease (SCD). It aims to assess the effectiveness of hematopoietic stem cell transplantation (HSCT) using BAH243 for SCD.
The investigators are studying how to prevent malnutrition in children with sickle cell disease (SCD) in northern Nigeria. Community health workers will teach mothers about nutritious local foods that might help children grow better than the usual advice from doctors. The investigators will enroll 148 children with SCD aged 6 to 18 months old. The investigators will determine if their weight and diet improve and check for low vitamin A levels. This information will help us find better ways to improve growth for children with SCD.
Objective: to gain insight in the pathogenesis, to identify biomarkers to recognize patients at risk for proliferative SCR and to investigate its associations with clinical and laboratory characteristics. Endpoints: The investigators will determine the difference in the above named parameters between patients with and without PSCR Study design: This case control study will include adult sickle cell disease patients with the HbSS or HbSC genotype. For both genotypes, 20 patients without sickle cell retinopathy (SCR) and 20 patients with PSCR will be included, resulting in a total of 80 patients. Venous blood samples and retinal imaging scans will be collected for each included patient.
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? Once unaffiliated individuals are found using the pathways, Investigators will employ linkage coordinators (trained staff) to engage these patients in care. Participants will be asked to fill out an assessment survey which will cover areas such as previous and current treatment, clinic and hospital experience, pain, and quality of life. Participants will also be given the option of participation in a 1-hour long interview how they feel about treatment for sickle cell disease including clinic experience, pain, and quality of life?
The study, known as the Peer suppoRt for adolescents and Emerging adults with Sickle cell pain: promoting ENgagement in Cognitive behavioral thErapy (PRESENCE), aims to determine the effectiveness of digital CBT in reducing pain, opioid use, and healthcare utilization among AYAs with SCD. It also seeks to understand the role of personalized peer support in enhancing engagement and outcomes of digital CBT interventions. By leveraging existing infrastructure for delivering virtual peer support interventions, tailored digital CBT programs for individuals with SCD, and partnerships with CBOs, the study aims to provide valuable insights into the feasibility and effectiveness of digital CBT as a pain management strategy for this vulnerable population.
The study participant is being asked to take part in this clinical trial, a type of research study, because the participant is a young child with sickle cell disease or the caregiver of a child with sickle cell disease. This study is being done to test a school readiness program for children with sickle cell disease (ages 4-6 years old). Primary Objective Assess feasibility and acceptability of an adapted school readiness intervention among preschool children (ages 4-6) diagnosed with sickle cell disease. Secondary Objectives Objective 1: Measure preliminary efficacy of the adapted school readiness intervention compared to routine care among preschool children ages (4-6) diagnosed with sickle cell disease. Objective 2: Examine implementation factors (i.e., barriers and facilitators) during post-intervention.
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.