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72 studies
Recruiting now, in plain language. Every study is watched over by an independent ethics board.
- Asthma
The Safety, Feasibility, and Repeatability of Inhaled ATP Cough Challenges
Testing whether inhaling ATP to trigger cough is safe and repeatable in people with asthma
📍 Respiratory Research Lab · Hamilton, Ontario · Get directions →
The inhalation of adenosine 5'-triphosphate (ATP) to evoke cough (ATP cough challenge) is becoming increasingly used as a tool to measure cough hypersensitivity in patients with chronic cough. However, the safety, feasibility, and repeatability of this procedure is not widely known.
- 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.
- 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.
- 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
- Asthma
A Study of CDX-622 in Participants With Mild to Moderate Asthma
Testing the safety and how a medicine affects the body in adults with mild to moderate asthma
📍 University of California, San Francisco · San Francisco, CA · +3 more US sites · Get directions →
This is a study to determine the safety, pharmacodynamics (PD), and pharmacokinetics (PK) of CDX-622 in adults with mild to moderate asthma.
- 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.
- Asthma
A Study to Learn How Different Amounts of the Study Medicine Called PF-08103402 Are Tolerated and Act in the Body in Healthy Adults or Adults With Mild To-moderate Asthma
Testing how different doses of a new medicine affect safety and the body in people with mild to
📍 Pfizer Clinical Research Unit - New Haven · New Haven, CT · Get directions →
The purpose of this study is to learn about the safety of a new study medicine called PF-08103402 in healthy adults (do not have disease) and or in adults with mild-to-moderate asthma. This is the first time the study medicine is being given to people.
- Crohn's / ulcerative colitis (IBD)
An Early-Stage Study in Multiple Clinics of How Afimkibart May Affect the Body's Processing of Medicines That Rely on Cytochrome P450 Enzymes in Participants With Ulcerative Colitis
Testing how afimkibart in people with ulcerative colitis affects drug processing in the liver
📍 Erick H. Alayo Medical Corporation - Gastro SB Clinic · Chula Vista, CA · +1 more US site · Get directions →
The purpose of this study is to evaluate the disease-drug-drug interaction (DDDI) potential of afimkibart (also known as RO7790121). This will be assessed by the characterization of the pharmacokinetics (PK) of cytochrome P450 (CYP) enzyme substrates alone and after administration of afimkibart in participants with moderately to severely active ulcerative colitis (UC).
- Crohn's / ulcerative colitis (IBD)
Autologous Stem Cell Transplantation for Crohn's Disease
Testing stem cell treatment for severe Crohn's disease in teens and adults
📍 UPMC Prebyterian- Adult Gastroenterology · Pittsburgh, PA · +1 more US site · Get directions →
The objective of this study is to evaluate the safety and effectiveness of administering high-dose chemotherapy followed by infusion of autologous CD34-selected peripheral blood stem cells (PBSC) in pediatric and adult patients with severe Crohn's disease.
- 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.
- Crohn's / ulcerative colitis (IBD)
An Open-Label, Proof of Consent Study of Vorinostat for the Treatment of Mdoerate-to-Severe Crohn s Disease and Maintenance Therapy With Ustekinumab
Testing vorinostat in people with moderate to severe Crohn’s disease and using ustekinumab to
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Crohn s disease (CD) is an inflammatory bowel disease. It causes inflammation of the gut.
- Crohn's / ulcerative colitis (IBD)
Treatment of Moderate to Severe Refractory Crohn's Disease
Testing different doses of a new treatment for Crohn’s disease
📍 Mayo Clinic Arizona · Scottsdale, AZ · +12 more US sites · Get directions →
This research study is testing an investigational research product called TRX103 as a possible treatment for individuals suffering from Crohn's Disease (CD). The primary purpose of this study is to learn how safe and effective different doses of TRX103 are when administered to individuals with CD.
- 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.
- 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).
- 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
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.
- Crohn's / ulcerative colitis (IBD)
R-3750 in Patients With Mild to Moderate Ulcerative Colitis
Testing the safety of an oral probiotic in people with mild to moderate ulcerative colitis
📍 Amicis Research Center · Valencia, CA · +4 more US sites · Get directions →
The goal of this study is to determine the safety and tolerability of orally taken probiotic (R-3750) in patients with mild to moderate ulcerative colitis. Patients will take an oral dosage of probiotic (R-3750) and provide patient-reported and physician scored measures of their colitis.
- 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.
- Crohn's / ulcerative colitis (IBD)
Fecal Microbiota Transplantation in an Expanded Ulcerative Colitis Population
Testing fecal transplant for ulcerative colitis in a feasibility study
📍 University of Calgary · Calgary, Alberta · Get directions →
This is a multi-centre, randomised controlled trial comparing fecal microbiota transplantation to placebo in an expanded ulcerative colitis population: a feasibility study (FRONTIER-UC) to determine whether a full-scale randomized controlled trial (RCT) to investigate fecal microbiota transplantation (FMT) in ulcerative colitis (UC) is feasible.
- 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.
- Crohn's / ulcerative colitis (IBD)
Beta-Hydroxybutyrate Feasibility Treating IBD
Testing whether taking a ketone supplement for 4 weeks is doable in people with Crohn’s disease
📍 University of Texas at Austin · Austin, TX · Get directions →
This clinical trial aims to understand the feasibility of patients taking ketone body supplement beta-hydroxybutyrate (BHB) for 4 weeks with a confirmed diagnosis of Crohn's disease and starting new therapy for active disease. The main questions it aims to answer are: * BHB supplementation will be feasible and acceptable to patients.
- Crohn's / ulcerative colitis (IBD)
Study of XmAb942 in Healthy Participants and Participants With Ulcerative Colitis
Testing the safety and effects of a drug called XmAb942 in ulcerative colitis
📍 Xencor Investigative Site · Scottsdale, AZ · +23 more US sites · Get directions →
Brief summary The Phase 1 study described herein will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of XmAb942 in healthy volunteers (Parts A and B). Part C of this study will be a Phase 2 study to evaluate XmAb942 in participants with ulcerative colitis (UC).
- 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.
- 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.
- Asthma
Phase 1 Study Evaluating Safety and Pharmacokinetics of ASY202 in Adults With Asthma
Testing safety and how a single inhaled dose works in adults with asthma
📍 Sun City Clinical Research · Glendale, AZ · +1 more US site · Get directions →
This is a phase 1 study, randomized, double-blind, placebo-controlled, 2 treatment, 2 period crossover study to evaluate the pharmacokinetics, safety, and tolerability of a single inhaled dose of ASY202 in adults with stable asthma. Following screening, eligible participants will be enrolled and randomized to one of two treatment sequences.
- 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.
- Crohn's / ulcerative colitis (IBD)
Low Dose IL-2 for the Treatment of Crohn's Disease
Testing low-dose interleukin-2 to find a safe dose for Crohn's disease
📍 Boston Children's Hospital · Boston, MA · +2 more US sites · Get directions →
The purpose of this study is to determine the safety and maximum effective dose (MED) of Interleukin-2 in subjects with moderate-to-severe crohn's disease.
- 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.
- Crohn's / ulcerative colitis (IBD)
Study to Evaluate the Fecal Microbiota Transplantation (FMT) in the Treatment of Ulcerative Colitis
Testing frozen stool transplant methods for people with active ulcerative colitis
📍 The University of Texas Health Science Center at Houston · Houston, TX · Get directions →
The study is to evaluate the safety, feasibility, and preliminary efficacy of frozen FMT delivery via retention enema compared to lyophilized powder given in oral capsules as induction FMT in subjects with active UC. This study will also determine changes in microbiome (diversity and genera) and proportion of antibody-coated microbiota from baseline to after completion of FMT.
- Multiple sclerosis (MS)
Central Nervous System Uptake of Anti-CD8+ T Cell Minibodies in Multiple Sclerosis and Progressive Multifocal Leukoencephalopathy
Testing whether anti–CD8 T cell medicines reach the brain and spinal cord in MS
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Multiple sclerosis (MS) and progressive multifocal leukoencephalopathy (PML) are disorders that affect the central nervous system (CNS). The CNS includes the brain, spinal cord, and optic nerves.
- Asthma
Pilot Study to Identify the Mediators and Inflammatory Cell Surface Receptors Involved in Allergic Airway Inflammation
Studying what body signals and immune cells drive asthma-related airway inflammation
📍 Massachusetts General Hospital · Boston, MA · Get directions →
Asthma is a heterogeneous disorder in which multiple potential inflammatory pathways contribute to airway obstruction. The biological basis for airway inflammation is the subject of intensive investigation.
- Dementia / Alzheimer'sMultiple sclerosis (MS)
PET Brain Imaging in Multiple Sclerosis, Alzheimer's Disease, and Other Neurological and Neuropsychiatric Diseases
Testing PET brain scans to measure immune cell activity in dementia
📍 Brigham MS Center, 60 Fenwood Road · Boston, MA · Get directions →
The specific aims of the study are: Primary: To determine the presence and regional distribution of microglial activation, as assessed by Fluorine-18 (18F) labeled "Peripheral Benzodiazepine Receptor 06" (PBR06) -PET, in subjects with active Relapsing Remitting Multiple Sclerosis (RRMS), Secondary Progressive Multiple Sclerosis (SPMS), and Alzheimer's Disease (AD) as compared to healthy controls Secondary: 1. To assess the relationship between microglial activation and clinical variables including disease severity and comorbidities (such as pain, fatigue and/or depression), as well as clinical MRI findings (such as lesions and atrophy) 2.
- Heart / cardiovascular diseaseHigh blood pressureCOPD (chronic lung disease)
: Vascular Function in Health and Disease
Studying how blood vessel function works in health and diseases like heart problems, high blood
📍 George E Wahlen VA Medical Center · Salt Lake City, UT · Get directions →
Many control mechanisms exist which successfully match the supply of blood with the metabolic demand of various tissues under wide-ranging conditions. One primary regulator of vasomotion and thus perfusion to the muscle tissue is the host of chemical factors originating from the vascular endothelium and the muscle tissue, which collectively sets the level of vascular tone.
- Multiple sclerosis (MS)
Targeting Residual Activity By Precision, Biomarker-Guided Combination Therapies of Multiple Sclerosis (TRAP-MS)
Testing how single or combined medicines affect MS biomarkers
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: In people with multiple sclerosis (MS), brain and cerebrospinal fluid (CSF) biomarkers indicate inflammation or disease. Researchers want to see if 4 drugs given alone or combined affect MS biomarkers.
- Multiple sclerosis (MS)
Molecular Imaging of NET Using [C-11]MRB-PET in MS
Using PET scans to study brain norepinephrine transporter injury in MS
📍 Brigham MS Center, 60 Fenwood Road · Boston, MA · Get directions →
This study aims to use \[C-11\]MRB PET (positron emission tomography) imaging to look at brain injury in patients with Multiple Sclerosis (MS) and healthy individuals. The overarching hypothesis is that there is decreased radioligand binding to the norepinephrine transporter in multiple sclerosis, reflecting injury to the noradrenergic system and that it plays a role in disease pathogenesis, its clinical manifestations and severity.
- Sickle cell disease
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
Testing a new transplant plan using immune-suppressing treatment before a blood stem cell transplant
📍 City of Hope Medical Center · Duarte, CA · Get directions →
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.
- COPD (chronic lung disease)
Hyper Polarized Xenon-129 MRI vs Xenon-133 Scintigraphy
Comparing a lung scan that uses xenon MRI with a radioactive scan for COPD
📍 University of Virginia Health System · Charlottesville, VA · Get directions →
Hyper polarized Xenon-129 MRI will be directly compared to a radioactive Xe-133 scintigraphy to detect defects in lung ventilation from airflow limitation. This study is conducted as a pilot study with intention to conduct a larger clinical trial.
- COPD (chronic lung disease)
Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure
Testing whether lung transplant first can improve lung function before bone marrow transplant for
📍 UPMC Presbyterian · Pittsburgh, PA · +1 more US site · Get directions →
The purpose of this study is to determine whether a lung transplantation prior to bone marrow transplantation (BMT) would allow for restoration of pulmonary function prior to BMT, allowing to proceed to BMT, to restore hematologic function.
- Sickle cell disease
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Testing whether using mismatched donor stem cells works for severe sickle cell disease and other
📍 Children's Hospital of Pittsburgh of UPMC · Pittsburgh, PA · Get directions →
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease.
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