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102 studies
Recruiting now, in plain language. Every study is watched over by an independent ethics board.
- Kidney disease
A Study to Evaluate the Safety and Therapeutic Activity of GI-102 As a Single Agent and in Combination with Conventional Anti-cancer Drugs, Pembrolizumab or Trastuzumab Deruxtecan(T-DXd) in Patients with Advanced Solid Tumors (KEYNOTE-G08)
Testing the safety and tumor effects of GI-102 alone and with cancer drugs in people with advanced
📍 Mayo Clinic in Arizona · Scottsdale, AZ · +4 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and therapeutic activity of GI-102 as a single agent and in combination with conventional anti-cancer drugs, pembrolizumab or trastuzumab deruxtecan(T-DXd) over a range of advanced and/or metastatic solid tumors.
- 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
- Multiple sclerosis (MS)
Open-label, Multi-center, Phase I/II Study to Assess Safety, Disease Progression and Cellular Kinetics Following YTB323 Administration in Participants With Non-active Progressive Multiple Sclerosis (PMS)
Testing the safety and effects on disease and immune cells of one dose of YTB323 in people with
📍 Novartis Investigative Site · Darlinghurst, New South Wales · Get directions →
This is an open-label, multi-center, non-confirmatory study to assess the safety, disease progression, and cellular kinetics following YTB323 administration to 28 participants with non-active Progressive Multiple Sclerosis (PMS). The study design utilizes an ascending single dose design consisting of 3 sentinel cohorts followed by an expansion cohort.
- Multiple sclerosis (MS)
An Open-label Study of AZD0120 in Adults With Multiple Sclerosis
Testing safety of a new immune cell therapy in adults with multiple sclerosis
📍 Research Site · Tucson, AZ · +8 more US sites · Get directions →
This trial is a Phase 1b, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with Multiple Sclerosis.
- 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'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.
- Multiple sclerosis (MS)
A Study Evaluating the Safety and Efficacy of KITE-363 in Relapsed/Refractory Autoimmune Neurologic Diseases
Testing the safety and effects of KITE-363 in people with multiple sclerosis
📍 Stanford Neuroscience Health Center · Palo Alto, CA · +3 more US sites · Get directions →
This study will have two Phases: Phase 1a and Phase 1b. The goals of this clinical study are to learn more about the study drug KITE-363, by evaluating its safety, tolerability and efficacy in participants with relapsed/refractory autoimmune neurologic diseases.
- Multiple sclerosis (MS)
A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell ( CAR-T) Therapy in Subjects With Non-relapsing and Progressive Forms of Multiple Sclerosis
Testing a CD19 CAR-T cell treatment for progressive multiple sclerosis
📍 Stanford Multiple Sclerosis Center · Palo Alto, CA · Get directions →
A Study of Anti-CD19 Chimeric Antigen Receptor T Cell Therapy in Subjects with Non-relapsing and Progressive Forms of Multiple Sclerosis
- Kidney disease
A Study to Investigate the Safety, Tolerability, and Preliminary Efficacy With AION-301 Administered by Intravenous Infusion Compared With Placebo Administered by Intravenous Infusion Investigational Intervention in Participants Aged 35 to 75 Years of Age With Chronic Kidney Disease (CKD)
Testing the safety of an intravenous medicine in adults with chronic kidney disease
📍 SouthCoast Research Center, Inc. · Miami, FL · +1 more US site · Get directions →
The goal of this clinical trial is to learn about the safety (good or bad) of giving two AION-301 intravenous (IV) infusions, in adults with Stage 3 Chronic Kidney Disease (CKD). It will also help to learn if AION-301 reduces the symptoms of CKD and/or progression.
- 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.
- 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.
- Kidney disease
Intradialytic Exercise Combined With Creatine Monohydrate in Patients With Chronic Kidney Disease Undergoing Hemodialysis.
Testing whether exercise during dialysis and creatine help people with chronic kidney disease
📍 IMSS-Bienestar State Haemodialysis Centre · Colima, Coliima · Get directions →
This study aims to evaluate the effects of creatine monohydrate supplementation and an intradialytic exercise program on body composition, biochemical parameters, and functional capacity in patients with chronic kidney disease (CKD) undergoing maintenance hemodialysis. The study will compare the independent and combined effects of these interventions over an 8-week period to determine their potential benefits in this patient population.
- 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.
- Kidney disease
A Study of Eloralintide (LY3841136) in Participants With Renal Impairment and in Participants With Normal Renal Function
Testing how a diabetes medicine in the bloodstream changes in kidney disease
📍 Floridian Clinical Research, LLC · Miami Lakes, FL · +1 more US site · Get directions →
The purpose of the study is to assess the amount of Eloralintide (LY3841136) that reaches the bloodstream and the time it takes for the body to get rid of it when given to participants with renal (kidney) impairment and to healthy participants. The study drug will be administered subcutaneously (SC) (under the skin).
- 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.
- Multiple sclerosis (MS)
A Study to Investigate the Safety, Tolerability, and Processing by the Body of Intravenous and Subcutaneous RO7121932 Administration in Participants With Multiple Sclerosis
Testing how a new medicine given through a vein or under the skin is handled and tolerated in people
📍 Stanford University Medical Center · Stanford, CA · +4 more US sites · Get directions →
The primary purpose of the study is to evaluate the safety and tolerability of a single-ascending intravenous (IV) dose (Part 1), a single-ascending subcutaneous (SC) dose (Part 2), and multiple ascending SC doses (Part 3) of RO7121932 in participants with multiple sclerosis (MS).
- Multiple sclerosis (MS)
Treatment of Participants With Primary or Secondary Progressive Multiple Sclerosis
Testing whether TRX319 is safe for people with progressive multiple sclerosis
📍 University of Kansas Medical Center · Kansas City, KS · +1 more US site · Get directions →
The goal of this clinical trial is to treat male and female participants with two types of Multiple Sclerosis (MS) called primary progressive or secondary progressive MS. The main questions the trial aims to answer are the following: * Is TRX319 safe when administered to patients with progressive forms of MS?
- 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).
- Kidney disease
Inflammation REduction to Prevent cArdiovascular Injury in Renal Disease (REPAIR)
Testing a daily anti-inflammatory medicine to reduce heart and blood vessel injury in people with
📍 University of Alberta Hospital · Edmonton, Alberta · Get directions →
Phase 1 basket trial including 2 open-label single-arm cohorts: REPAIR CKD cohort and REPAIR Dialysis cohort. Open label colchicine 0.3 mg daily for 8 weeks followed, in patients who tolerated the 0.3 mg dose, by forced titration to 0.6 mg daily for 8 weeks.
- Multiple sclerosis (MS)
A Study to Evaluate the Safety, Tolerability, Efficacy, and Drug Levels of CC-97540 in Participants With Relapsing Forms of Multiple Sclerosis, Progressive Forms of Multiple Sclerosis or Refractory Myasthenia Gravis (MG) (Breakfree-2)
Testing a new drug’s safety and effects in people with multiple sclerosis
📍 Local Institution - 0011 · Birmingham, AL · +16 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG).
- Kidney disease
Study to Evaluate the Safety and Efficacy of the 10 GE Xenokidney in Patients With ESRD
Testing a GE xenokidney transplant in people with ESRD for safety and effectiveness
📍 Northwestern Memorial Hospital · Chicago, IL · +1 more US site · Get directions →
The purpose of this study is to evaluate the safety and efficacy of the 10 GE Xenokidney in patients with ESRD who are either not eligible for conventional allogeneic kidney transplantation (Group 1) or are on an Organ Procurement and Transplantation Network (OPTN) kidney transplant waitlist, but are more likely to die or go untransplanted within 5 years than receive a kidney transplant (Group 2). The study consists of xenotransplantation followed by a 24-week Post-transplant Follow up Period (Part A) to evaluate the efficacy and safety objectives followed by a Long-term Follow-up Period (Part B) to evaluate participant survival, 10 GE Xenokidney survival, and screening for zoonotic infections.
- 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).
- 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.
- 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.
- 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.
- Kidney disease
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of OJR520 in Healthy Volunteers and Participants With Chronic Kidney Disease
Testing the safety and side effects of OJR520 and how the body handles it in people with chronic
📍 Quotient Sciences Sea View · Miami, FL · Get directions →
The purpose of this first-in-human (FIH) study is to evaluate safety, tolerability, pharmacokinetic (PK) of OJR520.
- 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.
- Kidney disease
Study to Evaluate the Safety and Efficacy of the GGTA1 KO Thymokidney in Patients With ESRD
Testing a new kidney tissue transplant to treat end-stage kidney disease
📍 New York University Langone Health · New York, NY · Get directions →
The purpose of this study is to evaluate the safety and efficacy of the GGTA1 KO Thymokidney in patients with end-stage renal disease (ESRD) who are either not eligible for conventional allogeneic kidney transplantation (Group 1) or are on an Organ Procurement and Transplantation Network (OPTN) kidney transplant waitlist, but are more likely to die or go untransplanted within 5 years than receive a kidney transplant (Group 2). The study consists of xenotransplantation followed by a 24-week Post-transplant Follow-up Period (Part A) to evaluate the efficacy and safety objectives followed by a Long-term Follow-up Period (Part B) to evaluate participant survival, GGTA1 KO Thymokidney survival, and screening for zoonotic infections.
- Kidney disease
A Study to Investigate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZD4248 in Healthy Participants and Participants With Chronic Kidney Disease and Type 2 Diabetes and to Assess Home Measurements of Creatinine in a Non Interventional Cohort
Testing safety and drug effects of AZD4248 in people with kidney disease and diabetes, and home
📍 Research Site · Glendale, CA · +4 more US sites · Get directions →
This study will evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single ascending doses (SAD) and multiple ascending doses (MAD) of AZD4248 administered as an oral solution and intravenous (IV) infusion. Additionally, the study investigates the non-interventional feasibility of home measurement of serum creatinine in participants with diabetic kidney disease (DKD).
- Kidney disease
BZLF1 Peptide Vaccine (OSU-2131) With QS-21 for the Prevention of Epstein-Barr Virus Related Cancer in Patients Awaiting Solid Organ Transplants
Testing a vaccine to prevent Epstein-Barr virus cancer in transplant patients
📍 Ohio State University Comprehensive Cancer Center · Columbus, OH · Get directions →
This phase 1B trial tests the safety, side effects and best dose of rh-Hsc70- BZLF1 peptide complex (OSU-2131) with Stimulon (Trademark) QS-21 and evaluates how well it works in preventing Epstein-Barr virus (EBV) infection and related cancers in healthy volunteers and patients awaiting a solid organ transplant. Currently, patients who receive an organ transplant receive immune suppression therapy which can make it harder for the body to fight infections.
- Kidney disease
A Study to Assess the Safety and Pharmacokinetics of INCB123667 When Administered Orally to Adult Participants With Severe Renal Impairment or End Stage Renal Disease
Testing the safety and how an oral medicine moves through the body in adults with severe kidney
📍 Floridian Clinical Research · Miami Lakes, FL · +1 more US site · Get directions →
This study will be conducted to assess the safety and pharmacokinetics of INCB123667 when administered orally to adult participants with severe renal impairment or end stage renal disease.
- 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.
- Kidney disease
Breakthrough - T1DM and Chronic Kidney Disease
Testing an experimental treatment of Tegoprubart plus islet cells for diabetes and chronic kidney
📍 The University of Chicago · Chicago, IL · Get directions →
Single arm- subject treated with Tegoprubart and everolimus. The purpose of this research is to gather information on the safety and effectiveness of investigational regimen containing 2 experimental components: * An investigational drug called Tegoprubart and * Human pancreatic islet cells Both Tegoprubart and human pancreatic islet cells are considered investigational because they are not approved for use in the United States by the Food and Drug Administration (FDA).
- 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.
- Multiple sclerosis (MS)
Obe-cel in Refractory Progressive Forms of Multiple Sclerosis
Testing whether obe-cel is safe and has early effects in adults with progressive multiple sclerosis
📍 Stanford University · Redwood City, CA · Get directions →
The main purpose of this study is to evaluate if obe-cel is safe or causes any side effects in adults with refractory progressive MS. The study also plans to assess if obe-cel can show early signs of efficacy in MS.
- Multiple sclerosis (MS)
Assessing Changes in Multi-parametric MRI in MS Patients Taking Clemastine Fumarate as a Myelin Repair Therapy
Testing whether clemastine fumarate changes MRI signs of nerve repair in people with multiple
📍 Sandler Neurosciences Building, Neurological Clinical Research Unit · San Francisco, CA · Get directions →
The clinical trial is intended to assess for clinical evidence of Clemastine Fumarate as a myelin repair therapy in patients with chronic inflammatory injury-causing demyelination as measured by multi-parametric MRI assessments. No reparative therapies exist for the treatment of multiple sclerosis.
- Multiple sclerosis (MS)
Non-invasive Electrical Spinal Cord Stimulation To Restore Upper Extremity Function in Multiple Sclerosis
Testing non-surgical electrical stimulation to improve arm function in people with multiple
📍 University of Washington · Seattle, WA · Get directions →
Current disease-modifying therapies for multiple sclerosis (MS) aim to prevent the development of new lesions; unfortunately, no current FDA-approved therapies promote central nervous system (CNS) repair mechanisms. Thus, strategies to promote functional recovery from lesion-related deficits in adults with MS remain an unmet need.
- 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.
- 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.
- 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.
- Multiple sclerosis (MS)
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Pharmacodynamics of a New Subcutaneous Formulation of Ocrelizumab in Participants With Multiple Sclerosis
Testing the safety and dosing of a new under-the-skin ocrelizumab for multiple sclerosis
📍 Profound Research, LLC · Carlsbad, CA · +5 more US sites · Get directions →
The main purpose of this study is to evaluate the safety and tolerability of the ocrelizumab subcutaneous (SC) test formulation in participants with multiple sclerosis (MS). The study consists of two treatment phases: a dose-escalation and dose-continuation phase.
- 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
- 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.
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