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📍 Banner Sun Health Research Institute · Sun City, AZ · +39 more US sites · Get directions →

The goal of this Phase 2 clinical trial is to investigate the efficacy and safety of NEU-411 in men and women aged 40-80 years with early Parkinson's Disease (PD) who have predicted elevations in the activity of the "leucine-rich repeat kinase 2" ("LRRK2" for short) pathway based on their genetic profile. A DNA test will be used to identify the "LRRK2-driven" population with predicted elevation in the LRRK2 pathway.

📍 University of Alabama at Birmingham · Birmingham, AL · +26 more US sites · Get directions →

This study will examine the long-term safety of buntanetap in participants with PD. This will be a 36-month open-label safety study.

📍 Mayo Clinic · Phoenix, AZ · +33 more US sites · Get directions →

The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.

📍 Velocity Clinical Research, Mobile · Mobile, AL · +35 more US sites · Get directions →

The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called PF-07275315) for the potential treatment of moderate-to-severe asthma. Asthma is a condition that makes it challenging to breathe, which negatively impacts the quality of life and functioning of people who are affected.

📍 Research Site · Bakersfield, CA · +39 more US sites · Get directions →

This is a dose-range finding, double-blind, placebo-controlled, phase IIb study designed to assess efficacy and safety of tozorakimab administered subcutaneously in adult participants with uncontrolled asthma receiving medium-to-high dose inhaled corticosteroids.

📍 University of Arkansas for Medical Sciences · Little Rock, AR · +24 more US sites · Get directions →

This is a Phase 2 study in people with Parkinson's disease who experience motor fluctuations while taking levodopa. The study will evaluate how effective two different doses of the study drug AP-472 are when added to levodopa treatment, compared with a placebo.

📍 Kymera Investigative Site · Lancaster, CA · +26 more US sites · Get directions →

This Phase 2b study is designed to evaluate the safety and efficacy of KT-621 in participants with uncontrolled moderate to severe eosinophilic asthma. The main goals of this study are to investigate how effective KT-621 is at treating uncontrolled moderate to severe eosinophilic asthma, the safety and tolerability of KT-621, and how KT-621 behaves in the body.

📍 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.

📍 Site-049 · Birmingham, AL · +14 more US sites · Get directions →

A study to determine if BHV-8000 is efficacious, safe and tolerable in adults diagnosed with early Parkinson's disease.

📍 Enveda Investigative Site · Birmingham, AL · +13 more US sites · Get directions →

This study will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential efficacy of ENV-294 in adults with moderate to severe asthma who are receiving background treatment with inhaled corticosteroids (ICS) and long-acting beta₂-agonists (LABA). Participants will take oral ENV-294 or placebo once daily for 12 weeks.

📍 AMR Clinical · Tempe, AZ · +16 more US sites · Get directions →

The purpose of this study is to assess the safety and efficacy of brenipatide at different dose levels compared with placebo in participants with moderate-to-severe asthma. Study participation will last approximately 65 weeks, including screening, treatment, and follow-up periods.

📍 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.

📍 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.

📍 Research Site · Bakersfield, CA · +10 more US sites · Get directions →

The purpose of this study is to evaluate the effect of 2 different GP metered dose inhaler (MDI) doses relative to placebo MDI as add-on treatment to BFF MDI on lung function in participants 4 to less than 12 years of age with asthma.

📍 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).

📍 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).

📍 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?

📍 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.

📍 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.

📍 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.

📍 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.

📍 Institute for Neurodegenerative Disorders · New Haven, CT · +1 more US site · Get directions →

The study is a longitudinal, multi-center study to assess progression of \[18F\] AV-133 imaging in Prodromal PD participants. Participants will be followed for up to 24 months.

📍 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.

📍 UCSF · San Francisco, CA · +2 more US sites · Get directions →

This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs. 18F-OP-801 is intended as a biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation.

📍 Johns Hopkins University · Baltimore, MD · +1 more US site · Get directions →

This is a randomized clinical trial of metformin among overweight or obese adults with not well-controlled asthma despite maintenance inhaler therapy.

📍 University of Kentucky Medical Center · Lexington, KY · +1 more US site · Get directions →

The Goal of this study is to evaluate the safety, tolerability, and clinical responses following implantation of DSP-1083. Study enrolls both male and female patients in 2 cohorts.

📍 Purdue University · West Lafayette, IN · +1 more US site · Get directions →

Airway protective disorders are a prevalent and progressive consequence of Parkinson's Disease (PD), and often result in aspiration pneumonia which is the leading cause of death in PD. Despite this, a large number of patients with PD do not access specialized services to address these critical deficits.

📍 Institute for Neuroimmune Medicine · Davie, FL · +2 more US sites · Get directions →

This study will be evaluating the safety and efficacy of insulin and glutathione in subjects with Parkinson's Disease compared to placebo.

📍 Ann and Robert H Lurie Childrens Hospital of Chicago · Chicago, IL · +1 more US site · Get directions →

The goal of this clinical trial is to compare soy isoflavones to placebo in children who at risk of asthma and have a genetic variation which results in them making more of a pro-inflammatory protein, plasminogen activator inhibitor-1. The main questions this trail seeks to answer is: will soy isoflavones decrease the proportion of infants with aT2 high endotype at the end of treatment, and secondarily decrease the number of wheezing episodes in these children when given in the first year of life.

📍 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).

📍 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.

📍 Jesse Brown VA Medical Center, Chicago, IL · Chicago, IL · +1 more US site · Get directions →

The purpose of this study is to examine safety, feasibility, and the behavioral and brain effects of a non-invasive treatment, repetitive transcranial magnetic stimulation (rTMS), for Veterans with Parkinson's disease or atypical parkinsonism and mild impairments in their thinking. The hypothesis is that rTMS can improve thinking for people with Parkinson's disease or atypical parkinsonism who are experiencing mild problems with their thinking ability.

📍 Duke University (Asthma, Allergy and Airway Center) · Durham, NC · Get directions →

Patients with obese late onset (after childhood) asthma can have lower FeNO levels, yet be highly symptomatic and poorly responsive to inhaled steroids. This is a common asthma phenotype, particularly among females.

📍 University of Colorado Anschutz Medical Campus · Aurora, CO · Get directions →

The main purpose of this Phase 2 double blind, placebo controlled crossover clinical study is to demonstrate the efficacy and safety of CXA-10 in obese adult asthmatics. Obesity induces a chronic systemic inflammatory state characterized by impaired adipokine signaling, pro-inflammatory cytokine responses, inflammatory cell activation and enhanced generation of oxidative inflammatory mediators.

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

The purpose of the study is to enroll participants with uncontrolled asthma to evaluate the large and small airways and drug delivery utilizing two study drugs: * Beclometasone dipropionate/Formoterol fumarate dihydrate/Glycopyrronium bromide (BDP/FF/G) or Trimbow * Fluticasone furoate/Umeclidinium/Vilanterol (FluF/UMEC/VI ) or Trelegy Ellipta

📍 Kymera Investigative Site · Tampa, FL · Get directions →

This Phase 2b open-label, long-term extension study is designed to evaluate the long-term safety and efficacy of KT-621 in participants with asthma who were previously enrolled in the parent study (KT621-AS-202) of KT-621 for asthma. The main goals of this study are to investigate the long-term safety and tolerability of KT-621, the long-term efficacy of KT-621 at treating asthma, and how KT-621 behaves in the body long-term.

📍 University of Texas Medical Branch · Galveston, TX · Get directions →

This is a mechanistic, controlled, open-label, single-site study to evaluate the effects of RG-RV16 inoculation on airway mucosal gene expression and airway remodeling in 25 healthy controls (HC), in 25 allergic rhinitis subjects (AR) with cat dander allergy, and in 25 allergic asthmatic subjects (AA) with cat dander allergy. Three groups (HC, AR, and AA) will undergo screening to establish clinical history, will undergo pulmonary function testing (spirometry), and will have blood drawn for clinical characterization (IgE, , ImmunoCAP, CBC and differential), and for assessing the presence of existing neutralizing antibody against RV16.

📍 National Jewish Health · Denver, CO · Get directions →

Reslizumab is a type of medicine called a monoclonal antibody that is made in the research clinic; it works by blocking a specific protein in the body called interleukin-5. The study medicine, reslizumab, is not yet approved for doctors to treat patients with EGPA.

📍 Indiana University · Indianapolis, IN · Get directions →

The purpose of this study is to learn more about if taking a supplement called 5-hydroxytryptophan (5HTP) can improve breathing and anxiety symptoms related to asthma. To help learn more subjects will either be assigned to a group that is taking the supplement (5HTP) or a group that is taking a placebo.

📍 National Jewish Health · Denver, CO · Get directions →

Service men and women returning from deployment are significantly more likely to develop asthma and severe respiratory symptoms from airway obstruction. Why this happens is not well known, but exposure to diesel, burn pits, biomass smoke, and sandstorms are thought to play a role.

📍 Phoenix Children's · Phoenix, AZ · Get directions →

Randomized controlled trial of soluble fiber (Fruitafit Inulin). Participants will complete an ASA 24 dietary recall questionnaire to access their fiber intake.

📍 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.

📍 Children's National Hospital · Washington D.C., DC · Get directions →

OBOE is a prospective, pilot, parallel group RCT with the overall aim of examining the effect of a single dose of anti-IgE (omalizumab) vs. placebo administered at the onset of URIs in the fall season among highly exacerbation-prone, urban, and atopic youth aged 6-17 years with persistent asthma.

📍 UT Southwestern, 1440 Empire Central, Ste. LD4.100 · Dallas, TX · Get directions →

Antidepressants, particularly selective serotonin reuptake inhibitors (SSRIs), such as escitalopram are widely used for mood and anxiety disorders. However, they have also been explored, with promising findings, for a variety of disorders outside of psychiatry.

📍 University of California, San Francisco · San Francisco, CA · Get directions →

The purpose of this study is to understand whether people with Parkinson's Disease and depression have improvement in their symptoms after psilocybin therapy.

📍 San Francisco VA Medical Center, San Francisco, CA · San Francisco, CA · Get directions →

Parkinson's disease (PD) is a devastating illness that has a growing impact on Veterans. One of the most disabling symptoms is depression, which is common in PD and linked to poor quality of life and higher risk of suicide.

📍 Yale University · New Haven, CT · Get directions →

The goal of this observational study is to identify targetable neural substrates of depression in Parkinson's Disease for the first time in people with Parkinson's between the ages of 40 and 80, who are experiencing symptoms of depression.

📍 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.

📍 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.

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

Randomized Controlled Trial (RTC) testing the efficacy of a telehealth adaptation of the Cognitive-Remediation of Executive and Adaptive Deficits in Youth (C-READY) intervention to prepare adolescents with sickle cell disease for transition of care.

📍 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.

📍 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

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.