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📍 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.

📍 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.

📍 Sunnybrook Health Sciences Centre · Toronto, Ontario · Get directions →

People living with rheumatoid arthritis (RA) often experience flares-periods where their symptoms suddenly get worse. These flares can cause significant pain, make it harder to move and do daily activities, and lower overall quality of life.

📍 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.

📍 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.

📍 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.

📍 University of Colorado Anschutz Medical Campus · Aurora, CO · +30 more US sites · Get directions →

The purpose of this study is to establish the tolerability, preliminary efficacy, and pharmacokinetics of CC-97540 in participants with severe, refractory autoimmune diseases (Breakfree-1).

📍 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.

📍 Rheumatology Clinic, St. Joseph's Health Care · London, Ontario · Get directions →

This study will evaluate the retention rates of two advanced therapies, Tumour Necrosis Factor (TNF) and Janus kinase inhibitor (JAK) in the treatment of adults with Rheumatoid Arthritis (RA) over a two year period. It will also examine the ability to embed a clinical trial in a clinical situation using an electronic medical record (EMR).

📍 Nkarta Investigational Site · Orange, CA · +14 more US sites · Get directions →

This is a Phase 1/2, open-label, multi-center, multi-cohort, non-randomized dose escalation and dose expansion basket study to determine the safety and tolerability of NKX019 (allogeneic CAR NK cells targeting CD19) in participants with autoimmune diseases.

📍 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.

📍 Mayo Clinic · Scottsdale, AZ · +8 more US sites · Get directions →

This study will test the safety and effects of SBT777101 when given as a single dose to subjects with rheumatoid arthritis. It is the first study of this treatment being done in humans.

📍 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).

📍 Pinnacle Research Group, LLC · Anniston, AL · +17 more US sites · Get directions →

The goal of this clinical study is to learn more about the study drug GS-0151. The study is done to find how safe, well-tolerated the drug is.

📍 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.

📍 Equity Medical Bowling Green · Bowling Green, KY · +2 more US sites · Get directions →

Randomized study of single and multiple doses of BBT002 in healthy volunteers and in adult patients with chronic obstructive pulmonary disease (COPD) or chronic rhinosinusitis with nasal polyps (CRSwNP).

📍 University of Rochester · Rochester, NY · Get directions →

Lymphatic transport was previously examined by these investigators using Near InfraRed Indocyanine Green fluorescence imaging (NIR-ICG) of the upper extremities. They established reliable and reproducible methodologies in RA patients.

📍 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.

📍 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.

📍 Pinnacle Research Group, LLC · Anniston, AL · +8 more US sites · Get directions →

The purpose of this study is to evaluate how well LY4213663 is tolerated and what side effects may occur in healthy participants and participants with rheumatoid arthritis (RA). The study drug will be administered either subcutaneously (SC) (under the skin) or intravenously (IV) (into a vein in the arm).

📍 Research Site · Tucson, AZ · +7 more US sites · Get directions →

This trial is a Phase 1b, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with systemic sclerosis (SSc), idiopathic inflammatory myopathies (IIM), or difficult-to-treat rheumatoid arthritis (D2T RA).

📍 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.

📍 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).

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

This study evaluates the safety, tolerability, and activity of inhaled GDC-6988 in participants with muco-obstructive 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.

📍 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.

📍 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.

📍 Pinnacle Research Group /ID# 278677 · Anniston, AL · +9 more US sites · Get directions →

Systemic lupus erythematosus (SLE) is a chronic, systemic autoimmune disease characterized by B cell hyperactivity. Rheumatoid Arthritis (RA) is a chronic inflammatory disease causing pain, stiffness, swelling and loss of joint function.

📍 Gold Coast University Hospital ( Site 1601) · Southport, Queensland · Get directions →

This study looks at a study medicine called MK-1045 in people with lupus and rheumatoid arthritis (RA). The main goal of the study is to learn about the safety of MK-1045 and if people tolerate it when they receive it at different dose levels (amounts).

📍 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.

📍 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.

📍 IRIS Research and Development, LLC · Plantation, FL · Get directions →

This study will evaluate the safety and activity of AB-101 in combination with rituximab in B-cell associated autoimmune diseases where rituximab is currently FDA approved (e.g., Rheumatoid Arthritis (RA), Pemphigus Vulgaris (PV), Granulomatosis with polyangiitis (GPA)/microscopic polyangiitis (MPA) as a therapeutic, or is recommended (e.g., in Systemic Lupus Erythematosus (SLE) as a cornerstone for disease management.

📍 Research Site · Birmingham, AL · +4 more US sites · Get directions →

This open-label, Phase I study will assess the safety and tolerability of surovatamig and characterise its PK and PD following subcutaneous administration to participants with RA or SLE.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 ICON plc. · Lenexa, KS · Get directions →

The purpose of this study is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple ascending oral doses of IA-14069 in healthy subjects and in patients with RA on stable dosese of MTX, with preliminary assessment of efficacy in RA patients.

📍 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.

📍 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.

📍 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.

📍 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 Alabama at Birmingham Cancer Center · Birmingham, AL · +39 more US sites · Get directions →

This phase Ib trial studies the side effects of nivolumab and to see how well it works alone and in combination with other treatments, such as ipilimumab, cabozantinib, platinum containing therapy, and fluoropyrimidine, in treating patients with autoimmune disorders and cancer that has spread from where it first started (primary site) to nearby tissue, lymph nodes, or distant parts of the body (advanced), to other places in the body (metastatic) or cannot removed by surgery (unresectable). Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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).

📍 Medical Surgical Associates Center · St John's, · Get directions →

This trial will study the safety and efficacy of intravenous infusion of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of Rheumatoid Arthritis

📍 Medical Surgical Associates Center · St John's, · Get directions →

This trial will study the safety and efficacy of intravenous infusion of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of Pulmonary Diseases

📍 Respiratory Investigation Unit, Kingston Health Sciences Center · Kingston, Ontario · Get directions →

Bronchodilators are medications that open the bronchi to help patients with COPD to breathe better. It is still not known exactly how this effect improves shortness of breath in people with COPD.

📍 Research Site · Anniston, AL · +6 more US sites · Get directions →

The purpose of this study is to measure the safety, tolerability, PK, and PD of AZD5492 administered subcutaneously in adult participants with SLE or IIM or RA Study details include: • The study duration will be a minimum of 180 days in addition to the screening period. Additional follow-up visits may be required up to 12 months from study start.

📍 University of Alabama at Birmingham - School of Medicine- Site Number : 8400003 · Birmingham, AL · +9 more US sites · Get directions →

LPS18583 is a multinational, randomized, double-blind, placebo-controlled, parallel-group, Phase 4 study with 2 treatment groups. The purpose of this study is to assess the effect of dupilumab compared with placebo on airway inflammation, resistance, and remodeling including mucus plugging and its association with improvement on lung function, exacerbations, and quality of life improvement in participants aged 40 years of age up to 85 years of age (inclusive).