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57 studies
Recruiting now, in plain language. Every study is watched over by an independent ethics board.
- Obesity / overweightHigh cholesterol
Evaluation of Milk Polar Lipids in Dyslipidemic Adults With Abdominal Obesity
Testing whether daily milk polar lipids affect heart and metabolic risk in adults with belly fat and
📍 University of Connecticut · Storrs, CT · Get directions →
The major objective of this project is to examine whether daily consumption of milk polar lipids (MPLs) influences cardiometabolic risk factors.
- Dementia / Alzheimer'sParkinson's disease
Phase 2 Trial of Tributyrin in People With Parkinson's Disease and Cognitive Impairments
Testing whether a tributyrin supplement helps memory, thinking, and balance in adults with Parkinson
📍 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.
- Parkinson's disease
Virtual tES Device Treatment for Parkinson's Disease Motor Symptoms
Testing a non-invasive brain stimulation device to improve Parkinson’s movement problems
📍 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.
- Sickle cell disease
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
Testing different doses of DISC-3405 to check safety in sickle cell disease
📍 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.
- Parkinson's disease
First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)
Testing an experimental treatment for early Parkinson’s disease in adults
📍 Montreal Neurological Institute and Hospital · Montreal, Quebec · Get directions →
This study is researching an experimental drug called ALN-SNCA (called "study drug"). The study is focused on people with early Parkinson's disease, a disorder of the nervous system that affects movement.
- Parkinson's disease
A Study to Evaluate Feasibility, Safety, and Clinical Responses of Implanting Autologous Peripheral Nerve Tissue Into the Brain for Non-motor or Motor Symptoms in Patients With Parkinson's Disease Undergoing DBS Surgery
Testing whether implanting a patient’s own nerve tissue during DBS surgery is safe in Parkinson’s
📍 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.
- Sickle cell disease💵 May compensate
Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
Testing higher doses of VAS-101 in people with stable sickle cell disease
📍 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.
- Dementia / Alzheimer'sParkinson's disease
PET Imaging Evaluation of [11C]SY08
Testing a PET scan tracer to detect alpha-synuclein protein clumps in dementia and Parkinson’s
📍 MGH · Charlestown, MA · Get directions →
The overall goal of the proposed research is to evaluate the use of \[11C\]SY08 as a PET radiotracer for aggregated alpha synuclein (αS) in individuals with Parkinson's disease (PD), Multiple system atrophy (MSA), Dementia with Lewy Bodies (DLB) and healthy controls. The purpose of this study is to evaluate the use of \[11C\]SY08 as a PET radiotracer for αS fibrils in individuals with PD, MSA, DLB and healthy controls.
- 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.
- Parkinson's disease
Evaluation of [¹⁸F]MODAG-009 PET Imaging in Synucleinopathies
Testing an injection for brain scans to study Parkinson’s disease and safety
📍 Institute for Neurodegenerative Disorders and XingImaging, LLC · New Haven, CT · Get directions →
This is a single-center, open-label clinical study designed to evaluate the imaging characteristics and safety of \[¹⁸F\]MODAG-009 in participants with Parkinson's disease (PD), Multiple system atrophy (MSA), and Healthy controls (HC). Approximately 13 participants will be enrolled in this study.
- 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.
- Parkinson's disease
Open-label Study to Evaluate Brain α-Synuclein Deposition Using PET and [18F]MK-0947 in Parkinson's Disease
Testing a new PET brain scan to measure nerve protein buildup in Parkinson’s
📍 Invicro (dba Perceptive) · New Haven, CT · +1 more US site · Get directions →
This clinical study is being conducted to learn more about a new imaging drug called \[18F\]MK-0947, which is designed to help doctors see changes in the brain related to Parkinson's disease (PD). PD is a condition that affects movement, balance, and thinking.
- 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.
- Parkinson's disease
A Study of LY4006896 in Healthy Participants and Participants With Parkinson's Disease
Testing an IV medicine in healthy people and people with Parkinson’s
📍 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.
- Parkinson's disease💵 May compensate
SAD Study in Patients With Parkinson's Disease and Motor Fluctuations
Testing a single dose of SER-252 in people with Parkinson’s disease
📍 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.
- 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.
- Parkinson's disease
Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell Exosomes for Parkinson's Disease
Testing the safety and effectiveness of nose drops made from donor stem cell exosomes for
📍 Medical Surgical Associates Center · St John's, · Get directions →
This trial will study the safety and efficacy of intranasal instillation of cultured allogeneic adult umbilical cord derived mesenchymal stem cell exosomes for the treatment of Parkinson's Disease
- Sickle cell disease
Gene Editing For Sickle Cell Disease
Testing whether gene editing is safe for people with sickle cell disease
📍 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.
- Parkinson's disease
A Pilot Study of In-lab Dynamic Standing in Parkinson's Disease
Testing a new adjustable standing desk for people with Parkinson’s
📍 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.
- Parkinson's disease
First-in-Human Single and Multiple Dose of HL-400
Testing an NLRP3 inhibitor taken by mouth for safety in healthy people
📍 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.
- Parkinson's disease
Using taVNS to Modulate Cardiovascular Function in Individuals With Neurologic Disease
Testing gentle ear nerve stimulation to see if it affects heart rate and blood pressure in people
📍 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.
- Parkinson's disease
Longitudinal TSPO PET Imaging With [18F]DPA-714 in PPMI (PPMI DPA-714 PET Imaging)
Studying how a brain imaging tracer binds in early Parkinson’s over 24 months
📍 UAB · Birmingham, AL · Get directions →
The overall goal of this protocol is to investigate \[18F\]DPA-714 binding in prodromal and early manifest Parkinson's Disease (PD) and to determine the baseline and change from baseline in \[18F\]DPA-714 binding in PD participants during a 24-month interval. Primary Objectives * To compare \[18F\]DPA-714 binding in prodromal and manifest PD and healthy volunteers.
- Parkinson's disease
Enrichment of Glutathione Using Gamma-glutamylcysteine Supplementation in Parkinson's Disease Patients.
Testing whether taking glutathione-building supplements helps early Parkinson’s patients
📍 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.
- 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.
- Sickle cell disease
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Measuring how long red blood cells last in sickle cell disease
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early.
- 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
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Testing how safe escalating doses of fostamatinib are in people with stable sickle cell disease
📍 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.
- Sickle cell disease
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Testing gene editing in stem cells for severe sickle cell disease
📍 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.
- Parkinson's disease
This Clinical Trial is Designed to Evaluate the Safety, Tolerability, Feasibility and Preliminary Efficacy of NouvNeu001 (Human Dopaminergic Progenitor Cells Injection) in Patients With Parkinson's Disease.
Testing an injected cell treatment for safety in people with advanced Parkinson’s
📍 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.
- Sickle cell disease
SCD Stem Cell Mobilization and Apheresis Using Motixafortide
Testing whether motixafortide is safe and can raise stem cells in people with sickle cell disease
📍 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.
- Parkinson's disease
Transplantation of Human iPS Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II)
Testing whether transplanting lab-made dopamine cells is safe for Parkinson’s
📍 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
- Parkinson's disease
Autologous suraL nervE Grafting to the Substantia nigrA in Patients With Synuclienopathies
Testing whether a nerve tissue graft can be safely placed in the brain in Parkinson’s or MSA
📍 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.
- Sickle cell disease
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Testing the safety and effects of panobinostat in adults with sickle cell disease
📍 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.
- Parkinson's disease
Title: A Multimodal Exercise Program With Gamification for Parkinson Disease (MEP+G)
Testing an 8-week exercise program with boxing and computer games for people with Parkinson disease
📍 Equilibrium Physical Therapy · Ventura, CA · Get directions →
In this pilot study the research will explore whether exercises and the outcome measures would be acceptable to conduct a more detailed research study. This 8 week pilot study will utilize a comprehensive exercise program that includes non-contact boxing along with computerized games for individuals with Parkinson Disease.
- Parkinson's disease
Noise-augmented Automatic Speech Recognition for Speech Treatment in Parkinson's Disease
Testing whether automatic speech recognition feedback improves speech clarity and social
📍 Teachers College, Columbia University · New York, NY · Get directions →
The goal of this clinical trial is to learn whether noise-augmented feedback using automatic speech recognition improves intelligibility and social participation in individuals with Parkinson's disease. The main questions the investigators will address are: Does noise-augmented feedback lead to increased intelligibility, as measured by transcription accuracy scores?
- 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.
- Parkinson's disease
A Clinical Trial of LY3962681 in Healthy Volunteers and in Patients With Parkinson's Disease
Testing how a Parkinson’s drug candidate affects safety and body handling in healthy people and
📍 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.
- Parkinson's disease
rTMS to Improve Cognition in Parkinson's
Testing whether noninvasive brain stimulation can improve thinking in people with Parkinson’s
📍 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.
- Dementia / Alzheimer'sParkinson's disease
[18F]F-DOPA Imaging in Patients With Autonomic Failure
Testing [18F]F-DOPA brain scans in people with autonomic failure
📍 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.
- Parkinson's disease
Treating Parkinson's Disease Through Transplantation of Autologous Stem Cell-Derived Dopaminergic Neurons
Testing surgery to transplant dopamine brain cells made from a patient’s own skin
📍 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.
- Sickle cell disease
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
Testing whether children with sickle cell disease can take daily ADHD medicine and monitoring its
📍 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.
- Dementia / Alzheimer'sParkinson's diseaseMultiple sclerosis (MS)
Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers
Testing safety of a PET imaging tracer in people with ALS, AD, MS, PD
📍 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.
- 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.
- Parkinson's disease
A Multicenter Study to Evaluate Safety, Tolerability, and Clinical Responses of DSP-1083 Into Subjects With Parkinson's Disease
Testing the safety and side effects of DSP-1083 implanted in people with Parkinson’s disease
📍 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.
- Obesity / overweightHeart / cardiovascular diseaseHigh cholesterol
Akkermansia Muciniphilia and Metabolic Side Effects of ADT
Testing how Akkermansia gut bacteria relate to body and blood lipid changes in people with obesity
📍 London Health Sciences Centre - Victoria Hospital · London, Ontario · Get directions →
The overriding objectives of this study are: 1. Primary outcomes: 1.
- Parkinson's disease
Multiple Objective Particle Swarm Optimization Postural Instability Gait Disorder
Testing how scans after deep brain stimulation help assess Parkinson’s gait
📍 University of Minnesota · Minneapolis, MN · Get directions →
Sixty patients will be enrolled in this study who are treated for Parkinson's disease (PD) with bilateral deep brain stimulation of subthalamic nucleus (STN) or globus pallidus (GP), who have a pre- operative 7 Tesla MRI including diffusion tensor imaging for tractography and a postoperative head CT for electrode localization, and in whom at least 3 months have passed since activation of their neurostimulators, for stabilization of clinical stimulator settings. Using their MRI and CT, the investigators will construct patient-specific models of electrical current spread to neuroanatomical tar- gets surrounding the electrode.
- Parkinson's disease
Effects of Midodrine and Droxidopa on Splanchnic Capacitance in Autonomic Failure
Testing midodrine and droxidopa effects on abdominal veins in Parkinsons
📍 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.
- Sickle cell disease
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
Testing a new transplant plan using immune-suppressing treatment before a blood stem cell transplant
📍 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.
- Parkinson's disease
The University of Alabama at Birmingham (UAB) Neuroinflammation in Parkinson's Disease-TSPO- Positron Emission Tomography (PET) Substudy
Testing brain inflammation in people with Parkinson’s using PET scans
📍 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.
- 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
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Testing how oral medicines may help people with high-risk sickle cell disease
📍 University of Illinois at Chicago College of Medicine · Chicago, IL · Get directions →
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
- Dementia / Alzheimer'sParkinson's disease
PET Imaging of Cyclooxygenases in Neurodegenerative Brain Disease
Testing brain scans to see inflammation enzymes in dementia and Parkinson’s
📍 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.
- Sickle cell disease
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Testing gene correction in a person’s own blood-forming stem cells for severe sickle cell disease
📍 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.
- High cholesterol
Liver Adiposity Effects on Pediatric Statin
Measuring how liver fat affects how children take and respond to statin
📍 Children's Mercy Hospital · Kansas City, MO · Get directions →
Single center, open-label, prospective investigation to quantify the effects hepatocellular fat has on hepatic statin transport and response in children and adolescents in obese and non-obese children and adolescents 8-21 years of age with normal, wild-type SLCO1B1 c.521TT genotype that are dosed rosuvastatin
- Sickle cell disease
PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD
Using PET scans to find blood vessel blockage markers in sickle cell disease
📍 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).