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📍 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.

📍 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.

📍 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?

📍 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.

📍 Harbor UCLA Medical Center · Torrance, CA · Get directions →

The purpose of this research is to see if ketamine is effective and safe in treating children and young adults with sickle cell disease experiencing sickle cell related pain. In this study, we will compare the outcomes (such as pain scores) in persons who receive standard of care pain medicine (an opioid such as morphine) plus a low dose (amount) of ketamine to those who receive only standard of care pain medicine.

📍 Alabama Neurology Associates · Birmingham, AL · +14 more US sites · Get directions →

The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986368 in participants with Multiple Sclerosis Spasticity

📍 Profound Research, LLC · Carlsbad, CA · +8 more US sites · Get directions →

The main purpose of this study is to assess the bioequivalence of ocrelizumab SC test formulation to the marketed ocrelizumab SC reference formulation in participants with either relapsing multiple sclerosis (RMS) or primary progressive multiple sclerosis (PPMS). The study consists of 2 phases: a controlled phase, where participants in each group will receive one dose of test or reference formulation and a continuation phase, where all participants in both groups will receive ocrelizumab SC test formulation.

📍 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.

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

Background: Multiple sclerosis (MS) affects the brain, spinal cord, and optic nerves. MS lesions can appear on the MRI (magnetic resonance imaging) scans in many ways.

📍 The Hospital for Sick Children · Toronto, Ontario · Get directions →

This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat.

📍 Johns Hopkins University · Baltimore, MD · Get directions →

Fatigue is a prevalent and disabling symptom in Multiple Sclerosis (MS), affecting up to 90% of patients. Current treatments, including off-label prescriptions of wake-promoting agents, have shown limited effectiveness.

📍 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.

📍 Amerita · Centennial, CO · Get directions →

The goal is to assess the safety and effectiveness of home ocrelizumab infusion.

📍 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?

📍 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).

📍 Johns Hopkins University · Baltimore, MD · +1 more US site · Get directions →

The goal of this clinical trial is to evaluate if the study drug will reduce brain and retinal atrophy by reducing inflammation and subsequently slowing neurodegeneration in people with Multiple Sclerosis. The main outcome for the trial is change in normalized brain parenchymal volume (nBPV), measured by magnetic resonance imaging (MRI).

📍 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.

📍 Byers Hall · San Francisco, CA · Get directions →

The main purpose of this study is to assess whether hyperpolarized carbon imaging in relapsing remitting multiple sclerosis (MS) patients can be used to predict response to anti-CD20 disease modifying therapy. Study procedures will include magnetic resonance imaging (MRI) assessments with a hyperpolarized pyruvate sequence, clinical assessment as well as blood markers of disease progression.

📍 Inova Schar Cancer · Fairfax, VA · +1 more US site · Get directions →

This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.

📍 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.

📍 UCSF Benioff Children's Hospital Oakland · Oakland, CA · +5 more US sites · Get directions →

The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover?

📍 Masonic Cancer Center · Minneapolis, MN · Get directions →

A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.

📍 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.

📍 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.

📍 UBMD Neurolgy · Buffalo, NY · Get directions →

The goal of the study is to learn about treating older people with multiple sclerosis (MS) with metformin. Metformin may be used as a single therapy or as an add-on therapy.

📍 Memorial Sloan Kettering at Basking Ridge (Consent only) · Basking Ridge, NJ · +5 more US sites · Get directions →

Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD.

📍 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.

📍 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.

📍 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.

📍 University of Illinois Chicago, Sickle Cell Center · Chicago, IL · Get directions →

Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA.

📍 Children's National Hospital · Washington D.C., DC · Get directions →

This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies.

📍 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.

📍 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.

📍 Brigham and Women's Hospital · Boston, MA · Get directions →

Only subjects that have completed TILS-021, a Phase 2a Randomized, Double-Blind, Placebo-Controlled, Multicenter Dose-Ranging Study of Nasal Foralumab in Non-Active Secondary Progressive Multiple Sclerosis Patients are eligible to be enrolled in TILS-022. TILS-022 is a 6-month open-label extension study with an opportunity for dose to be escalated based on the subject's clinical status.

📍 University of Kansas Medical Center · Kansas City, KS · Get directions →

The incidence of insomnia is estimated to be as high as 90% in individuals with MS due to insomnia being underdiagnosed. Sleep disturbances in people with MS have been associated with reduced cognitive performance, physical function, psychological well-being, quality of life, and occupational function, as well as increased prevalence of fatigue, pain, depression, and anxiety.

📍 University of Kansas Medical Center- Sleep, Health and Wellness Laboratory · Kansas City, KS · Get directions →

The objective of this RCT is to assess the efficacy of one-on-one telehealth CBT-I (tCBT-I) compared to web-based CBT-I (wCBT-I) and treatment as usual (TAU) to improve sleep outcomes (Aim 1), fatigue and quality of life (Aim 2), and promote neuroprotection (Exploratory Aim 3), and to explore the characteristics of participants that predict improvement in sleep outcomes (Exploratory Aim 4). Reassessment of outcomes will be completed after the 6-week intervention and 6 months following completion of interventions.

📍 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.

📍 INECO Neurociencias Orono · Rosario, Santa Fe Province · Get directions →

This open label, single arm study will evaluate the PK and PD effects of fenebrutinib in children and adolescents with RMS aged between 10 and \< 18 years. This study consists of a Dose Exploration Period and an Optional Extension Period.

📍 Anaheim Clinical Trials · Anaheim, CA · +29 more US sites · Get directions →

This study is researching an experimental drug called alirocumab, referred to as "study drug". In the United States, alirocumab is approved for the treatment of hypercholesterolemia (high blood cholesterol levels).

📍 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.

📍 Boston Clinical Trials Inc - Internal Medicine · Boston, MA · +7 more US sites · Get directions →

The primary objective of this trial is to evaluate the safety and reactogenicity of mRNA-1195 in participants with multiple sclerosis.

📍 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).

📍 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.

📍 Alberta Children's Hospital · Calgary, Alberta · Get directions →

The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression.

📍 New York Medical College · Valhalla, NY · Get directions →

Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion. All other treatment is standard of care.

📍 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.

📍 Children's National Hospital · Washington D.C., DC · Get directions →

The goal of this clinical trial is to learn if intravenous citrulline works to treat acute pain in hospitalized patients with sickle cell disease. It will also learn about the safety of intravenous citrulline.

📍 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.

📍 UAMS Medical Center · Little Rock, AR · +4 more US sites · Get directions →

This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. The primary aim of the study is to assess the safety of anumigilimab in participants with SCD.

📍 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.

📍 Univ. of California, San Francisco Health Care · Oakland, CA · +15 more US sites · Get directions →

This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.

📍 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.

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

This early phase, open label, single arm clinical trial will determine the intraindividual safety, tolerability and effects of NPC1 (parthenolide and ipriflavone) on blood-based biomarkers of Alzheimer's disease (AD) pathology among adults with subjective cognitive decline, mild cognitive impairment, or Alzheimer's disease and objective indicators of seeding AD pathology

📍 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.

📍 UC Davis Comprehensive Cancer Center · Sacramento, CA · Get directions →

A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis

📍 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.

📍 University of California Los Angeles · Los Angeles, CA · +3 more US sites · Get directions →

This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS.

📍 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.

📍 University of Illinois at Chicago · Chicago, IL · Get directions →

The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v.

📍 Hospital for Special Surgery · New York, NY · +1 more US site · Get directions →

This treatment trial evaluates the addition of an anti-tumor necrosis factor-alpha drug, certolizumab, to usual treatment (a heparin agent and low-dose aspirin) in pregnant women with antiphospholipid syndrome (APS) and repeatedly positive tests for lupus anticoagulant (LAC) to determine if this regimen will improve pregnancy outcomes. All enrolled patients will receive certolizumab, and pregnancy outcomes will be compared to those of women with APS and repeatedly positive tests for LAC enrolled in a previous study by the investigators.