View clinical trials related to Sickle Cell Disease.
Filter by: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.
The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell disease patients. Given this lack of data, primary-care Sickle Cell Disease (SCD) physicians and transplant physicians are prevented from a factual debate over the benefit/risk ratio for each patient and refining indications of transplant in patients. Therefore, the present study seeks to describe and compare the very long-term outcomes after either Human Leukocyte Antigen (HLA) -matched sibling transplantation (study arm) and "non-transplant care" for severe sickle cell disease SCA patients in order to yield robust comparative data regarding both arms. The main objective is to assess the benefit of Hematopoietic stem cell transplantation (HSCT) regarding quality of life compared to standard care after 10 years, in patients with severe Sickle Cell Disease (SCD).
Large knowledge gaps remain regarding strategies to promote the adoption of hydroxyurea (HU), particularly in sub-Saharan African countries including Nigeria, where more than 75% of annual sickle cell anemia births occur. The vast majority of people with SCD in Africa do not receive evidenced-based health care (e.g., newborn screening, health education, prophylaxis for infection, optimal nutrition and hydration, blood transfusion, transcranial Doppler screening, and HU therapy), despite its effectiveness in reducing SCD-related adverse outcomes and mortality. The use of HU in SSA is <1% among SCD patients. The investigators' preliminary findings indicate that provider-level barriers are significant and must be addressed to improve HU adoption. To address HU adoption, the investigators will use the NIH-funded study (e.g., Realizing Effectiveness Across Continents with Hydroxyurea (REACH) Clinical Trial (NCT01966731)) that developed an evidence-informed, clinical, practical, and easy-to-follow algorithm to 1) Screen patients for sickle cell disease (SCD), 2) Initiate HU treatment, and 3) Maintain HU dosage over time (SIM) for the improved management of SCD as our intervention. The Nigerian government released guidelines supporting the SIM intervention for HU adoption for improved SCD management, and HU is on the list of essential medicines for Nigeria. The investigators' implementation strategy for improving SCD management in Nigeria uses a practical and replicable evidence-based task-sharing strategy, TAsk-Strengthening Strategy for Hemoglobinopathies (TASSH), adopted from the TAsk-Strengthening Strategy for Hypertension control (TASSH) trials in Ghana and Nigeria containing the essential components of i) Training healthcare workers/providers to be more patient-centered in clinical consultations, ii) Clinical reminders, and iii) Practice facilitation (TCP) known as (TASSH TCP) for SCD management. Using a sequential exploratory mixed-methods study design, the investigators will conduct this study using the Exploration, Preparation, Implementation, and Sustainment (EPIS) framework in four sequential phases to assess the effectiveness of SIM adoption by providers in the context of the TASSH TCP implementation strategy in Nigeria.
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101).
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101).
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)
The primary purpose of this study is to evaluate the effect of mitapivat on albumin creatinine ratio (ACR) response in participants with sickle cell disease (SCD) and nephropathy.
This study is for caregivers of young children with sickle cell disease and adolescents with sickle cell disease who are currently prescribed hydroxyurea and are receiving care at one of the study sites. The study will assess retention and engagement during a pilot randomized control trial comparing video directly observed therapy (VDOT) to attention control. We also hope to understand more about patient and family preferences longer-term adherence monitoring and intervention. Participants will use an electronic adherence monitor (provided by the study team) to measure how often they are taking their hydroxyurea. Participants will also be asked to complete questionnaires throughout the study period to provide information about their expectations for, experience with, and satisfaction with the study materials.