View clinical trials related to Sickle Cell Disease.
Filter by:This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks. The study also assesses the effects of this amino acid composition on the structure and function of the vascular system. Physiological effects on structure and function will be assessed by Magnetic Resonance Imaging (MRI) to assess blood flow in the brain and kidneys and the 6-Minute walk with pulse oximetry. Changes in blood biomarkers of inflammation will also be assessed.
Sickle cell disease (SCD) is the most common genetic disorder in the United States affecting approximately 100,000 individuals primarily of African ancestry. Pain is the most common complication of SCD. Currently, the mainstay therapy for pain in SCD is opioids. The CDC recommends using non-opioid, non-pharmacologic therapies for pain. There is a growing body of literature to support the use of various integrative therapies for pain. Acupuncture therapy is a non-pharmacological Chinese medicine approach which has been used in many non-SCD conditions associated with pain. Proposed study will test acceptability and feasibility of use of acupuncture in SCD patients hospitalized for pain. It is hypothesized that the use of acupuncture as an adjuvant therapy will be acceptable to SCD patients admitted for pain control. Its impact on opioid use and circulating cytokines and neuropeptides will also be determined.
MitoQ is commercially available as a dietary supplement and it has been tested as a potential drug in other diseases, but it has never been tested in patients with sickle cell disease. The goal of this research is to study if MitoQ, a molecule that works as an antioxidant by removing potentially damaging agents in a living organism, improves platelet function in patients with sickle cell disease (SCD).
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion. All other treatment is standard of care.
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.
The SCD-CARRE trial is a Phase 3, prospective, randomized, multicenter, controlled, parallel two-arm study aimed to determine if automated exchange blood transfusion and standard of care administered to high mortality risk adult SCD patients reduces the total number of episodes of clinical worsening of SCD requiring acute health care encounters (non-elective infusion center/ER/hospital visits) or resulting in death over 12 months as compared with standard of care.
This project proposes to develop, test and evaluate targeted interventions to improve clinical provider prescribing of and patient adherence to hydroxyurea (HU). Using a stepped-wedge design, The investigators will test two innovative interventions utilizing mobile health to address both patients' and providers' needs: 1) an mHealth application for patients (InCharge Health app) that includes multi-component features to address the memory, motivation, and knowledge barriers to hydroxyurea use, and 2) an mHealth toolbox application for providers (HU Toolbox app) that addresses clinical knowledge barriers in prescribing and monitoring hydroxyurea use. These two interventions will be tested through the following aims: Aim 1. Improve Patient Adherence to Hydroxyurea: Addressing Memory, Motivation, and Knowledge Barriers to Hydroxyurea Use. Primary hypothesis: The investigators hypothesize that among adolescents and adults with SCD, the adherence to hydroxyurea, as measured by percentage of days covered (PDC), will increase by at least 20% at 24 weeks after receiving the InCharge Health app, compared to their hydroxyurea adherence at baseline. Sub-aim 1.a. To examine and assess both patient engagement and behaviors related to use of the InCharge Health app, the investigators will evaluate consistent use of the app among enrolled patients, patient satisfaction, and continued use of the app beyond the study period. Sub-Aim 1.b. To examine the clinical influence of the use of the InCharge Health app on PDC, patients' clinical outcomes, perceived health literacy, health related quality of life, and perceived self-efficacy between baseline and 24 weeks. Aim 2. Improve Provider Hydroxyurea Awareness, Prescribing and Monitoring Behaviors. Sub-Aim 2.a. To examine and assess provider engagement and behaviors related to use of the HU Toolbox, the investigators will evaluate consistent use of the app among enrolled providers, providers' satisfaction, and continued use of the app beyond the study period. Sub-Aim 2.b. To assess the combined effects of the patient and provider mHealth interventions on hydroxyurea and health care utilization, the investigators will examine if the changes in hydroxyurea adherence are enhanced by the use of both provider and patient interventions compared to those not exposed to one or both interventions. Aim 3. Identify and Evaluate the Barriers and Facilitators to the use of mHealth Interventions.
The main objective of this study is to prove the superiority of a procedure which calculates the volume of RBCs to transfuse and the time between apheresis based on this algorithm, compared to the current procedure. The primary endpoint would be the number of patients with individually achieved objectives in terms of % HbS before each apheresis (which reflects the effectiveness of the previous apheresis) over a period of 12 months. The secondary objectives would be to compare the volume differences of transfused RBCs in both groups over a period of 12 months, the occurrence of clinical events and the satisfaction of patients and physicians. The investigators hope that this study would improve the efficiency and the performance of apheresis in sickle cell patients. The investigators also hope to facilitate the organization of procedures with the flexibility that would allow the use of this algorithm.
Aim. Pilot FOCUS. A pilot randomized controlled trial will compare FOCUS to standard care. Investigators will randomize a total of 60 12- to 18-year-old patients to either FOCUS intervention (n=15 with SCD; n=15 with cancer) or treatment as usual (n=15 with SCD; n=15 with cancer). Randomization will be stratified to match patients based on age, sex, and medical condition (SCD type, cancer type). FOCUS participants will engage in the intervention and complete measures for 10 days post hospital discharge. Control participants will complete similar measures but not receive the intervention. Mixed qualitative and quantitative measures of feasibility, acceptability, and preliminary outcomes will be conducted to evaluate both the intervention and study procedures.
Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP). Those subjects who pass Screening will enter into the 2-week Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the 2-week Run-in Period will be randomized into active and control groups (2 active to 1 placebo) and enter the 12-week Treatment Period. At each visit during the Treatment Period, the target ulcer will be measured by digital photographic planimetry, the Principal Investigator will assess the wound qualitative attributes, and the DIDP (or placebo patch) will be placed as the primary wound dressing. At each visit the subject will also receive/review a daily diary to document pain , study drug compliance, and analgesic use.