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📍 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.

📍 Local Institution - 0011 · Birmingham, AL · +16 more US sites · Get directions →

The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG).

📍 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 · Tucson, AZ · +8 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 Multiple Sclerosis.

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

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

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

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

📍 Stanford University Medical Center · Stanford, CA · +4 more US sites · Get directions →

The primary purpose of the study is to evaluate the safety and tolerability of a single-ascending intravenous (IV) dose (Part 1), a single-ascending subcutaneous (SC) dose (Part 2), and multiple ascending SC doses (Part 3) of RO7121932 in participants with multiple sclerosis (MS).

📍 Collaborative Neuroscience Network - CNS · Los Alamitos, CA · +11 more US sites · Get directions →

The purpose of this study is to generate evidence of the safety, tolerability, and pharmacokinetics/pharmacodynamics of IV LY4006896 compared with placebo in healthy participants and participants with Parkinson's disease.

📍 Profound Research, LLC · Carlsbad, CA · +5 more US sites · Get directions →

The main purpose of this study is to evaluate the safety and tolerability of the ocrelizumab subcutaneous (SC) test formulation in participants with multiple sclerosis (MS). The study consists of two treatment phases: a dose-escalation and dose-continuation phase.

📍 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.

📍 Stanford Neuroscience Health Center · Palo Alto, CA · +3 more US sites · Get directions →

This study will have two Phases: Phase 1a and Phase 1b. The goals of this clinical study are to learn more about the study drug KITE-363, by evaluating its safety, tolerability and efficacy in participants with relapsed/refractory autoimmune neurologic diseases.

📍 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.

📍 Rocky Mountain Clinical Research · Englewood, CO · +3 more US sites · Get directions →

This is a randomized, placebo-controlled, single ascending dose (SAD) study of SER-252 in participants with Parkinson's Disease (PD) and motor fluctuations.

📍 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.

📍 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.

📍 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.

📍 University of Kansas Medical Center · Kansas City, KS · +1 more US site · Get directions →

The goal of this clinical trial is to treat male and female participants with two types of Multiple Sclerosis (MS) called primary progressive or secondary progressive MS. The main questions the trial aims to answer are the following: * Is TRX319 safe when administered to patients with progressive forms of MS?

📍 Stanford University · Redwood City, CA · Get directions →

The main purpose of this study is to evaluate if obe-cel is safe or causes any side effects in adults with refractory progressive MS. The study also plans to assess if obe-cel can show early signs of efficacy in MS.

📍 Austin Clinic PPD · Austin, TX · Get directions →

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics/pharmacodynamics (PK/PD) of LY3962681 in healthy volunteers and patients with Parkinson's disease. The study consists of two parts, the Single Ascending Dose (SAD) study and the Multiple Ascending Dose (MAD) study.

📍 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).

📍 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.

📍 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.

📍 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.

📍 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.

📍 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).

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

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

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

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

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

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

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

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

📍 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.

📍 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.

📍 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).

📍 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.

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

Alpha-synucleinopathies refer to age-related neurodegenerative and dementing disorders, characterized by the accumulation of alpha-synuclein in neurons and/or glia. The anatomical location of alpha-synuclein inclusions (Lewy Bodies) and the pattern of progressive neuronal death (e.g.

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

The overall objective is to obtain an assessment of the pharmacokinetics of \[18F\]3F4AP in healthy volunteers and subjects with demyelinating diseases such as mild cognitive impairment (MCI), Alzheimer's Disease (AD), Multiple Sclerosis (MS), Spinal Cord Injury (SCI) and Spinal radiculopathy (SR).

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

Background: About 5 million adults in the U.S. have Alzheimer s disease or another adult-onset neurodegenerative disorder.

📍 Brigham MS Center, 60 Fenwood Road · Boston, MA · Get directions →

The specific aims of the study are: Primary: To determine the presence and regional distribution of microglial activation, as assessed by Fluorine-18 (18F) labeled "Peripheral Benzodiazepine Receptor 06" (PBR06) -PET, in subjects with active Relapsing Remitting Multiple Sclerosis (RRMS), Secondary Progressive Multiple Sclerosis (SPMS), and Alzheimer's Disease (AD) as compared to healthy controls Secondary: 1. To assess the relationship between microglial activation and clinical variables including disease severity and comorbidities (such as pain, fatigue and/or depression), as well as clinical MRI findings (such as lesions and atrophy) 2.

📍 Domino's Farms · Ann Arbor, MI · Get directions →

The goal of this clinical trial is to learn if taking a tributyrin supplement works to improve memory and thinking and walking and balance in adults with Parkinson disease Parkinson disease dementia. It will also learn about the safety of tributyrin supplementation.

📍 U LLC Headquarters · Cleveland, OH · Get directions →

This study tests a new, non-invasive brain stimulation device called Sphere V3.0 to help improve movement problems in people with Parkinson's disease. The device delivers gentle electrical signals to the scalp using small electrodes placed on the head.

📍 University of Kentucky · Lexington, KY · Get directions →

The investigators propose a Phase I single surgical-center, double-blinded randomized parallel clinical trial involving bilateral autologous peripheral nerve tissue (PNT) delivery into the NBM or the alternate target also affecting cognition in this population, the substantia nigra (SN), to address "repair cell" support of these areas. Twenty-four participants with idiopathic Parkinson's Disease (PD) who have selected, qualified and agreed to receive as standard of care deep brain stimulation (DBS) will be enrolled and randomly allocated to receive bilateral PNT deployment to either the NBM or SN at the time of DBS surgery.

📍 Domino's Farms · Ann Arbor, MI · Get directions →

This research is studying the use of a new type of standing desk in a small number of people to learn about the user experience for people with Parkinson's disease. 12 4-hour sessions will be performed to test the primary hypothesis that dynamic standing improves gait function compared to static standing and control sitting.

📍 Cornell University Weill Medical College · New York, NY · Get directions →

This is a phase 1/2 clinical study to evaluate the safety, tolerability, feasibility, and preliminary efficacy of NouvNeu001 in patients with advanced Parkinson's Disease.

📍 University of Kentucky · Lexington, KY · Get directions →

This phase I double-blind study focuses on the safety and feasibility of implanting autologous peripheral nerve tissue (PNT) into the substantia nigra area of the brain in persons who have been diagnosed with either Parkinson's disease (PD) or Multiple System Atrophy (MSA). 7 participants will be enrolled, with 4 participants receiving the graft and 3 receiving a sham surgery.

📍 University of California, San Diego · La Jolla, CA · Get directions →

To evaluate the safety and efficacy of transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors, CT1-DAP001, into the corpus striatum in patients with Parkinson's disease

📍 UPMC Presbyterian Hospital · Pittsburgh, PA · Get directions →

This study is designed l to evaluate the effects of GGC oral supplementation in early Parkinson's disease (PD) patients. The main objectives of the study are to evaluate: 1.

📍 Wellness, Health and Research Facility at UAB · Birmingham, AL · Get directions →

The purpose of this study is to find out whether a type of gentle nerve stimulation, called transcutaneous auricular Vagus Nerve Stimulation (taVNS), can help improve how the body regulates heart rate and blood pressure in people with Parkinson's Disease (PD). Problems with heart rate and blood pressure control are common and can make it harder for people to exercise or do daily activities.

📍 Pharmaron CPC, Inc. · Baltimore, MD · Get directions →

This is a randomized, double-blind, placebo-controlled, dose-escalation study in healthy subjects to evaluate the safety, tolerability, pharmacokinetics of HL-400 (a NLRP3 inhibitor) following oral single and multiple ascending dose administration.

📍 UAB Advanced Imaging Facility · Birmingham, AL · Get directions →

The primary objective of this substudy is to measure the concentration and the regional brain distribution of activated brain microglia/macrophages using the PET ligand \[18F\]DPA-714 in participants enrolled in the UAB Innate and Adaptive Immunity in Parkinson's Disease (Clinical Research Core) and Longitudinal \[18F\]DPA-714 Imaging in a Parkinson Disease Cohort studies. The PET tracer \[18F\]DPA-714 binds to the 18 kDa translocator protein (TSPO, also known as the peripheral benzodiazepine receptor) in the mitochondria of activated microglia/macrophages and provides a non-invasive measure of neuroinflammation.

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

The goal of this clinical trial is to assess the safety and tolerability of the surgical transplantation of dopaminergic progenitor cells into the brains of participants with Parkinson's disease. The transplanted dopaminergic cells will be derived from the participant's own skin cells.

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

The purpose of this study is to learn more about the effects of midodrine and droxidopa, two medications used for the treatment of orthostatic hypotension (low blood pressure on standing), on the veins of the abdomen of patients with autonomic failure. The study will be conducted at Vanderbilt University Medical Center, and consists of 2 parts: a screening and 2 testing days.

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

The primary objective of this study is to measure the concentration and the regional brain distribution of activated brain microglia/macrophages using the PET radiopharmaceutical \[F-18\]DPA-714 in individuals with chronic pain and fatigue suspected to be associated with neuroinflammation. The PET tracer \[F-18\]DPA-714 binds to the 18 kDa translocator protein (TSPO, also known as the peripheral benzodiazepine receptor) in the mitochondria of activated microglia/macrophages and provides a non-invasive measure of neuroinflammation.

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

Background: In people with multiple sclerosis (MS), brain and cerebrospinal fluid (CSF) biomarkers indicate inflammation or disease. Researchers want to see if 4 drugs given alone or combined affect MS biomarkers.

📍 Stanford Multiple Sclerosis Center · Palo Alto, CA · Get directions →

A Study of Anti-CD19 Chimeric Antigen Receptor T Cell Therapy in Subjects with Non-relapsing and Progressive Forms of Multiple Sclerosis

📍 Brigham MS Center, 60 Fenwood Road · Boston, MA · Get directions →

This study aims to use \[C-11\]MRB PET (positron emission tomography) imaging to look at brain injury in patients with Multiple Sclerosis (MS) and healthy individuals. The overarching hypothesis is that there is decreased radioligand binding to the norepinephrine transporter in multiple sclerosis, reflecting injury to the noradrenergic system and that it plays a role in disease pathogenesis, its clinical manifestations and severity.

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

Our overall objective is to obtain an initial assessment of the potential value of using \[18F\]3F4AP for imaging demyelinating diseases such as multiple sclerosis: * Aim 1) Assess the safety of \[18F\]3F4AP in healthy volunteers and subjects with multiple sclerosis (MS). Hypothesis 1: Administration of \[18F\]3F4AP will result in no changes in vitals or other adverse events.

📍 University of Washington · Seattle, WA · Get directions →

Current disease-modifying therapies for multiple sclerosis (MS) aim to prevent the development of new lesions; unfortunately, no current FDA-approved therapies promote central nervous system (CNS) repair mechanisms. Thus, strategies to promote functional recovery from lesion-related deficits in adults with MS remain an unmet need.

📍 Sandler Neurosciences Building, Neurological Clinical Research Unit · San Francisco, CA · Get directions →

The clinical trial is intended to assess for clinical evidence of Clemastine Fumarate as a myelin repair therapy in patients with chronic inflammatory injury-causing demyelination as measured by multi-parametric MRI assessments. No reparative therapies exist for the treatment of multiple sclerosis.