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72 studies
Recruiting now, in plain language. Every study is watched over by an independent ethics board.
- COPD (chronic lung disease)
A Study to Investigate the Effect of AZD6793 in Participants With Moderate to Very Severe Chronic Obstructive Pulmonary Disease
Testing two doses of a medicine versus placebo for COPD symptoms
📍 Research Site · Birmingham, AL · +39 more US sites · Get directions →
This Phase IIb dose-ranging study will evaluate the efficacy and safety of 2 different doses of AZD6793 compared with placebo tablets in participants with moderate to very severe chronic obstructive pulmonary disease.
- COPD (chronic lung disease)
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
Testing how a genetic change in alpha-1 antitrypsin affects immune and lung responses to a pneumonia
📍 University of Alabama at Birmingham · Birmingham, AL · Get directions →
The goal of this study is to learn whether patients who have a genetic mutation in the genes that cause alpha 1 antitrypsin deficiency also have genetic variation in nearby genes that can increase risk for reduced immune function and respiratory infections. To investigate this hypothesis, we will compare immune responses to the 20-valent pneumococcal conjugate vaccine (PCV20, Pfizer) between participants who have one abnormal copy of the SERPINA1 gene and either no COPD exacerbations, vs those with 2 or more COPD exacerbations in the past year.
- High blood pressureKidney disease
A Phase IIb Study to Evaluate the Effect of Dapagliflozin in Combination With Baxdrostat Compared With Baxdrostat on Albuminuria in Participants With Chronic Kidney Disease and High Blood Pressure.
Testing how adding a diabetes medicine to baxdrostat affects urine protein in people with chronic
📍 Research Site · Surprise, AZ · +17 more US sites · Get directions →
International, Multicenter and Double-Blind study. The purpose is to measure the effect of baxdrostat in combination with dapagliflozin compared with baxdrostat/placebo on albuminuria, as well as safety, in participants with chronic kidney disease and high blood pressure.
- Type 2 diabetesHigh blood pressure
Polypill for Prevention of Cardiomyopathy
Testing a pill combination to prevent heart muscle disease in people with type 2 diabetes and high
📍 UT Southwestern Medical Center · Dallas, TX · Get directions →
This study will investigate the utility of a polypill-based strategy for patients with type 2 diabetes mellitus and high risk of heart failure (HF), as assessed via the WATCH-DM risk score. Polypill therapy will consist of empagliflozin 12.5 mg, losartan 25, 50 or 100 mg, and finerenone 10 mg daily.
- Sickle cell disease
A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)
Testing the safety and how an oral carbon monoxide liquid works in sickle cell disease
📍 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.
- COPD (chronic lung disease)Asthma
The Effects of Positive Airway Pressure on the Mucolytic Effects of NAC (TEAM)
Testing whether positive airway pressure helps a lung medicine (NAC) reach mucus plugs in asthma or
📍 UCSF Airway Clinical Research Center · San Francisco, CA · Get directions →
The goal of this clinical trial is to determine if positive pressure during inspiration will improve penetration of aerosolized N-Acetylcysteine (NAC) into airway mucus plugs in the lungs of patients with asthma or Chronic Obstructive Pulmonary Disease (COPD). The main questions it aims to answer are: * Does delivery of aerosolized NAC with positive inspiratory pressure have a greater effect on mucus plug burden in the lungs than delivery of NAC without positive pressure.
- Sickle cell disease
Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults
Testing whether a low dose of ketamine helps treat sickle cell pain in children and young adults
📍 Harbor UCLA Medical Center · Torrance, CA · Get directions →
The purpose of this research is to see if ketamine is effective and safe in treating children and young adults with sickle cell disease experiencing sickle cell related pain. In this study, we will compare the outcomes (such as pain scores) in persons who receive standard of care pain medicine (an opioid such as morphine) plus a low dose (amount) of ketamine to those who receive only standard of care pain medicine.
- COPD (chronic lung disease)
To Evaluate the Effect of EXPD-101/FXS7553 Compared With Placebo in Patients With COPD Over 52 Weeks of Treatment
Testing two doses of a daily medicine versus placebo in people with COPD over 52 weeks
📍 DM Clinical Research - Phoenix · Phoenix, AZ · +11 more US sites · Get directions →
This is a Phase 2 study of the efficacy, safety, tolerability and pharmacokinetics of two doses of EXPD-101 in participants with COPD. Study participants will be randomized to receive either study drug or placebo administered once daily for 52 weeks.
- Obesity / overweightHigh blood pressure
Impact GLP-1 Agonists Following Bariatric
Testing whether GLP-1 medicines after bariatric surgery affect weight and blood sugar
📍 University of Missouri Hospital · Columbia, MO · Get directions →
Glucagon-like peptide 1 (GLP-1) is a hormone that helps regulate blood glucose levels through improved insulin sensitivity and release of insulin from the pancreas, control hunger, induce satiety and plays a role in the metabolic health of a person. GLP-1 receptor agonists (GLP1-RAs) have been shown to be effective in achieving weight loss in patients with type 2 diabetes while improving blood glucose control.
- Sickle cell disease
Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease
Testing whether extra arginine lowers pain in children with sickle cell
📍 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.
- Sickle cell disease
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Testing a once-daily mouth medicine in teens with sickle cell disease
📍 The Hospital for Sick Children · Toronto, Ontario · Get directions →
This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat.
- Sickle cell disease
RH Genotype Matched RBC Transfusions
Testing whether matching donor red blood cells by RH type helps people with sickle cell who need
📍 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.
- High blood pressureCOPD (chronic lung disease)
Breathe Easier With Tadalafil Therapy for Dyspnea in COPD-PH
Testing tadalafil to see if it improves shortness of breath in COPD with high blood pressure in the
📍 Rocky Mountain Regional VA Medical Center, Aurora, CO · Aurora, CO · +4 more US sites · Get directions →
The investigators will study whether the drug tadalafil improves shortness of breath in 126 Veterans with Chronic Obstructive Pulmonary Disease (COPD) and high blood pressure in the lungs. The investigators will also assess whether tadalafil improves quality of life, home daily physical activity, exercise endurance, the frequency of acute flares of COPD, blood pressure in the lungs, and lung function.
- Sickle cell disease
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
Testing a one-time blood stem cell transplant using gene editing for severe sickle cell disease
📍 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).
- Sickle cell disease
Cannabidiol in Sickle Cell Disease
Testing twice-daily cannabidiol doses for people with sickle cell disease
📍 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.
- COPD (chronic lung disease)
Rademikibart Add-on Treatment of an Acute COPD Exacerbation (Seabreeze STAT COPD)
Testing an add-on treatment for adults having a COPD flare with type 2 inflammation
📍 University of Alabama at Birmingham Lung Health Center · Birmingham, AL · +21 more US sites · Get directions →
This is a Phase 2, multicenter study in adult participants with an acute COPD exacerbation and type 2 inflammation
- Sickle cell disease
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
Testing early heart screening and iron overload treatment in people with sickle cell disease
📍 Inova Schar Cancer · Fairfax, VA · +1 more US site · Get directions →
This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.
- Sickle cell disease
Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease
Testing whether routine red blood cell transfusions in pregnancy reduce sickle cell flare-ups or
📍 Grady Health System · Atlanta, GA · Get directions →
The goal of this study is to determine if there is a positive effect of prophylactic red blood cell (RBC) transfusion of leukoreduced, ABO, Rh (D/Cc/Ee) and Kell matched blood compared to standard of care on the number of episodes of acute sickle cell disease (SCD) manifestations or pregnancy-related complications requiring acute health care encounters (acute care/ER/Hospital visits) or resulting in death over the entirety of pregnancy until 2 months post-partum in women with SCD. RBC transfusion is the only disease-modifying therapy for pregnant women with SCD, and it is considered a standard treatment option however, there exists no consensus on the role of transfusion therapy in preventing SCD-related pregnancy complications.
- Sickle cell disease
Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial
Testing two daily doses of zinc in people with sickle cell disease to see changes in bone-related
📍 UCSF Benioff Children's Hospital Oakland · Oakland, CA · +5 more US sites · Get directions →
The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover?
- Sickle cell disease
Allo HSCT for High Risk Hemoglobinopathies
Testing donor stem cell transplant for people with high-risk sickle cell
📍 Masonic Cancer Center · Minneapolis, MN · Get directions →
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
- High blood pressureDementia / Alzheimer's
Impact of Intensive Treatment of SBP on Brain Perfusion, Amyloid, and Tau (IPAT Study)
Testing whether tightly lowering high blood pressure slows brain changes tied to Alzheimer’s
📍 University of Texas Southwestern Medical Center · Dallas, TX · Get directions →
The purpose of this study is to determine if intensive lowering of systolic blood pressure (SBP), using FDA approved medications (antihypertensive), reduces Alzheimer's Disease pathology (i.e., excessive brain amyloid and tau protein deposition) in older adults at high risk for memory decline or dementia.
- COPD (chronic lung disease)
A Phase IIb Ensifentrine-glycopyrrolate Fixed-dose Combination Dose Ranging Study in Subjects With COPD
Testing different doses of two inhaled medicines for COPD symptoms
📍 SEC Clinical Research, LLC · Dothan, AL · +39 more US sites · Get directions →
This study will assess the safety and efficacy of fixed dose combinations of ensifentrine with two different glycopyrrolate dose levels compared to placebo and to the individual components of the fixed dose combinations, each administered twice a day via standard jet nebulizer, in adult subjects with chronic obstructive pulmonary disease (COPD).
- COPD (chronic lung disease)💵 May compensate
Effect of the Inhaled Triple Therapies Over the Small Airway in Biomass Exposure
Comparing inhaled triple versus double medicines for COPD lung airways after wood smoke exposure
📍 Insituto Nacional de Enfermedades Respiratorias "Ismael Cosío Villegas" · Mexico City, Tlalpan · Get directions →
This phase IV randomized controlled clinical trial intend to compare the effect of three close standard inhaled triple therapies and one close standard inhaled double therapy on the small airway in patients with Chronic Obstructive Pulmonary Disease (COPD-B) and chronic bronchitis without obstruction (BCNO) exposed to wood smoke. The treatment phase duration is of 3 months.
- COPD (chronic lung disease)
Compare the Effects of Nebulizer Versus Inhaler Based Therapy for COPD Using Long-acting Bronchodilators
Comparing nebulizer and inhaler treatments for COPD symptoms
📍 The University of Tennessee Graduate School of Medicine · Knoxville, TN · Get directions →
The purpose of this study is to compare the effectiveness of inhaled bronchodilators delivered via nebulizers vs. dry powder inhalers (DPIs) in symptomatic participants with Chronic Obstructive Pulmonary Disease (COPD) who have airflow obstruction (FEV1/FVC ≤ 70%) and show significant air trapping (RV ≥ 120% of predicted).
- Obesity / overweightHigh blood pressure💵 May compensate
Optimizing Portal Hypertension With TIPS and Interval Metabolic Surgery for Advanced Liver Disease
Testing treatments to lower high pressure in liver veins in people with advanced liver disease and
📍 Cleveland Clinic Main Campus · Cleveland, OH · Get directions →
Cirrhosis is a form of advanced liver disease that can lead to serious complications, especially when combined with severe obesity. Many patients with cirrhosis also develop a condition called clinically significant portal hypertension (CSPH), which is increased pressure in the veins of the liver.
- COPD (chronic lung disease)
A Study to Learn About the Study Medicine Called PF-07275315 in People With Moderate to Severe Chronic Obstructive Pulmonary Disease
Testing how a study medicine affects safety and symptoms in people with COPD
📍 University of Alabama at Birmingham Lung Health Center · Birmingham, AL · +39 more US sites · Get directions →
The purpose of this clinical trial is to learn about the effects and safety of the study medicine PF-07275315 for the potential treatment of COPD. COPD is a condition that makes it difficult to breathe, which negatively impacts the quality of life and functioning of people who are affected.
- Sickle cell disease
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Testing immune-suppressing treatment before stem cell transplant for people with sickle cell disease
📍 Memorial Sloan Kettering at Basking Ridge (Consent only) · Basking Ridge, NJ · +5 more US sites · Get directions →
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD.
- Obesity / overweightHigh blood pressureCOPD (chronic lung disease)
Feasibility of Semaglutide in Advanced Lung Disease
Testing whether semaglutide can be given safely to people with advanced lung disease
📍 University of Pennsylvania Perelman School of Medicine · Philadelphia, PA · Get directions →
The goal of this clinical trial is to learn whether semaglutide, an FDA-approved treatment for diabetes and obesity, is feasible and tolerable in patients with advanced lung disease. The main question\[s\] it aims to answer are: 1.
- Sickle cell disease
A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease
Testing how BMS-986470 affects the body and safety in sickle cell disease
📍 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.
- COPD (chronic lung disease)
A Study to Evaluate Solrikitug in Participants With COPD (ZION)
Testing solrikitug in people with COPD to check safety and effects
📍 University of Alabama at Birmingham Hospital · Birmingham, AL · +39 more US sites · Get directions →
A Randomized, Double-blind, Placebo-controlled, Multiple Dose-Ranging Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Solrikitug in Participants with Chronic Obstructive Pulmonary Disease (COPD).
- Sickle cell disease
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
Testing a partial stem cell transplant from matched donors for severe sickle cell disease or
📍 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.
- Sickle cell disease
Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin
Testing empagliflozin to treat kidney disease related to sickle cell
📍 University of Illinois Chicago, Sickle Cell Center · Chicago, IL · Get directions →
Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA.
- Sickle cell disease
Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody
Testing a transplant plan with daratumumab to reduce blood cell failure in sickle cell disease
📍 Children's National Hospital · Washington D.C., DC · Get directions →
This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies.
- COPD (chronic lung disease)
A Study of WIN378 in Participants With Moderate to Severe COPD (Sirius)
Testing the right dose of a long-acting medicine for moderate to severe COPD
📍 WB Contracted Clinical Research Site · Miami Lakes, FL · +3 more US sites · Get directions →
This study is trying to identify the right dose of a long-acting medicine called WIN378 for people with moderate - severe chronic obstructive pulmonary disease (COPD). WIN378 blocks the action of a protein called TSLP that causes inflammation in the lung and may contribute to COPD control and symptoms.
- Sickle cell disease
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
Testing different ways to use hydroxyurea for treating sickle cell anemia
📍 Cincinnati Children's Hospital Medical Center · Cincinnati, OH · Get directions →
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for people with SCA.
- High blood pressureKidney disease
Equol and Vascular Function in Women With Chronic Kidney Disease
Testing whether equol affects blood vessel function in women with chronic kidney disease
📍 University of Colorado Anschutz Medical Campus · Aurora, CO · Get directions →
The risk of cardiovascular disease (CVD) is significantly elevated in patients with chronic kidney disease (CKD). Notably, women with CKD commonly experience menstrual disturbances induced by CKD, which may contribute to impaired vascular function and elevated CVD risk.
- Sickle cell disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Testing how a new medicine works in healthy people and sickle cell patients
📍 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).
- Sickle cell disease
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Testing a bone marrow transplant with lower-intensity treatment for kids with sickle cell and
📍 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.
- Sickle cell disease
Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
Testing a stem cell transplant plan for children with sickle cell disease using a mismatched blood
📍 Alberta Children's Hospital · Calgary, Alberta · Get directions →
The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression.
- Sickle cell disease
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Testing stem cell transplant with specific immune cell removal for sickle cell
📍 New York Medical College · Valhalla, NY · Get directions →
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.
- Sickle cell disease
Intravenous L-Citrulline for Vaso-occlusive Pain Episode in Sickle Cell Disease
Testing whether an IV dose of citrulline can treat sudden pain in hospitalized sickle cell patients
📍 Children's National Hospital · Washington D.C., DC · Get directions →
The goal of this clinical trial is to learn if intravenous citrulline works to treat acute pain in hospitalized patients with sickle cell disease. It will also learn about the safety of intravenous citrulline.
- Sickle cell disease
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Testing an injectable birth control shot to reduce pain in women with sickle cell disease
📍 Emory University · Atlanta, GA · +1 more US site · Get directions →
This research is being conducted to see if using an injectable contraception, Depot Medroxyprogesterone Acetate (Depo-Provera), can reduce the pain experienced by women with sickle cell disease. Participants in this study will be adult women with sickle cell disease who regularly experience sickle cell pain.
- Sickle cell disease
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
Testing the safety of a medicine (given under the skin) in adults with sickle cell disease
📍 UAMS Medical Center · Little Rock, AR · +4 more US sites · Get directions →
This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. The primary aim of the study is to assess the safety of anumigilimab in participants with SCD.
- COPD (chronic lung disease)
The Cardiovascular Consequences of Sleep Apnea Plus COPD (Overlap Syndrome)
Studying how sleep apnea together with COPD affects heart health
📍 UCSD Sleep Research · La Jolla, CA · Get directions →
Major progress has been made in the area of cardiovascular disease, but we believe that further progress will involve mechanistically addressing underlying respiratory causes including chronic obstructive pulmonary disease (COPD) and obstructive sleep apnea (OSA). The most common cause of death in COPD is cardiovascular, although mechanisms are unknown.
- Sickle cell disease
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
Testing a vein drug (hemopexin) to treat pain crises from sickle cell
📍 Univ. of California, San Francisco Health Care · Oakland, CA · +15 more US sites · Get directions →
This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.
- Sickle cell diseaseDementia / Alzheimer's
Biomarker-based Trial of NPC-1 for Alzheimer's Pathology
Testing an early treatment to change Alzheimer’s blood markers in adults with memory decline
📍 Massachusetts General Hospital · Boston, MA · Get directions →
This early phase, open label, single arm clinical trial will determine the intraindividual safety, tolerability and effects of NPC1 (parthenolide and ipriflavone) on blood-based biomarkers of Alzheimer's disease (AD) pathology among adults with subjective cognitive decline, mild cognitive impairment, or Alzheimer's disease and objective indicators of seeding AD pathology
- Sickle cell disease
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Testing if adding ruxolitinib lowers graft failure after transplant in children with sickle cell
📍 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.
- Sickle cell disease
Alendronate for Osteonecrosis in Adults With Sickle Cell Disease
Testing oral alendronate in adults with sickle cell disease and bone damage
📍 UC Davis Comprehensive Cancer Center · Sacramento, CA · Get directions →
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
- Sickle cell disease
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
Testing defibrotide to prevent liver blood vessel problems in sickle cell patients getting a stem
📍 University of California Los Angeles · Los Angeles, CA · +3 more US sites · Get directions →
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS.
- Sickle cell disease
Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
Testing a stem cell transplant plan to treat aggressive sickle cell disease
📍 University of Illinois at Chicago · Chicago, IL · Get directions →
The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v.
- COPD (chronic lung disease)
Hyper Polarized Xenon-129 MRI vs Xenon-133 Scintigraphy
Comparing a lung scan that uses xenon MRI with a radioactive scan for COPD
📍 University of Virginia Health System · Charlottesville, VA · Get directions →
Hyper polarized Xenon-129 MRI will be directly compared to a radioactive Xe-133 scintigraphy to detect defects in lung ventilation from airflow limitation. This study is conducted as a pilot study with intention to conduct a larger clinical trial.
- Sickle cell disease
Relationship Between Abnormal Myocardial Perfusion and Diastolic Dysfunction in Sickle Cell Disease Using PET
Testing how blood flow to the heart during stress relates to heart stiffness in people with sickle
📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →
There is limited information on what causes injury to the heart in individuals with Sickle Cell Disease (SCD). Researchers in this study want to see if decreased blood flow to the heart during stress could be causing the heart damage seen in SCD patients.
- COPD (chronic lung disease)
Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure
Testing whether lung transplant first can improve lung function before bone marrow transplant for
📍 UPMC Presbyterian · Pittsburgh, PA · +1 more US site · Get directions →
The purpose of this study is to determine whether a lung transplantation prior to bone marrow transplantation (BMT) would allow for restoration of pulmonary function prior to BMT, allowing to proceed to BMT, to restore hematologic function.
- Sickle cell disease
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
Testing a lower-toxicity blood stem cell transplant for children with sickle cell disease
📍 Children's National Health System · Washington D.C., DC · +4 more US sites · Get directions →
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
- Sickle cell disease
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Testing whether using mismatched donor stem cells works for severe sickle cell disease and other
📍 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.
- Sickle cell disease
Tocilizumab for Acute Chest Syndrome
Testing a low dose medicine to see if it helps treat acute chest syndrome in people with sickle cell
📍 University of Chicago · Chicago, IL · Get directions →
The investigators are evaluating the role of a low dose of tocilizumab in treating acute chest syndrome in patients with sickle cell disease. Tocilizumab inhibits interleukin-6 (IL-6) receptors and is used to treat rheumatoid arthritis and severe cytokine release syndrome, which can be seen with chimeric antigen receptor T-cell (CAR-T) therapy, and it is also authorized for treatment of COVID-19.
- COPD (chronic lung disease)💵 May compensate
Feasibility, Safety and Efficacy of Nebulized Long-Acting Bronchodilators (Formoterol and Revefenacin) vs. Short-Acting Bronchodilators (Albuterol and Ipratropium) in Hospitalized Patients With AECOPD
Testing nebulized long-acting breathing medicines vs usual short-acting ones in hospitalized COPD
📍 University of Tennessee Medical Center · Knoxville, TN · Get directions →
The purpose of this study is to assess the feasibility, safety and efficacy of a combination of nebulized Formoterol and Revefenacin among patients hospitalized for AECOPD compared with standard-of-care therapy with nebulized Albuterol and Ipratropium.
- Sickle cell disease
Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study
Study allowing people with sickle cell disease who finished a prior treatment study to keep getting
📍 University Of Alabama · Birmingham, AL · +6 more US sites · Get directions →
This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator.
- Heart / cardiovascular diseaseHigh blood pressure
Sympathetic-vascular Dysfunction in Obesity and Insulin Resistance (Vitamin C Study)
Studying how vitamin C affects blood vessel function in obese adults with insulin resistance and
📍 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).
- High blood pressureCOPD (chronic lung disease)
Pulmonary Hypertension SOLAR
Testing a noninvasive MRI pattern to study lung blood vessel changes in pulmonary hypertension
📍 Duke University Medical Center · Durham, NC · Get directions →
The main goal of this study is to develop a noninvasive signature for pulmonary vascular remodeling in Group 3 PH patients, using hyperpolarized 129Xe magnetic resonance imaging (129Xe MRI). Such a signature may identify Group 3 PH responders to PAH-specific therapies.
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