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📍 Research Site · Mobile, AL · +27 more US sites · Get directions →

A study to evaluate the efficacy and safety of benralizumab administered subcutaneously in patients ≥ 6 to \< 18 years of age with severe eosinophilic asthma, including a well-documented history of asthma exacerbations and uncontrolled asthma receiving high-dose inhaled corticosteroid (ICS) plus at least one additional controller medication.

📍 Respiratory Research Lab · Hamilton, Ontario · Get directions →

The inhalation of adenosine 5'-triphosphate (ATP) to evoke cough (ATP cough challenge) is becoming increasingly used as a tool to measure cough hypersensitivity in patients with chronic cough. However, the safety, feasibility, and repeatability of this procedure is not widely known.

📍 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.

📍 Research Site · Montgomery, AL · +29 more US sites · Get directions →

To assess the efficacy and safety of tezepelumab in pediatric participants with severe uncontrolled asthma on medium to high-dose inhaled corticosteroids (ICS) and at least one additional asthma controller medication with or without oral corticosteroids.

📍 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.

📍 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.

📍 University of California, San Francisco · San Francisco, CA · +3 more US sites · Get directions →

This is a study to determine the safety, pharmacodynamics (PD), and pharmacokinetics (PK) of CDX-622 in adults with mild to moderate asthma.

📍 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.

📍 Research Site 13 · Bakersfield, CA · +12 more US sites · Get directions →

The objective of this study is to assess the potential for GB-0895 treatment to improve the health of adolescents and adults with severe asthma that is uncontrolled by inhaled corticosteroids (ICS) and conventional asthma controllers. The study details include: Study treatment: randomized to receive either GB-0895 or placebo administered every 6 months over 52 weeks.

📍 Research Site · Chandler, AZ · +33 more US sites · Get directions →

This study evaluates the efficacy and safety of benralizumab as an add-on therapy in uncontrolled eosinophilic asthma participants treated with medium-dose ICS-LABA compared to the conventional treatment step of escalation of inhaled therapy to high-dose ICS-LABA.

📍 Pfizer Clinical Research Unit - New Haven · New Haven, CT · Get directions →

The purpose of this study is to learn about the safety of a new study medicine called PF-08103402 in healthy adults (do not have disease) and or in adults with mild-to-moderate asthma. This is the first time the study medicine is being given to people.

📍 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).

📍 GSK Investigational Site · Brooksville, FL · +8 more US sites · Get directions →

Asthma is a chronic condition marked by narrowed and swollen airways due to inflammation leading to recurring symptoms that can vary and worsen unpredictably.\\xa0The purpose of this study is to assess how depemokimab, a monoclonal antibody, affects the structure and function of the lungs in asthmatic participants with type 2 inflammation, characterized by an eosinophilic phenotype.

📍 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.

📍 GSK Investigational Site · Newport Beach, CA · +7 more US sites · Get directions →

The aim of this study is to evaluate the efficacy of depemokimab administered as an adjunctive therapy, in participants with Type 2 asthma at risk of exacerbations compared to the guideline recommended standard of care (SoC).

📍 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.

📍 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.

📍 Phoenix Children's Hospital- Site Number : 8400001 · Phoenix, AZ · +13 more US sites · Get directions →

This is a parallel, Phase 3, 2-arm study to evaluate the efficacy and long-term safety of dupilumab treatment in children 2 to \<6 years of age with uncontrolled asthma and/or recurrent severe asthmatic wheeze. The study will be conducted in 2 parts.

📍 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).

📍 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.

📍 Johns Hopkins All Children's Hospital · St. Petersburg, FL · Get directions →

The CR-AZI Study will assess the immunomodulatory effects of Azithromycin for pediatric Critical Asthma.

📍 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.

📍 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.

📍 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.

📍 Research Site 23 · Northridge, CA · +33 more US sites · Get directions →

The objective of this study is to assess the potential for GB-0895 treatment to improve the health of adolescents and adults with severe asthma that is uncontrolled by inhaled corticosteroids (ICS) and conventional asthma controllers. The study details include: Study treatment: randomized to receive either GB-0895 or placebo administered every 6 months over 52 weeks.

📍 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.

📍 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.

📍 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).

📍 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.

📍 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.

📍 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.

📍 Research Site · Anchorage, AK · +39 more US sites · Get directions →

The purpose of this study is to compare the effect of budesonide/albuterol metered-dose inhaler (BDA MDI) with albuterol sulfate metered-dose inhaler (AS MDI), both administered as needed, on the annualized rate of severe asthma exacerbations in adolescents with a documented clinical diagnosis of asthma and at least one severe exacerbation in the prior year.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 Alberta Children's Hospital · Calgary, Alberta · Get directions →

Despite optimal initial emergency department (ED) therapy, 50% of children with severe acute asthma have ongoing moderate-severe respiratory distress. Guidelines recommend intravenous magnesium (IVMg) for them, yet evidence for IVMg efficacy is scant and disparate.

📍 Sun City Clinical Research · Glendale, AZ · +1 more US site · Get directions →

This is a phase 1 study, randomized, double-blind, placebo-controlled, 2 treatment, 2 period crossover study to evaluate the pharmacokinetics, safety, and tolerability of a single inhaled dose of ASY202 in adults with stable asthma. Following screening, eligible participants will be enrolled and randomized to one of two treatment sequences.

📍 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.

📍 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.

📍 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).

📍 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.

📍 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.

📍 Teva Investigational Site 12087 · Phoenix, AZ · +39 more US sites · Get directions →

The primary objective of the trial is to evaluate the efficacy of fluticasone propionate/albuterol sulfate multidose dry powder inhaler with electronic module (Fp/ABS eMDPI). Secondary objectives are: * To evaluate the efficacy of Fp/ABS eMDPI administered four times daily * To evaluate the safety and tolerability of Fp/ABS eMDPI administered four times daily over four weeks * To investigate the pharmacokinetics of Fp/ABS eMDPI, ABS eMDPI and Fp eMDPI after administration of a single dose The planned study duration for each participant is approximately 10 weeks, excluding an optional prescreening visit.

📍 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.

📍 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.

📍 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.

📍 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.

📍 Sir Charles Gairdner Hospital · Nedlands, Western Australia · Get directions →

This study tests whether an asthma medication called dupilumab can help people achieve complete asthma control (called "remission") when given earlier in their disease, before asthma becomes severe. Currently, most people with asthma only receive advanced treatments like biologics after their condition has worsened significantly and caused lung damage.

📍 Massachusetts General Hospital · Boston, MA · Get directions →

Asthma is a heterogeneous disorder in which multiple potential inflammatory pathways contribute to airway obstruction. The biological basis for airway inflammation is the subject of intensive investigation.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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 purpose of this study is to develop and evaluate the usefulness of hyperpolarized (HP) 129Xe gas MRI for regional assessment of pulmonary function.

📍 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.