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85 studies
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- COPD (chronic lung disease)
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
Testing how a genetic change in alpha-1 antitrypsin affects immune and lung responses to a pneumonia
📍 University of Alabama at Birmingham · Birmingham, AL · Get directions →
The goal of this study is to learn whether patients who have a genetic mutation in the genes that cause alpha 1 antitrypsin deficiency also have genetic variation in nearby genes that can increase risk for reduced immune function and respiratory infections. To investigate this hypothesis, we will compare immune responses to the 20-valent pneumococcal conjugate vaccine (PCV20, Pfizer) between participants who have one abnormal copy of the SERPINA1 gene and either no COPD exacerbations, vs those with 2 or more COPD exacerbations in the past year.
- 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.
- Fatty liver disease (NASH/MASH)
A Study to Evaluate ALN-CIDEB in Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease or With Metabolic Dysfunction-Associated Steatohepatitis (MASLD/MASH)
Testing an experimental medicine for MASLD or MASH in adults
📍 Arizona Liver Health · Chandler, AZ · +1 more US site · Get directions →
This study is researching an experimental drug called ALN-CIDEB, also referred to as "study drug". The study is focused on participants with metabolic dysfunction-associated steatotic liver disease (MASLD) (Part A) and metabolic dysfunction-associated steatohepatitis (MASH) (Part B).
- Fatty liver disease (NASH/MASH)
A Research Study of a Potential New Medicine (NNC4005-0001) for Liver Disease in Adult Participants With Increased Body Weight and Liver Fat
Testing a new medicine for liver disease in adults with extra body fat
📍 Altasciences Clinical Company, Inc · Montreal, Quebec · Get directions →
The purpose of this clinical study is to find out if NNC4005-0001 is well-tolerated and safe for people who have increased body weight and increased liver fat. Participants will receive either NNC4005-0001, which is the treatment being tested, or a placebo, which is a treatment that contains no active medicine.
- 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.
- Multiple sclerosis (MS)
Ublituximab (Briumvi) for Early Forms of Relapsing Multiple Sclerosis
Testing safety of a twice-yearly medicine for early relapsing MS
📍 Northwestern Memorial Hospital · Chicago, IL · Get directions →
In this prospective, open-label, single-arm, single-institution trial, the investigators will accomplish the following two aims: 1. study the safety and tolerability of Ublituximab (Briumvi) twice annually in participants with early MS over a treatment observation period of \~12 months.
- Type 2 diabetesFatty liver disease (NASH/MASH)
A Phase 1b Study of QX1206 in T2DM Patients With NAFLD
Testing QX1206 in people with type 2 diabetes and fatty liver
📍 Centricity Research Toronto LMC. · Toronto, · Get directions →
This is an open label phase 1b trial of QX1206 in patients with T2DM and with NAFLD. Laboratory tests and other measurements will be assessed prior to the first dose of study treatment and throughout the study to determine the recommended phase 2 dose.
- 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.
- 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)
Dalfampridine Combined With Physical Therapy for Mobility Impairment in Multiple Sclerosis
Testing whether combining a walking medication with physical therapy improves mobility in people
📍 MGH Institute of Health Professions · Boston, MA · Get directions →
The goal of this clinical trial is to evaluate if combining a medication that can help improve walking in people with multiple sclerosis (MS) with a physical therapy program is better for improving walking than either treatment alone. The main questions this study will answer are: * Does combining dalfampridine with physical therapy improve mobility more than physical therapy without concurrent dalfampridine?
- 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
- 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.
- COPD (chronic lung disease)Asthma
The Effects of Positive Airway Pressure on the Mucolytic Effects of NAC (TEAM)
Testing whether positive airway pressure helps a lung medicine (NAC) reach mucus plugs in asthma or
📍 UCSF Airway Clinical Research Center · San Francisco, CA · Get directions →
The goal of this clinical trial is to determine if positive pressure during inspiration will improve penetration of aerosolized N-Acetylcysteine (NAC) into airway mucus plugs in the lungs of patients with asthma or Chronic Obstructive Pulmonary Disease (COPD). The main questions it aims to answer are: * Does delivery of aerosolized NAC with positive inspiratory pressure have a greater effect on mucus plug burden in the lungs than delivery of NAC without positive pressure.
- 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.
- Obesity / overweightFatty liver disease (NASH/MASH)💵 May compensate
Human Models of Selective Insulin Resistance: Alpelisib, Part I
Testing how insulin works in healthy people versus people at risk for type 2 diabetes
📍 Columbia University Irving Medical Center · New York, NY · Get directions →
The goal of this clinical trial is to understand how the blood sugar-lowering hormone insulin works in healthy adults versus those who are at risk for type 2 diabetes. The study will use a drug called alpelisib, which interferes with insulin's actions in the body, to answer the study's main question: does the liver continue to respond to insulin's stimulation of fat production even when it loses the ability to stop making glucose (sugar) in response to insulin.
- 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.
- Obesity / overweightFatty liver disease (NASH/MASH)💵 May compensate
Fibrosis Lessens After Metabolic Surgery
Testing whether metabolic surgery can reduce liver scarring in obesity
📍 Banner Health Center · Phoenix, AZ · +3 more US sites · Get directions →
Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), a major global public health concern, is commonly associated with obesity, diabetes, and dyslipidemia. MASLD is currently the most common cause of chronic liver disease affecting about 80% of people with obesity, ranging from simple fat deposits in the liver to Metabolic Dysfunction-Associated Steatohepatitis (MASH), cellular injury, advanced fibrosis, cirrhosis, or hepatocellular carcinoma.
- 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)
Home Based Infusions for Ocrelizumab
Testing the safety and effectiveness of home infusions of ocrelizumab for multiple sclerosis
📍 Amerita · Centennial, CO · Get directions →
The goal is to assess the safety and effectiveness of home ocrelizumab infusion.
- 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).
- 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).
- 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.
- COPD (chronic lung disease)
A Study of BBT002 in Healthy Volunteers (HVs) and in Adult Patients With Chronic Obstructive Pulmonary Disease (COPD) or Chronic Rhiosininusitis With Nasal Polyps (CRSwNP)
Testing different doses of BBT002 in people with COPD and in healthy adults
📍 Equity Medical Bowling Green · Bowling Green, KY · +2 more US sites · Get directions →
Randomized study of single and multiple doses of BBT002 in healthy volunteers and in adult patients with chronic obstructive pulmonary disease (COPD) or chronic rhinosinusitis with nasal polyps (CRSwNP).
- Sickle cell disease
Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease
Testing whether routine red blood cell transfusions in pregnancy reduce sickle cell flare-ups or
📍 Grady Health System · Atlanta, GA · Get directions →
The goal of this study is to determine if there is a positive effect of prophylactic red blood cell (RBC) transfusion of leukoreduced, ABO, Rh (D/Cc/Ee) and Kell matched blood compared to standard of care on the number of episodes of acute sickle cell disease (SCD) manifestations or pregnancy-related complications requiring acute health care encounters (acute care/ER/Hospital visits) or resulting in death over the entirety of pregnancy until 2 months post-partum in women with SCD. RBC transfusion is the only disease-modifying therapy for pregnant women with SCD, and it is considered a standard treatment option however, there exists no consensus on the role of transfusion therapy in preventing SCD-related pregnancy complications.
- 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.
- Multiple sclerosis (MS)💵 May compensate
Prospective Evaluation of Sequencing From antiCD-20 Therapies to Ozanimod
Testing whether ozanimod is safe and works as a step-down treatment for stable multiple sclerosis
📍 University of Colorado Anschutz Medical Campus · Aurora, CO · +2 more US sites · Get directions →
A multi-center pilot study to evaluate safety and efficacy of ozanimod as de-escalation therapy in clinically stable MS patients previously treated with anti-CD20 therapy.
- Fatty liver disease (NASH/MASH)💵 May compensate
Evaluating the Pharmacokinetics and Safety of Miricorilant
Testing how a drug works in the body and its safety in adults with MASH
📍 Site# 433 · San Antonio, TX · Get directions →
A Phase 1b, Open-Label Study Evaluating the Pharmacokinetics and Safety of Miricorilant in Adult Patients With Presumed Metabolic Dysfunction-Associated Steatohepatitis (MASH)
- Fatty liver disease (NASH/MASH)
Bicalutamide Therapy in Young Women With NAFLD and PCOS
Testing bicalutamide in young women with fatty liver inflammation (NASH) and PCOS
📍 University of California San Francisco · San Francisco, CA · Get directions →
Nonalcoholic steatohepatitis (NASH), or fat-related liver inflammation and scarring is projected to be the leading cause of cirrhosis in the United States (U.S.) within the next few years. Women are at disproportionate risk for NASH, with approximately 15 million U.S.
- Multiple sclerosis (MS)
Study of Ublituximab for Ocrelizumab Wearing-Off in Multiple Sclerosis
Testing whether switching from ocrelizumab to ublituximab helps MS symptoms that return
📍 Johns Hopkins University · Baltimore, MD · Get directions →
The proposed study is a pilot study of ublituximab involving people with multiple sclerosis (MS) who are experiencing a "wearing off" phenomenon (return or worsening of MS-related symptoms) while being treated with ocrelizumab, and exploring whether switching to ublituzimab can resolve, improve or delay this phenomenon.
- COPD (chronic lung disease)💵 May compensate
Effect of the Inhaled Triple Therapies Over the Small Airway in Biomass Exposure
Comparing inhaled triple versus double medicines for COPD lung airways after wood smoke exposure
📍 Insituto Nacional de Enfermedades Respiratorias "Ismael Cosío Villegas" · Mexico City, Tlalpan · Get directions →
This phase IV randomized controlled clinical trial intend to compare the effect of three close standard inhaled triple therapies and one close standard inhaled double therapy on the small airway in patients with Chronic Obstructive Pulmonary Disease (COPD-B) and chronic bronchitis without obstruction (BCNO) exposed to wood smoke. The treatment phase duration is of 3 months.
- COPD (chronic lung disease)
Compare the Effects of Nebulizer Versus Inhaler Based Therapy for COPD Using Long-acting Bronchodilators
Comparing nebulizer and inhaler treatments for COPD symptoms
📍 The University of Tennessee Graduate School of Medicine · Knoxville, TN · Get directions →
The purpose of this study is to compare the effectiveness of inhaled bronchodilators delivered via nebulizers vs. dry powder inhalers (DPIs) in symptomatic participants with Chronic Obstructive Pulmonary Disease (COPD) who have airflow obstruction (FEV1/FVC ≤ 70%) and show significant air trapping (RV ≥ 120% of predicted).
- COPD (chronic lung disease)
A Study to Test the Safety and Effects of Inhaled GDC-6988 in Participants With Muco-obstructive Disease
Testing the safety and effects of an inhaled medicine for COPD
📍 University of Alabama at Birmingham · Birmingham, AL · +6 more US sites · Get directions →
This study evaluates the safety, tolerability, and activity of inhaled GDC-6988 in participants with muco-obstructive disease.
- Obesity / overweightHigh blood pressureCOPD (chronic lung disease)
Feasibility of Semaglutide in Advanced Lung Disease
Testing whether semaglutide can be given safely to people with advanced lung disease
📍 University of Pennsylvania Perelman School of Medicine · Philadelphia, PA · Get directions →
The goal of this clinical trial is to learn whether semaglutide, an FDA-approved treatment for diabetes and obesity, is feasible and tolerable in patients with advanced lung disease. The main question\[s\] it aims to answer are: 1.
- Sickle cell disease
A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease
Testing how BMS-986470 affects the body and safety in sickle cell disease
📍 University of Alabama at Birmingham · Birmingham, AL · +18 more US sites · Get directions →
The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.
- Multiple sclerosis (MS)
Glucose Consumption in MS Using [F-18]FDG-PET
Testing how [F-18]FDG PET shows glucose use in people with MS and controls
📍 Brigham & Women's Hospital · Boston, MA · Get directions →
Given the need for better diagnostic imaging techniques in multiple sclerosis (MS), the study aims to investigate the utility of \[F-18\]FDG positron emission tomography (PET) in MS. The study will be assessing glucose consumption patterns in subjects with progressive MS (PMS) and relapsing-remitting MS (RRMS), as well as healthy controls.
- 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.
- Multiple sclerosis (MS)
Silodosin for Urinary Symptoms in Female Patients With Multiple Sclerosis
Testing whether silodosin helps lower urinary tract symptoms in women with multiple sclerosis
📍 Atlantic Health · Morristown, NJ · Get directions →
The goal of this clinical trial is to learn if silodosin works to treat lower urinary tract symptoms (LUTS) in female patients with multiple sclerosis (MS). It will also learn about the effect of the drug on voiding parameters.
- 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.
- Multiple sclerosis (MS)
ELIOS - Investigational Biomarkers to Track Disease Modification in Active RRMS
Testing eye-tracking measures to track disease changes in relapsing-remitting multiple sclerosis
📍 Novartis Investigative Site · Calgary, Alberta · Get directions →
The exploratory ELIOS study aims to assess the value of novel investigational Eye Movement Biomarkers (EMBs) in tracking disease-related changes among a real-world cohort of Canadian patients with active RRMS, within the context of disease-modifying treatment (i.e., ofatumumab). To that end, the study will use the patented investigational, Eye Tracking Neurological Assessment (ETNA-ProgMS) SaMD (v1.0.11 or later), which has not yet received Health Canada approval, to reliably and accurately track eye movements with precision.
- 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.
- Sickle cell disease
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
Testing different ways to use hydroxyurea for treating sickle cell anemia
📍 Cincinnati Children's Hospital Medical Center · Cincinnati, OH · Get directions →
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for people with SCA.
- 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.
- 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.
- Sickle cell disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Testing how a new medicine works in healthy people and sickle cell patients
📍 University of Alabama Birmingham · Birmingham, AL · +5 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).
- Sickle cell disease
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Testing a bone marrow transplant with lower-intensity treatment for kids with sickle cell and
📍 Yale School of Medicine · New Haven, CT · +3 more US sites · Get directions →
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
- Multiple sclerosis (MS)
Assessment of Neuroinflammation in Central Inflammatory Disorders Using [F-18]DPA-714.
Testing a brain scan using a PET tracer to measure inflammation cells in people with multiple
📍 University of Alabama at Birmingham Medical Center · Birmingham, AL · Get directions →
The primary objective of this study is to measure the concentration and the regional brain distribution of activated brain microglia/macrophages using the PET radiopharmaceutical \[F-18\]DPA-714 in individuals with chronic pain and fatigue suspected to be associated with neuroinflammation. The PET tracer \[F-18\]DPA-714 binds to the 18 kDa translocator protein (TSPO, also known as the peripheral benzodiazepine receptor) in the mitochondria of activated microglia/macrophages and provides a non-invasive measure of neuroinflammation.
- Multiple sclerosis (MS)
A Phase I Double Blind Study of Metformin Acting on Endogenous Neural Progenitor Cells in Children and Young Adults With Multiple Sclerosis
Testing whether starting metformin at different times is feasible in youth with multiple sclerosis
📍 The Hospital for Sick Children · Toronto, Ontario · Get directions →
A randomized multiple baseline feasibility trial where participants will start taking metformin at one of 3 randomly determined points (3-months, 6-months or 9 months) during the 12-month trial. All subjects will be on a daily dose of metformin for a minimum of 3 months and a maximum of 9 months.
- Sickle cell disease
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Testing an injectable birth control shot to reduce pain in women with sickle cell disease
📍 Emory University · Atlanta, GA · +1 more US site · Get directions →
This research is being conducted to see if using an injectable contraception, Depot Medroxyprogesterone Acetate (Depo-Provera), can reduce the pain experienced by women with sickle cell disease. Participants in this study will be adult women with sickle cell disease who regularly experience sickle cell pain.
- Multiple sclerosis (MS)
Ocrelizumab Discontinuation in Relapsing Multiple Sclerosis
Testing stopping ocrelizumab in people with relapsing multiple sclerosis
📍 Yale School of Medicine · New Haven, CT · +12 more US sites · Get directions →
This study is a prospective, multi-center, randomized, double blinded, placebo-controlled study of OCR treatment-discontinuation in patients with early RMS. All eligible participants will be initiated on OCR using the standard approved administration schedule of two 300 mg infusions separated by 14 days (i.e., Days 0 and 14) for a total of 600 mg, followed by 600 mg infusions at Month 6,12, 18, and 24.
- Sickle cell disease
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
Testing whether children with sickle cell disease can take daily ADHD medicine and monitoring its
📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →
The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork problems and if they have any side effects. The study will assess any thinking or attention problems participants may have both before taking this drug and after.
- Dementia / Alzheimer'sParkinson's diseaseMultiple sclerosis (MS)
Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers
Testing safety of a PET imaging tracer in people with ALS, AD, MS, PD
📍 UCSF · San Francisco, CA · +2 more US sites · Get directions →
This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs. 18F-OP-801 is intended as a biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation.
- Sickle cell disease
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Testing if adding ruxolitinib lowers graft failure after transplant in children with sickle cell
📍 Children's Hospital of Colorado · Aurora, CO · +3 more US sites · Get directions →
This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants.
- Fatty liver disease (NASH/MASH)
A Study of Efimosfermin Alfa in Adults With Hepatic Impairment
Testing how a single dose affects drug levels and safety in adults with liver impairment and
📍 GSK Investigational Site · Rialto, CA · +2 more US sites · Get directions →
This study is designed to study the pharmacokinetic (PK) and safety profiles of a single dose of efimosfermin alfa in participants with varying degrees of Hepatic Impairment (HI) (assessed by Child-Pugh score) due to steatotic liver disease, with and without significant alcohol consumption.
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