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93 studies
Recruiting now, in plain language. Every study is watched over by an independent ethics board.
- 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.
- 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.
- 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
- 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.
- Lupus (SLE)Eczema / atopic dermatitis
Evaluating the Safety and Tolerability of Baricitinib in Patients With Job Syndrome With Lupus-Like Disease and/or Atopic Dermatitis
Testing whether baricitinib is safe in people with Job syndrome and lupus or eczema
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system. It can cause skin and lung infections and problems with blood vessels, connective tissues, and bones.
- 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.
- Lupus (SLE)
CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Lupus
Testing a cell therapy made from a person’s own immune cells for lupus
📍 Children's Hospital of Philadelphia · Philadelphia, PA · Get directions →
This is a single-center, single-arm, open-label phase 1/2 study of CART19 in children and young adults with refractory Systemic lupus erythematosus (SLE), including both patients diagnosed with lupus nephritis (LN) and patients with non-renal Systemic lupus erythematosus (SLE). Phase 1 will evaluate the safety of CART19 in 6-12 patients with Systemic lupus erythematosus (SLE).
- Lupus (SLE)
US Zamto-cel Autoimmune Diseases
Testing the safety of an experimental cell therapy for lupus
📍 Froedtert Hospital and the Medical College of Wisconsin · Milwaukee, WI · Get directions →
AID is a phase I multi-cohort study to assess the safety and tolerability of zamtocabtagene autoleucel (zamto-cel) in patients with refractory autoimmune diseases (SLE-Non renal, SLE-LN, SSc/dcSSc) after receiving standard therapy.
- 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.
- 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.
- 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.
- Lupus (SLE)
A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease
Testing the safety and early effects of a new treatment for lupus in adults
📍 Research Site 4 · Redwood City, CA · +6 more US sites · Get directions →
This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).
- 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.
- Lupus (SLE)
A Phase 1b Study of Budoprutug in Systemic Lupus Erythematosus (SLE)
Testing the safety and tolerance of budoprutug in adults with lupus
📍 Climb Bio Investigative Site #100104 · Allen, TX · +1 more US site · Get directions →
The main objective is to assess the safety and tolerability of budoprutug in adults with SLE. Pharmacokinetics, pharmacodynamics, and preliminary clinical efficacy will also be assessed.
- Lupus (SLE)Rheumatoid arthritis
A Study of CC-97540, CD-19-Targeted Nex-T CAR T Cells, in Participants With Severe, Refractory Autoimmune Diseases (Breakfree-1)
Testing a CD19-targeted CAR T cell treatment in people with severe lupus or rheumatoid arthritis
📍 University of Colorado Anschutz Medical Campus · Aurora, CO · +30 more US sites · Get directions →
The purpose of this study is to establish the tolerability, preliminary efficacy, and pharmacokinetics of CC-97540 in participants with severe, refractory autoimmune diseases (Breakfree-1).
- 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.
- 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.
- Lupus (SLE)
A Study of GSK5926371 in Participants With B-cell Driven Autoimmune Rheumatic Diseases (ARD)
Testing different doses of an experimental medicine for lupus
📍 GSK Investigational Site · Buenos Aires, · Get directions →
This is a 2-part study of GSK5926371 in participants with autoimmune rheumatic diseases (ARD). In part 1, participants will receive different doses of GSK5926371 to find a suitable priming dose.
- Lupus (SLE)
A Phase 1 Study of FT819 in B-cell Mediated Autoimmune Disease
Testing an immune treatment for safety and effects on B-cells in lupus
📍 Wallace Rheumatic Center · Beverly Hills, CA · +15 more US sites · Get directions →
This is a phase 1 study designed to evaluate the safety, pharmacokinetics (PK), and anti-B-cell activity of FT819 following treatment with or without auxiliary medicinal product (AMP) in participants with moderate-to-severe active systemic lupus erythematosus (SLE) with or without nephritis, antineutrophilic cytoplasmic antibody (ANCA)-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). The study will consist of a dose-escalation stage, followed by an expansion stage to further evaluate the safety and activity of FT819.
- Lupus (SLE)
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
Testing the safety and effects of CABA-201 in people with active lupus
📍 University of California Irvine · Orange, CA · +20 more US sites · Get directions →
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
- Lupus (SLE)
BAFF CAR-T Cells (LMY-920) for Systemic Lupus Erythematosus
Testing safety and best dose for CAR-T cell treatment in adults with lupus
📍 Nationwide Children's · Columbus, OH · Get directions →
This phase 1 study seeks to examine the safety and recommended phase 2 dose (RP2D) of BAFF-ligand CAR-T cells (LMY-920) in adult patients with refractory systemic lupus erythematosus (SLE). It is hypothesized that BAFF CAR-T cells will be safe and will improve SLE disease activity scores.
- 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.
- 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.
- 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.
- Lupus (SLE)
A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE
Testing a CAR T cell treatment for adults with hard-to-treat lupus
📍 Research Site · San Francisco, CA · +14 more US sites · Get directions →
This is a Phase 1b/2, single-arm, open-label, multi-center, clinical study of AZD0120, a CD19/BCMA dual CAR T cell therapy, to evaluate the safety, tolerability, and efficacy in adult participants with refractory Systemic Lupus Erythematosus.
- Lupus (SLE)
A Study to Assess Adverse Events, How the Drug Moves Through the Body and Effectiveness of Intravenous Infusions of ABBV-319 in Adult Participants With Systemic Lupus Erythematosus (SLE) and Sjogren's Disease (SjD)
Testing side effects and how an intravenous drug works in lupus and Sjogren’s
📍 Life Clinical Trials - Colonial Drive - Margate /ID# 276050 · Margate, FL · +3 more US sites · Get directions →
Systemic lupus erythematosus (SLE) is a chronic, systemic autoimmune disease characterized by B cell hyperactivity and Sjorgren's disease (SjD) is a chronic, multisystem autoimmune disease characterized by lacrimal and salivary gland inflammation, with resultant dryness of the eyes and mouth and occasional glandular enlargement. ABBV-319 exhibits potential B cell depletion in SLE and SjD which are characterized by B cell hyperactivity.
- 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).
- 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).
- Lupus (SLE)
REACT-01: Reversing Autoimmunity Through Cell Therapy
Testing a modified immune cell therapy for children and young adults with lupus
📍 Seattle Children's Hospital · Seattle, WA · Get directions →
This is a phase 1, open-label, non-randomized study enrolling pediatric and young adult research participants with treatment-refractory Systemic Lupus Erythematosus (SLE), to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to express CD19 specific chimeric antigen receptor (CAR) A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have their T cells collected. The T cells will then be bioengineered into a CAR T cell that targets circulating and tissue residing B cells.
- 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.
- Lupus (SLE)
Alnuctamab for Refractory SLE (LATTE Study)
Testing alnuctamab in people with moderate to severe lupus not helped
📍 Icahn School of Medicine at Mount Sinai · New York, NY · Get directions →
This study will assess the safety and preliminary efficacy of the bi-specific TCE, alnuctamab (known as BMS-986349, CC-93269, EM901), targeting BCMA in patients with moderate to severe SLE, refractory to standard-of-care treatments.
- 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.
- Lupus (SLE)
Phase I/II Study of AD-PluReceptor Plus Tafasitamab-cxix and Lymphodepleting Chemotherapy in Patients With Autoimmune Disorders
Testing the safety and best dose of an immune cell treatment with lupus drugs and chemotherapy
📍 MD Anderson Cancer Center · Houston, TX · +1 more US site · Get directions →
The goal of Safety Lead-In is to confirm the safety of tafasitamab when given to patients with SSc, SLE, and LN. The goal of Phase 1 is to find the recommended dose of AD-PluReceptor-NK cells in combination with tafasitamab and lymphodepleting chemotherapy that can be given to patients with the 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.
- 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.
- 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.
- Lupus (SLE)
A Study of GSK4527363 in Healthy Participants, Systemic Lupus Erythematosus (SLE) Participants, Healthy Chinese, and Japanese Participants and CTD-ILD Participants
Testing the safety and immune effects of a drug in people with lupus and related lung disease
📍 GSK Investigational Site · Scottsdale, AZ · +5 more US sites · Get directions →
This study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of GSK4527363 in healthy participants (Part A), participants with active SLE (Part B), healthy participants of Chinese and Japanese descent (Part C), and participants with interstitial lung disease associated with connective tissue disease (Part D)
- 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.
- 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.
- 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.
- 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.
- Lupus (SLE)
A Study to Investigate the Safety and Preliminary Efficacy of ALLO-329, an Allogeneic CAR T-cell Therapy, in Adults With Autoimmune Disease
Testing safety and early effects of a CAR T-cell treatment in adults with lupus
📍 Mayo Clinic · Phoenix, AZ · +12 more US sites · Get directions →
This is a first-in-human, single-arm, open-label study evaluating the safety, tolerability, and preliminary efficacy of ALLO-329 in adults with autoimmune diseases: systemic lupus erythematosus (SLE) with and without renal involvement, idiopathic inflammatory myopathy (IIM), and systemic sclerosis (SSc).The purpose of this trial is to evaluate the safety and tolerability of ALLO-329, an allogeneic anti-CD19, anti-CD70 dual chimeric antigen receptor (CAR) T cell therapy, in adults with autoimmune disorders, provide initial evidence of biological activity and clinical response to the treatment and determine the recommended Phase 2 regimen (RP2R).
- Lupus (SLE)
Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders
Testing the safety and effects of Descartes-08 in young people with lupus
📍 H01- Children's National Hospital · Washington D.C., DC · +1 more US site · Get directions →
Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis
- 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.
- 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.
- 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.
- Lupus (SLE)Rheumatoid arthritis
A Study to Assess Adverse Events, How the Drug Moves Through the Body and Effectiveness of Intravenous Infusions and Subcutaneous Injections of ABBV-519 in Adult Participants With Systemic Lupus Erythematosus (SLE) or Rheumatoid Arthritis (RA)
Testing side effects, how a medicine works in the body, and how well shots work in adults with lupus
📍 Pinnacle Research Group /ID# 278677 · Anniston, AL · +9 more US sites · Get directions →
Systemic lupus erythematosus (SLE) is a chronic, systemic autoimmune disease characterized by B cell hyperactivity. Rheumatoid Arthritis (RA) is a chronic inflammatory disease causing pain, stiffness, swelling and loss of joint function.
- Lupus (SLE)
Double-blind Placebo Controlled Study to Evaluate the Effect of NAD+ Boosting With Nicotinamide Riboside on Immunometabolism and Immunity in Systemic Lupus Erythematosus
Testing whether boosting NAD+ with a supplement affects immunity in lupus
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Study Description: Systemic lupus erythematosus (SLE) occurs predominantly in women and is driven by type I interferon dysregulation and neutrophil hyperresponsiveness. Neutrophils in females have reduced mitochondrial bioenergetic capacity which affects immunometabolism.
- 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.
- Lupus (SLE)Rheumatoid arthritis
A Clinical Study of MK-1045 in People With Lupus or Rheumatoid Arthritis (MK-1045-004)
Testing a study medicine for safety in people with lupus or rheumatoid arthritis
📍 Gold Coast University Hospital ( Site 1601) · Southport, Queensland · Get directions →
This study looks at a study medicine called MK-1045 in people with lupus and rheumatoid arthritis (RA). The main goal of the study is to learn about the safety of MK-1045 and if people tolerate it when they receive it at different dose levels (amounts).
- Dementia / Alzheimer's
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7812653 in Participants With Early Symptomatic Alzheimer's Disease (eAD)
Testing the safety and how a medicine affects the body in early 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.
- Lupus (SLE)
A Phase 1/2 Study of NKX019 in Subjects With Autoimmune Disease (Ntrust-1)
Testing safety of an immune-cell treatment for people with lupus
📍 Nkarta Investigational Site · Little Rock, AR · +16 more US sites · Get directions →
This is a Phase 1/2, open-label, multi-center, multi-cohort, non-randomized dose escalation and dose expansion basket study to determine the safety and tolerability of NKX019 (allogeneic CAR NK cells targeting CD19) in participants with autoimmune diseases.
- 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.
- 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.
- 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.
- Lupus (SLE)Rheumatoid arthritis
Open-label Single-Center Study to Evaluate the Safety and Efficacy of Combining Rituximab and AB-101 in B-cell Associated Autoimmune Diseases.
Testing safety and effects of AB-101 with rituximab in lupus and rheumatoid arthritis
📍 IRIS Research and Development, LLC · Plantation, FL · Get directions →
This study will evaluate the safety and activity of AB-101 in combination with rituximab in B-cell associated autoimmune diseases where rituximab is currently FDA approved (e.g., Rheumatoid Arthritis (RA), Pemphigus Vulgaris (PV), Granulomatosis with polyangiitis (GPA)/microscopic polyangiitis (MPA) as a therapeutic, or is recommended (e.g., in Systemic Lupus Erythematosus (SLE) as a cornerstone for disease management.
- Lupus (SLE)Rheumatoid arthritis
Study Evaluating Safety, Tolerability, PK/PD of Surovatamig in Adult RA or SLE Participants
Testing the safety and side effects of surovatamig in adults with RA or lupus
📍 Research Site · Birmingham, AL · +4 more US sites · Get directions →
This open-label, Phase I study will assess the safety and tolerability of surovatamig and characterise its PK and PD following subcutaneous administration to participants with RA or SLE.
- 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
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