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📍 Uni of Alabama at Birmingham · Birmingham, AL · +39 more US sites · Get directions →

This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo.

📍 Univ of Alabama Birmingham · Birmingham, AL · +38 more US sites · Get directions →

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.

📍 Icahn School of Medicine at Mount Sinai · Manhattan, NY · Get directions →

Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.

📍 University of Alabama at Birmingham- Site Number : 8400003 · Birmingham, AL · +15 more US sites · Get directions →

This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B).

📍 University of Alabama at Birmingham · Birmingham, AL · +18 more US sites · Get directions →

The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.

📍 University Of Alabama · Birmingham, AL · +13 more US sites · Get directions →

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.

📍 Stanford University Medical Center · Stanford, CA · +12 more US sites · Get directions →

Powerful new drugs that can prevent or delay end stage kidney disease (ESKD) - so called sodium-glucose cotransporter-2 inhibitors (SGLT2i) - are now available for patients with type 2 diabetes. Whether these drugs have similar effects in patients with type 1 diabetes (T1D) remains unknown because of the few studies in this population, due to concerns about the increase in risk of diabetic ketoacidosis (DKA, a serious, potentially fatal acute complication of diabetes due to the accumulation of substances called ketone bodies) observed with SGLT2i therapy in T1D.

📍 Smilow Cancer Hospital · New Haven, CT · +23 more US sites · Get directions →

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

📍 University of Alabama Saint Vincent's Birmingham · Birmingham, AL · +39 more US sites · Get directions →

This trial will examine if maridebart cafraglutide as an adjunct to standard of care will lead to a reduction in heart failure (HF) events such as HF hospitalizations and urgent HF visits, cardiovascular (CV) deaths and improvement in HF symptoms in participants with HF with preserved ejection fraction (HFpEF) and HF with mildly reduced ejection fraction (HFmrEF) who are obese. This is a phase 3, global, multicenter, 2-part trial with a double-blind period and an open-label extension (OLE).

📍 Pinnacle Research Group, LLC · Anniston, AL · +39 more US sites · Get directions →

This study is open to adults with type 2 diabetes, high blood pressure, and cardiovascular disease. People can join the study if they have these conditions and do not have a history of heart failure.

📍 University of Alabama at Birmingham · Birmingham, AL · +6 more US sites · Get directions →

This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.

📍 University of Alabama Birmingham · Birmingham, AL · +5 more US sites · Get directions →

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).

📍 Alliance For Multispecialty Research - Daphne · Daphne, AL · +39 more US sites · Get directions →

The primary objective of this trial is to demonstrate that maridebart cafraglutide is superior to placebo when given as an adjunct to standard of care with respect to reducing cardiovascular (CV) morbidity and mortality.

📍 Advanced Cardiovascular, LLC · Alexander City, AL · +39 more US sites · Get directions →

This study is being done to look at the safety and effect of NNC0487-0111 in people with Heart Failure with preserved Ejection Fraction (HFpEF) or Heart Failure with mildly reduced Ejection Fraction (HFmrEF) and excess body weight when compared to placebo. The purpose of this clinical study is to find out if NNC0487-0111 is safe and effective for treating people who have HFpEF or HFmrEF and excess body weight.

📍 Site # 8400006 · Atlanta, GA · +4 more US sites · Get directions →

The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV (licensed pneumococcal vaccine) when given as a single dose to children aged 2 to 17 years with sickle cell disease who had received or not a previous vaccination with pneumococcal conjugate or pneumococcal polysaccharide vaccine.

📍 Children's Hospital Los Angeles · Los Angeles, CA · +5 more US sites · Get directions →

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

📍 Children's Hospital of Colorado · Aurora, CO · +3 more US sites · Get directions →

This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants.

📍 Yale School of Medicine · New Haven, CT · +3 more US sites · Get directions →

This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.

📍 Nephrology of North Alabama · Decatur, AL · +39 more US sites · Get directions →

This is a two-part, phase 2b and phase 3 combined prospective, interventional, multicenter, randomized, double-blind, placebo-controlled study. Part 1: Phase 2b is a dose-finding study for CSL300 vs placebo.

📍 New York Presbyterian Hospital - Morgan Stanley Children's Hospital · New York, NY · +2 more US sites · Get directions →

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe 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) using CTX001.

📍 University of Alabama at Birmingham · Birmingham, AL · +2 more US sites · Get directions →

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.

📍 Greenstone Biosciences · Palo Alto, CA · +1 more US site · Get directions →

Semaglutide is a glucagon-like peptide-1 (GLP-1) receptor agonist, primarily used for treatment of type-2 diabetes mellitus. GLP-1 receptors are present on pancreatic islet β-cells, δ-cells and α-cells.

📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.

📍 Children's Healthcare fo Atlanta at Hughes Spalding · Atlanta, GA · +1 more US site · Get directions →

The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit.

📍 University of California, Los Angeles · Los Angeles, CA · +1 more US site · Get directions →

This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).

📍 University of Alabama at Birmingham · Birmingham, AL · Get directions →

Obese individuals have a higher prevalence of nocturnal hypertension and non-dipping blood pressure (BP). These conditions are associated with an increased risk of cardiovascular (CV) events and death.

📍 Vanderbilt University Medical Center · Nashville, TN · Get directions →

The overall purpose of this study is to learn more about the metabolic effects of angiotensin-(1-7) in the insulin resistant state associated with obesity. Pharmacologic approaches to increase angiotensin-(1-7) levels or its actions are currently in development for treatment of metabolic-related diseases such as obesity and type II diabetes, based on findings from animal studies.

📍 University of Illinois at Chicago College of Medicine · Chicago, IL · Get directions →

A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).

📍 University of Kansas Medical Center · Kansas City, KS · Get directions →

The main purpose of research is to examine and understanding the development of hypertension in obese adults with insulin resistance. Findings from our studies will identify unique mechanisms that can be targeted to limit increases in vascular dysfunction and reduce the excessively high prevalence of hypertension and risk for cardiovascular disease (CVD).

📍 University of Texas Health Science Center at San Antonio · San Antonio, TX · Get directions →

The investigators are studying how to help people with prediabetes (Pre-DM) and obesity. The goal is to use new and affordable treatments to bring blood sugar levels back to normal and help participants to lose weight.

📍 University of Texas Health Science Center at San Antonio · San Antonio, TX · Get directions →

This study will provide insight into whether cardiac function changes with oral Ketone Esters (KE) administered to patients with Type 2 Diabetes Mellitus (T2DM) and Heart failure with reduced ejection fraction (HFrEF). Plasma ketones are avidly extracted by cardiac muscle and their uptake is not dependent upon insulin or influenced by insulin resistance.

📍 Northwestern University · Chicago, IL · Get directions →

The goal of this study is to better understand the effects of a sodium-glucose transport protein 2 inhibitor, dapagliflozin, added on to standard of care on heart and lung function and circulating metabolites (substances created when our bodies break down food, drugs, or its own tissues) in patients with chronic kidney disease.

📍 George E Wahlen VA Medical Center · Salt Lake City, UT · Get directions →

Many control mechanisms exist which successfully match the supply of blood with the metabolic demand of various tissues under wide-ranging conditions. One primary regulator of vasomotion and thus perfusion to the muscle tissue is the host of chemical factors originating from the vascular endothelium and the muscle tissue, which collectively sets the level of vascular tone.

📍 UPMC · Pittsburgh, PA · Get directions →

The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET).

📍 Ralph H. Johnson VA Medical Center, Charleston, SC · Charleston, SC · Get directions →

The purpose of this study is to demonstrate whether Behavioral Activation for depression delivered via home based telehealth (BA-HT) is effective in improving social and role functioning in Veterans recently discharged from Cardiovascular disease (CVD-related) inpatient care. Eligible participants will receive either (1) twelve sessions of BA-HT or (2) standard best practices post CVD hospitalization care.

📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early.

📍 Augusta University · Augusta, GA · Get directions →

The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor.

📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →

The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork problems and if they have any side effects. The study will assess any thinking or attention problems participants may have both before taking this drug and after.

📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens.

📍 Boston Children's Hospital · Boston, MA · Get directions →

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.

📍 City of Hope Medical Center · Duarte, CA · Get directions →

This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.

📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves.

📍 Children's Hospital of Pittsburgh of UPMC · Pittsburgh, PA · Get directions →

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease.

📍 Children's Hospital of Philadelphia · Philadelphia, PA · Get directions →

To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.

📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →

This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.

📍 Southern California Hospital at Culver City / Southern California Hospital at Hollywood / Multiple US-based Sub-Investigator Sites · Culver City, CA · Get directions →

This multi-arm, multi-site study investigates the safety, tolerability, and efficacy of stem cell therapy for the treatment of various acute and chronic conditions. Clinically observed initial findings and an extensive body of research indicate regenerative treatments are both safe and effective for the treatment of multiple conditions.

📍 Instituto de Investigaciones Clínicas Bahia Blanca · Bahía Blanca, · Get directions →

The purpose of this study is to measure cardiovascular outcomes with orforglipron compared with placebo in participants with atherosclerotic cardiovascular disease (ASCVD) and/or chronic kidney disease (CKD). Participation in the study will last about 5 years.

📍 Curtin University · Bentley, Western Australia · Get directions →

To evaluate the efficacy and safety of colchicine and non-enteric coated aspirin, combined or alone, to improve cardiovascular outcomes in high-risk patients with type 2 diabetes.

📍 Cevaxin - The Panama Clinic · Panama City, · Get directions →

This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days.

📍 London Health Sciences Centre - Victoria Hospital · London, Ontario · Get directions →

The overriding objectives of this study are: 1. Primary outcomes: 1.

📍 University of Ottawa Heart Institute · Ottawa, Ontario · Get directions →

CANTOR SING is a pilot single center double blinded randomized study. The investigators will compare the effect of canagliflozin (300 mg daily - intervention arm) vs.

📍 Lyndhurst Centre · Toronto, Ontario · Get directions →

Cardiometabolic Disease (CMD) is the silent killer within the spinal cord injury/disease (SCI/D) population. Many SCI/D patients' initial CMD presentation is with a heart attack, stroke, or sudden cardiac death, highlighting the need for interventions to reduce CMD risk.