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62 studies

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  • 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.

    ✓ Study care freePhase 1Ages 45+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Dementia / Alzheimer's

    A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease

    Testing the safety and how a drug works in people with Alzheimer’s

    📍 Clinical Trial Site · Montreal, · Get directions →

    The purpose of this study is to: * Evaluate the safety and tolerability of intrathecal (IT) ALN-5288 in patients with Alzheimer's Disease (AD) * Evaluate the pharmacodynamic (PD) and pharmacokinetic (PK) effects of ALN-5288 after dose administration

    ✓ Study care freePhase 1Ages 40–80Learn more →
  • Sickle cell disease

    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

    💵 May compensate

    📍 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.

    ✓ Study care freePhase 1Ages 18–90Learn more →
  • Dementia / Alzheimer's

    Rifaximin SSD in Dementia Trial

    Testing a new form of rifaximin to change gut microbes in people with dementia

    📍 Richmond VA Medical Center · Richmond, VA · Get directions →

    Using a new formulation of rifaximin, a non-absorbable antibiotic, to test if it can affect microbes in the gut of patients with dementia favorably.

    ✓ Study care freePhase 1Ages 65+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–55Learn more →
  • Dementia / Alzheimer's

    A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)

    Testing a gene therapy in people with a genetic form of frontotemporal dementia

    📍 The Ohio State University (OSU) Wexner Medical Center · Columbus, OH · +2 more US sites · Get directions →

    The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). FTD-GRN is an early-onset form of dementia, a progressive brain disorder that affects behavior, language and movement.

    ✓ Study care freePhase 1Ages 30–75Learn more →
  • Dementia / Alzheimer's

    SV2A & TSPO PET Imaging Measures to Reveal Mechanisms of HIV Neuropathogenesis During Antiretroviral Therapy

    Using brain scans to study how HIV affects the brain during treatment

    📍 Yale School of Medicne, Neuro ID Research Program · New Haven, CT · Get directions →

    The purpose of this study is to longitudinally characterize and evaluate changes in synaptic density in the brain using novel positron-emission tomography (PET) scans; magnetic resonance imaging (MRI), and clinical laboratory markers associated with HIV-related injury in the central nervous system. This study will test hypotheses relating to the presence and mechanisms of aberrant brain structure at the synaptic level in living humans with virologically controlled HIV on antiretroviral therapy.

    ✓ Study care freePhase 1Ages 18–80Learn more →
  • Dementia / Alzheimer's

    Exploration of an Online Education Program to Support Caregivers' Knowledge Transfer

    Testing an online program to help dementia caregivers share what they learn

    📍 Texas Christian University · Fort Worth, TX · +1 more US site · Get directions →

    The overarching goal of this work is to determine methods that improve caregiver education about the behavioral and psychological symptoms (BPSD) of dementia. The specific goal of this project is to extend our prior work to evaluate the impact of our educational intervention on caregivers' ability to transfer knowledge about the management of ADRD to real-world applied examples.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 7–21Learn more →
  • Dementia / Alzheimer's

    Ferritin and Iron Burden in SAH sIRB

    Testing how iron and ferritin levels relate to brain injury after ruptured aneurysms with dementia

    📍 Univesity of Iowa Hospital and Clinics · Iowa City, IA · +1 more US site · Get directions →

    Ruptured cerebral aneurysms lead to subarachnoid hemorrhage (SAH),that has a high morbidity and mortality rate, the severity of which is predicted by the "Hunt-Hess grade" (HHG). SAH leads to iron (Fe) and hemoglobin (Hb) accumulation in the brain, which is toxic for neurons.

    ✓ Study care freePhase 1Ages 18–75Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 1+Learn more →
  • Dementia / Alzheimer's

    A Trial Evaluating the Effect of NIO752 on Tau Synthesis Measured by a Process Known as SILK

    Testing a medicine in people with dementia to lower brain tau protein

    📍 Washington University in St. Louis · St Louis, MO · Get directions →

    This study will assess if drug (NIO752) reduces production of a protein, tau, by the brain. Normally tau maintains the internal skeleton of nerve cells.

    ✓ Study care freePhase 1Ages 21–80Learn more →
  • Dementia / Alzheimer's

    Clinical Study of SNK01 in Participants With Moderate Alzheimer's Disease

    Testing a treatment called SNK01 in people with moderate Alzheimer’s disease

    📍 Behavioral Research Specialists, LLC · Glendale, CA · +6 more US sites · Get directions →

    The goal of this clinical trial is to test SNK01 in participants with moderate Alzheimer's Disease. The main questions it aims to answer are: 1.

    ✓ Study care freePhase 1Ages 40–85Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 12–35Learn more →
  • Dementia / Alzheimer'sMultiple sclerosis (MS)

    Exploring the Utility of [18F]3F4AP for Demyelination Imaging

    Testing how an imaging tracer works in people with memory loss and multiple sclerosis

    📍 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).

    ✓ Study care freePhase 1Ages 18–90Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Dementia / Alzheimer's

    Study of ARO-MAPT-SC in Healthy Participants and Participants With Early Alzheimer's Disease

    Testing how a new medicine works in healthy adults and early Alzheimer’s disease

    📍 Research Site 3 · East York, Ontario · Get directions →

    Study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of ARO-MAPT-SC compared to placebo in adult healthy volunteers and in participants with early Alzheimer's disease (AD), defined as mild cognitive impairment due to AD and mild AD dementia.

    ✓ Study care freePhase 1Ages 18–80Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–24Learn more →
  • Dementia / Alzheimer's

    TREAD: Time Restricted Eating Intervention for Alzheimer's Disease

    Testing time-limited eating to see if it improves sleep and brain changes in early Alzheimer’s or

    📍 Shiley Marcos Alzheimer's Disease Center · San Diego, CA · Get directions →

    The goal of this clinical trial is to learn if restricting the time of eating to allow for prolonged fasting at night may reduce sleep disturbances, cognitive decay, and pathology in patients diagnosed with Mild Cognitive Impairment (MCI) or early to moderate Alzheimer's disease (AD). It will also learn about the feasibility of practicing 14 h of nightly fasting in this group of older adults.

    ✓ Study care freePhase 1Ages 60+Learn more →
  • Dementia / Alzheimer's

    A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)

    Testing the safety and effects of ACI-24.060 in Alzheimer’s and Down syndrome

    📍 Barrow Neurological Institute · Phoenix, AZ · +12 more US sites · Get directions →

    The purpose of this study is to assess the safety, tolerability, immunogenicity and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer's disease and in non-demented adults with Down syndrome.

    ✓ Study care freePhase 1Ages 35–85Learn more →
  • Dementia / Alzheimer's

    Nicotinic Acid for the Treatment of Alzheimer's Disease

    Testing how well extended-release niacin reaches spinal fluid in dementia

    📍 IU Health Neuroscience Center · Indianapolis, IN · Get directions →

    Increased dietary intake of niacin is correlated with reduced risk of Alzheimer's Disease and age-associated cognitive decline. The goal of this study is to collect data on the penetration of commercially available, FDA approved, extended-release niacin into the spinal fluid.

    ✓ Study care freePhase 1Ages 60–85Learn more →
  • Dementia / Alzheimer's

    A First-In-Human Study of LY3954068 in Participants With Early Symptomatic Alzheimer's Disease

    Testing whether a new drug is safe in people with early Alzheimer’s

    📍 K2 Medical Research, LLC · Maitland, FL · +5 more US sites · Get directions →

    The main purpose of this study is to evaluate the safety of LY3954068 in participants with early symptomatic Alzheimer's Disease (AD). The study will also investigate how much LY3954068 gets into the bloodstream and will test the effects of LY3954068 on markers of AD.

    ✓ Study care freePhase 1Ages 50–85Learn more →
  • Dementia / Alzheimer's

    Deep Repetitive Transcranial Magnetic Stimulation (rTMS) of the Precuneus for Alzheimer Disease (AD)

    Testing deep rTMS on a brain area for people with probable Alzheimer dementia to see if it is safe

    📍 UCLA TMS Clinical and Research Service · Los Angeles, CA · Get directions →

    The goal of this clinical trial is to learn if using deep repetitive transcranial magnetic stimulation (rTMS) targeting the precuneus is feasible, tolerable, and potentially efficacious for memory in Probable Alzheimer's Dementia. Previous work studying rTMS in Alzheimer's is mixed, but recent work studying rTMS of the precuneus is encouraging for both its short-term and long-term effects.

    ✓ Study care freePhase 1Ages 60–100Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 4–65Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 13–40Learn more →
  • Dementia / Alzheimer's

    A Study to Assess the Safety and Effects of ABBV-1758 Following Subcutaneous or Intravenous Injections in Participants With Alzheimer's Disease

    Testing the safety and effects of ABBV-1758 in people with Alzheimer’s disease given as injections

    📍 Irvine Center for Clinical Research /ID# 277752 · Irvine, CA · +10 more US sites · Get directions →

    Alzheimer's disease (AD) is a progressive, irreversible neurological disorder and is the most common cause of dementia in the elderly population. Clinical symptoms of the disease may begin with occasional forgetfulness such as misplacement of items, forgetting important dates or events, and may progress to noticeable memory loss, increased confusion and agitation, and eventually, loss of independence and non-responsiveness.

    ✓ Study care freePhase 1Ages 50–90Learn more →
  • Dementia / Alzheimer's

    Door-Through-Door Companion Rideshare Technology for Individuals With Alzheimer's Disease and Related Dementias (AD/ADRD)

    Testing a rideshare with a trained driver to help people with dementia get to medical visits

    📍 Onward Health HQ · San Leandro, CA · Get directions →

    The goal of this pilot randomized controlled trial is to assess the impact of D2D rideshare services with a trained companion driver on the rate of medical appointments for older adults and individuals with AD/ADRD. Participants will be assigned either door-through-door (D2D) rideshare or curb-to-curb (C2C) rideshare services.

    ✓ Study care freePhase 1Ages 60+Learn more →
  • Dementia / Alzheimer's

    AV-1980R (Tau Vaccine) in Preclinical Alzheimer's Disease (TAURUS-1980)

    Testing a vaccine for early Alzheimer’s disease biomarkers for safety

    📍 Comprehensive Center for Brain Health · Boca Raton, FL · +2 more US sites · Get directions →

    This is a Phase 1, multicenter, randomized, double-blind, placebo-controlled, multiple dose-escalating trial to evaluate the safety, tolerability, and immune response of AV-1980R, an investigational vaccine targeting tau protein, in participants with preclinical Alzheimer's disease. Up to 48 cognitively unimpaired adults aged 65-80 with biomarker evidence of early Alzheimer's disease will be enrolled into three ascending dose cohorts.

    ✓ Study care freePhase 1Ages 65–80Learn more →
  • Dementia / Alzheimer's

    📍 Montreal Neurological Institute Clinical Research Unit · Montreal, Quebec · Get directions →

    This study aims to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, and pharmacodynamics following administration of RO7812653 in participants with eAD.

    ✓ Study care freePhase 1Ages 50–75Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Dementia / Alzheimer's

    PET Imaging of Phosphodiesterase-4 (PDE4) in Volunteers With Alzheimer Disease (AD) or Mild Cognitive Impairment (MCI)

    Testing brain scans that measure PDE4 in people with dementia

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

    Background: About 5 million adults in the United States have age-related brain disorders. These include Alzheimer disease (AD), mild cognitive impairment (MCI), and other dementias.

    ✓ Study care freePhase 1Ages 50–100Learn more →
  • Dementia / Alzheimer's

    Interleukine-2 (IL-2) Plus Semaglutide in Alzheimer's Disease

    Testing a treatment with IL-2 and semaglutide in people with Alzheimer's

    📍 Houston Methodist Research Institute · Houston, TX · Get directions →

    Alzheimer's disease (AD) is the most common cause of dementia. Despite major research efforts, effective treatments that slow or stop disease progression remain limited.

    ✓ Study care freePhase 1Ages 50–86Learn more →
  • Dementia / Alzheimer's

    Low-Dose Radiotherapy for Alzheimer's Disease

    Testing low-dose whole-brain radiation safety in people with Alzheimer’s or dementia

    📍 Renaissance Institute of Precision Oncology & Radiosurgery · Winter Park, FL · Get directions →

    To evaluate the safety and tolerability of low-dose whole brain radiotherapy (WBRT) delivered as an induction course of 0.3 Gy × 10 fractions (3.0 Gy total) followed by 12 months of monthly maintenance LDRT (0.3 Gy × 10 fractions; 3.0 Gy total) in patients with Alzheimer's disease or dementia with inflammatory components

    ✓ Study care freePhase 1Ages 50+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 12–55Learn more →
  • 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.

    ✓ Study care freePhase 1Learn more →
  • Dementia / Alzheimer's

    UAB Alzheimer's Disease Center Core Cohort - Tau Imaging Substudy

    Studying brain tau in people with dementia using a PET scan

    📍 UAB · Birmingham, AL · Get directions →

    The primary objective of this study is to measure the concentration and the regional brain distribution of pathologic tau deposition using the PET tracer AV-1451 in participants in the UAB-ADC cohort. The amount and distribution of AV-1451 in the brain will be correlated to demographic, clinical, genetic, and biospecimen data acquired through the separate ongoing UAB-ADC study.

    ✓ Study care freePhase 1Ages 50+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Dementia / Alzheimer's

    A Study of LY4006895 in Healthy Participants With Early Symptomatic Alzheimer's Disease (AD)

    Testing the safety and side effects of a medicine in early Alzheimer’s

    📍 Collaborative Neuroscience Network - CNS · Los Alamitos, CA · +10 more US sites · Get directions →

    The main purpose of this study is to evaluate the safety and tolerability of the study drug known as LY4006895. Part A will administer a single-ascending dose in healthy participants or Part B will administer multiple-ascending doses in participants with early symptomatic Alzheimer's Disease (AD).

    ✓ Study care freePhase 1Ages 45–85Learn more →
  • Dementia / Alzheimer's

    A Trial to Test Intermittent Deep Brain Stimulation of Nucleus Basalis of Meynert to Treat Alzheimers.

    Testing an implanted brain stimulation procedure to treat dementia from Alzheimer’s

    📍 Wellstar MCG Hospital, Neurology Memory Clinic · Augusta, GA · Get directions →

    The purpose of this study is to test a new procedure to treat Alzheimer's disease. The procedure is called intermittent Deep Brain Stimulation (DBS) of the nucleus basalis of Meynert.

    ✓ Study care freePhase 1Ages 65–85Learn more →
  • Dementia / Alzheimer's

    A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

    Testing the safety and side effects of a medicine given into the spinal fluid for early-onset

    📍 Clinical Trial Site · La Jolla, CA · +1 more US site · Get directions →

    The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple doses of ALN-APP administered by intrathecal (IT) injection in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 8–17Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–80Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 12–45Learn more →
  • Dementia / Alzheimer's

    Innate Immunity Stimulation Via TLR9 in Early AD

    Testing a treatment that boosts the body’s immune response in early Alzheimer’s

    📍 NYU Langone Health · New York, NY · Get directions →

    This single-center, double-blind, placebo-controlled study will recruit in total 39 participants with either Mild Cognitive Impairment due to Alzheimer's disease (MCI) or Mild Alzheimer's disease dementia (mild AD). There will be 3 Dose levels.

    ✓ Study care freePhase 1Ages 60–85Learn more →
  • Dementia / Alzheimer'sMultiple sclerosis (MS)

    PET Brain Imaging in Multiple Sclerosis, Alzheimer's Disease, and Other Neurological and Neuropsychiatric Diseases

    Testing PET brain scans to measure immune cell activity in dementia

    📍 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.

    ✓ Study care freePhase 1Ages 18–85Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 1–30Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 5–40Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–99Learn more →
  • Dementia / Alzheimer's

    Repurposing Nucleoside Reverse Transcriptase Inhibitors for Treatment of AD

    Testing whether a daily emtricitabine pill affects Alzheimer’s dementia

    📍 Cedars-Sinai Medical Center · Los Angeles, CA · +1 more US site · Get directions →

    This is a randomized, double-blind clinical trial of a daily oral dose of 200 mg emtricitabine vs. placebo in 35 participants with biomarker-confirmed MCI or mild to moderate dementia due to Alzheimer's disease.

    ✓ Study care freePhase 1Ages 50–85Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 12–50Learn more →
  • 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

    ✓ Study care freePhase 1Ages 8–21Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Dementia / Alzheimer's

    A Clinical Trial of AAV2-BDNF Gene Therapy in Early Alzheimer's Disease and Mild Cognitive Impairment

    Testing a gene therapy that makes BDNF in the brain for early Alzheimer’s

    📍 University of California - San Diego · La Jolla, CA · +1 more US site · Get directions →

    This is a first-in-human clinical trial to test whether a protein administered into the brain continuously by gene therapy, Brain-Derived Neurotrophic Factor (BDNF), will slow or prevent cell loss in the brains of people affected by Alzheimer's disease and Mild Cognitive Impairment. The protein may also activate cells in the brain that have not yet deteriorated.

    ✓ Study care freePhase 1Ages 50–80Learn more →
  • Dementia / Alzheimer's

    Assess Fibrin in Brains With AD/ADRD

    Measuring brain fibrin in people with dementia using PET scans

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

    The goal of this project is to quantify brain fibrin content using 64Cu-FBP8-PET in the brains of subjects ranging from cognitively normal to clinically diagnosed with ADRD to evaluate potential regional differences.

    ✓ Study care freePhase 1Ages 55–90Learn more →
  • Dementia / Alzheimer's

    First-in-Human Evaluation of an Astrocytic Glutamate Transporter (EAAT2) PET Tracer in Dementia

    Testing a brain scan tracer to check safety and diagnose dementia

    📍 China Basin, UCSF · San Francisco, CA · Get directions →

    This is a first in human study that will assess the safety and diagnostic performance of \[18F\]RP-115 (fluorine-18 labeled RP115), a positron emission tomography (PET) agent. This agent has the potential to identify the early changes that occur in the brains of patients with Alzheimer's disease (AD) and frontotemporal dementia (FTD).

    ✓ Study care freePhase 1Ages 40–75Learn more →
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