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135 studies
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- 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).
- Multiple sclerosis (MS)
Efficacy and Safety of Remibrutinib After Switching From Ocrelizumab in Participants Living With Relapsing Multiple Sclerosis.
Testing remibrutinib in people with relapsing multiple sclerosis who switch from ocrelizumab versus
📍 Perseverance Research Center · Scottsdale, AZ · +39 more US sites · Get directions →
The purpose of this Phase 3b study is to assess the efficacy, safety and tolerability of remibrutinib after switching from ocrelizumab and compared to continuous ocrelizumab treatment, in patients living with relapsing multiple sclerosis (plwRMS).
- Fatty liver disease (NASH/MASH)💵 May compensate
A Pivotal Clinical Study to Investigate Efimosfermin Alfa in Participants With Biopsy-confirmed F2- or F3-stage MASH
Testing how efimosfermin alfa compares with placebo for MASH with scarred liver tissue
📍 GSK Investigational Site · Arcadia, CA · +39 more US sites · Get directions →
The purpose of this study is to assess the safety and efficacy of efimosfermin alfa in the resolution of steatohepatitis and improvement of liver-related clinical outcome compared to placebo in individuals with MASH and biopsy-confirmed F2- or F3-stage fibrosis.
- 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)
Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of a Modified Regimen of Ublituximab
Testing a modified ublituximab treatment for relapsing multiple sclerosis
📍 TG Therapeutics Investigational Trial Site · Birmingham, AL · +34 more US sites · Get directions →
The primary purpose of this phase 3b study is to assess the efficacy of a modified regimen of ublituximab in participants with relapsing multiple sclerosis (RMS) as measured by T1 Gadolinium (Gd)-enhancing lesions in Part A; PK in Part B along with efficacy of ublituximab as measured by T1 Gd-enhancing lesions in participants who had a suboptimal experience on prior anti-CD20 therapy in Part C. The study consists of 3 parts: Part A is single-armed and open-label, Part B is randomized, double-blind, placebo-controlled, and Part C is single-armed and open-label.
- Lupus (SLE)
A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2a Study With an Open-Label Extension Evaluating the Efficacy and Safety of VENT-03 in Adult Participants With Active Cutaneous Lupus Erythematosus With or Without Systemic Lupus Erythematosus
Testing a treatment called VENT-03 for skin lupus, with or without body lupus
📍 Investigative Site · Beverly Hills, CA · +9 more US sites · Get directions →
The goal of this clinical trial is to learn if VENT-03 works to treat patients with cutaneous lupus erythematosus (CLE) who may or may not have systemic lupus erythematosus (SLE). Another goal is to learn about the safety of VENT-03 and how it is processed by the body.
- Fatty liver disease (NASH/MASH)
A Study of a Thyroid Hormone Receptor Beta Isoform (THRβ) Agonist and an Semicarbazide Sensitive Amine Oxidase (SSAO) Inhibitor, Alone and in Combination, in Adults With Presumed Metabolic Dysfunction-associated Steatohepatitis (MASH)
Testing drugs together or alone to lower liver fat in adults with MASH
📍 Arizona Liver Health · Chandler, AZ · +39 more US sites · Get directions →
The primary objective of this trial is to evaluate the dose-dependent and comparative effects of ECC4703 (low and high dose), ECC0509 (low and high dose), and their combination on hepatic fat reduction as assessed by change in magnetic resonance imaging proton density fat fraction (MRI-PDFF) at Week 12.
- Lupus (SLE)
CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Lupus
Testing a cell therapy made from a person’s own immune cells for lupus
📍 Children's Hospital of Philadelphia · Philadelphia, PA · Get directions →
This is a single-center, single-arm, open-label phase 1/2 study of CART19 in children and young adults with refractory Systemic lupus erythematosus (SLE), including both patients diagnosed with lupus nephritis (LN) and patients with non-renal Systemic lupus erythematosus (SLE). Phase 1 will evaluate the safety of CART19 in 6-12 patients with Systemic lupus erythematosus (SLE).
- 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.
- Lupus (SLE)
A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease (ELOWEN-2)
Testing enpatoran over 24 weeks for skin lupus symptoms, with or without whole-body disease
📍 Inland Rheumatology Clinical Trials, Inc. · Upland, CA · +7 more US sites · Get directions →
The purpose of this global, multicenter, Phase 3 study is to evaluate the efficacy and safety of enpatoran over 24 weeks in participants with active cutaneous manifestations of lupus erythematosus with or without systemic disease. Study details include: Study Duration: Up to 35 weeks.
- Sickle cell disease
Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults
Testing whether a low dose of ketamine helps treat sickle cell pain in children and young adults
📍 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.
- Sickle cell disease
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Testing rilzabrutinib for safety and effectiveness in sickle-cell disease
📍 University of Alabama at Birmingham- Site Number : 8400003 · Birmingham, AL · +15 more US sites · Get directions →
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B).
- Multiple sclerosis (MS)
A Study to Evaluate the Efficacy, Safety and Tolerability of BMS-986368 in Participants With Multiple Sclerosis Spasticity
Testing a drug called BMS-986368 for muscle stiffness in multiple sclerosis
📍 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
- Multiple sclerosis (MS)
A Study of Orelabrutinib in Patients With Primary Progressive Multiple Sclerosis
Testing a drug called orelabrutinib versus placebo in people with PPMS
📍 Arizona Neuroscience Research, LLC · Pheonix, AZ · +12 more US sites · Get directions →
Orelabrutinib is a CNS-penetrable BTK inhibitor. This is a phase 3, randomized, double-blind, parallel-group, multicenter study to evaluate the efficacy and safety of orelabrutinib compared with placebo in patients with PPMS.
- Lupus (SLE)
A Study to Test Whether Different Doses of BI 3000202 Help People With Systemic Lupus Erythematosus (SLE)
Testing whether different doses of BI 3000202 help adults with lupus
📍 Wallace & Lee Center · Beverly Hills, CA · +29 more US sites · Get directions →
This study is open to adults with systemic lupus erythematosus (SLE). The purpose of this study is to find out whether a medicine called BI 3000202 helps people with SLE.
- Multiple sclerosis (MS)
A Study to Assess Bioequivalence of Two Subcutaneous (SC) Formulations of Ocrelizumab in Participants With Multiple Sclerosis (MS)
Checking if two under-the-skin versions of ocrelizumab match in the body in people with multiple
📍 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.
- Fatty liver disease (NASH/MASH)
Impact of Soymilk on Liver Disease Severity of Children With Non-alcoholic Fatty Liver Disease (NAFLD)
Testing whether daily soy milk affects liver disease in children with fatty liver
📍 Cincinnati Children's Hospital Medical Center · Cincinnati, OH · Get directions →
A randomized, controlled study of standard soy milk consumption compared to 2% fat cow's milk consumption in children with Non-alcoholic Fatty Liver Disease (NAFLD). The investigators hypothesize that the daily consumption of soy isoflavones found in the soy milk will be beneficial in reducing NAFLD and other obesity-related comorbidities.
- 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.
- Multiple sclerosis (MS)
Effect of Corticosteroids on Inflammation at the Edge of Acute Multiple Sclerosis Plaques
Testing whether corticosteroids change inflammation in acute MS lesions
📍 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.
- Multiple sclerosis (MS)
Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)
Comparing stem cell transplant vs best available treatment for relapsing multiple sclerosis
📍 Stanford Multiple Sclerosis Center · Palo Alto, CA · +21 more US sites · Get directions →
This is a multi-center prospective rater-masked (blinded) randomized controlled trial of 156 participants, comparing the treatment strategy of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) to the treatment strategy of Best Available Therapy (BAT) for treatment-resistant relapsing multiple sclerosis (MS). Participants will be randomized at a 1 to 1 (1:1) ratio.
- Sickle cell disease
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Testing a once-daily mouth medicine in teens with sickle cell disease
📍 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.
- Fatty liver disease (NASH/MASH)
A Study Evaluating Efruxifermin in Subjects With Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis
Testing a drug to treat fat buildup liver disease in people with stage 2 or 3 fibrosis
📍 Akero Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →
This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with non-cirrhotic NASH/MASH and fibrosis stage 2 or 3 (F2 or F3). The study will enroll subjects in two cohorts for a total samples size of 1650 subjects.
- Lupus (SLE)
Study to Evaluate the Efficacy of Ruxolitinib 1.5% Cream in Adult Subjects with Discoid Lupus Erythematosus
Testing a 1.5% ruxolitinib cream for discoid lupus in adults
📍 INNO-6051 Site 03 · Fredericton, New Brunswick · Get directions →
This study is a single-blind, intraindividual study to evaluate the efficacy of ruxolitinib 1.5 % cream in adult subjects with discoid lupus erythematosus.
- Lupus (SLE)
The Pediatric Lupus Nephritis Mycophenolate Mofetil (PLUMM) Study
Testing two ways of giving mycophenolate to children with lupus kidney disease
📍 University of California, San Francisco · San Francisco, CA · +18 more US sites · Get directions →
The study is a 1-year 2-part double-blinded placebo controlled 2-arm clinical trial. Treatment arms are (1) MMF dosed as per body-surface area (MMFBSA; 600mg/m2 body surface area per dose about every 12 hours) and (2) pharmacokinetically-guided precision-dosing of MMF (MMFPK; MMF dosed twice daily to achieve an area under the concentration-time curve (AUC0-12h) of MPA \>60-70 mg\*h/L.
- Fatty liver disease (NASH/MASH)💵 May compensate
Non-Alcoholic Fatty Liver Disease, the HEpatic Response to Oral Glucose, and the Effect of Semaglutide (NAFLD HEROES)
Testing how the liver responds to glucose in fatty liver disease, and semaglutide’s effect
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: In non-alcoholic fatty liver disease (NAFLD), fat accumulates in the liver and can cause damage. Researchers want to learn what causes the damage NAFLD, and to see if a medication can help.
- Fatty liver disease (NASH/MASH)
A Trial to Investigate Safety, Exposure, and Efficacy of HU6 Compared With Placebo in Adult Participants With Metabolic Dysfunction-associated Steatohepatitis (MASH)
Testing how safe and effective HU6 is for adults with MASH
📍 Arizona Liver Health - Chandler · Chandler, AZ · +30 more US sites · Get directions →
Rivus Pharmaceuticals. Inc. is sponsoring this research study to assess the safety and tolerability of HU6 as a possible treatment for patients diagnosed with metabolic dysfunction-associated steatohepatitis (MASH). The study will also asse
- Lupus (SLE)
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
Testing the safety and effects of CABA-201 in people with active lupus
📍 University of California Irvine · Orange, CA · +20 more US sites · Get directions →
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
- Fatty liver disease (NASH/MASH)
A Study Evaluating the Efficacy and Safety of Pegozafermin in Participants With MASH and Fibrosis (ENLIGHTEN-Fibrosis)
Testing whether two doses of pegozafermin are safe and work for liver scarring in adults with MASH
📍 89bio Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →
The study will assess the efficacy and safety of 2 dose regimens of pegozafermin compared to placebo for the treatment of liver fibrosis stage F2 or F3 in adult participants with MASH.
- Lupus (SLE)
Phase 3 Extension Study to Evaluate Long-term Safety of Ianalumab in Participants With Systemic Lupus Erythematosus (SIRIUS-SLE Extension).
Testing long-term safety of ianalumab for people with lupus
📍 Pinnacle Research Group Llc · Anniston, AL · +20 more US sites · Get directions →
The purpose of this study is to evaluate long-term safety and tolerability of ianalumab in participants with systemic lupus erythematosus who have previously completed the treatment period in one of the two SIRIUS-SLE core studies (CVAY736F12301 or CVAY736F12302).
- Multiple sclerosis (MS)
Solriamfetol for the Treatment of Multiple Sclerosis Fatigue
Testing solriamfetol to treat tiredness from multiple sclerosis
📍 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.
- Fatty liver disease (NASH/MASH)💵 May compensate
Mechanisms of SGLT2 Inhibition in Pediatric Steatotic Liver Disease
Testing how SGLT2 inhibitors work for fatty liver disease in obese teens
📍 Ann & Robert H Lurie Children's Hospital of Chicago · Chicago, IL · Get directions →
This study is a randomized, double-blind, placebo-controlled trial specifically designed to evaluate the preliminary feasibility, initial efficacy and safety of SGLT2 inhibitors for treating NAFLD in adolescents with obesity.
- Multiple sclerosis (MS)
A Study of Orelabrutinib in Patients With Secondary Progressive Multiple Sclerosis
Testing orelabrutinib versus placebo for people with non-active secondary progressive multiple
📍 Arizona Neuroscience Research, LLC · Phoenix, AZ · +12 more US sites · Get directions →
Orelabrutinib is a CNS-penetrable BTK inhibitor. This is a phase 3, randomized, double-blind, parallel-group, multicenter study to evaluate the efficacy and safety of orelabrutinib compared with placebo in patients with non-active Secondary Progress MS.
- 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.
- Lupus (SLE)
Effects of Stopping Hydroxychloroquine in Elderly Lupus Disease
Testing what happens to older people with lupus if they stop their hydroxychloroquine
📍 University of California, Los Angeles · Los Angeles, CA · +9 more US sites · Get directions →
Hydroxychloroquine (HCQ) is a systemic lupus erythematosus (SLE) medication that has been very effective in reducing lupus disease activity and keeping patients stable with reduced symptoms. Despite a track record of safety with regard to infection compared to traditional immunosuppressive agents, the risk of HCQ retinal toxicity escalates with continued use.
- Multiple sclerosis (MS)
Study to Evaluate the Effectiveness and Safety of Ozanimod Compared to Fingolimod in Children and Adolescents With Relapsing Remitting Multiple Sclerosis
Testing ozanimod versus fingolimod in children with multiple sclerosis
📍 Local Institution - 0114 · Loma Linda, CA · +11 more US sites · Get directions →
The purpose of this study is to evaluate the effectiveness, safety, tolerability, drug levels and drug effects of ozanimod compared to fingolimod in children and adolescents with relapsing remitting multiple sclerosis (RRMS).
- 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?
- Lupus (SLE)
A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE
Testing a CAR T cell treatment for adults with hard-to-treat lupus
📍 Research Site · San Francisco, CA · +14 more US sites · Get directions →
This is a Phase 1b/2, single-arm, open-label, multi-center, clinical study of AZD0120, a CD19/BCMA dual CAR T cell therapy, to evaluate the safety, tolerability, and efficacy in adult participants with refractory Systemic Lupus Erythematosus.
- Fatty liver disease (NASH/MASH)💵 May compensate
A Study to Evaluate the Efficacy and Safety of Pegozafermin in Participants With Compensated Cirrhosis Due to MASH
Testing whether pegozafermin is safe and works for MASH-related liver cirrhosis
📍 89bio Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →
The study will assess the efficacy and safety of pegozafermin administered in participants with compensated cirrhosis due to MASH (biopsy-confirmed fibrosis stage F4 MASH \[previously known as nonalcoholic steatohepatitis, NASH\]).
- 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).
- Lupus (SLE)💵 May compensate
A Research Trial to Assess if Cenerimod is Efficacious and Safe to Treat Active Lupus Nephritis on Top of Regular Treatment
Testing whether cenerimod added to usual treatment helps adults with active lupus nephritis
📍 Alloy Clinical Research LLC · Kissimmee, FL · +10 more US sites · Get directions →
The goal of this clinical trial is to learn if cenerimod, on top of regular treatment, works to treat active lupus nephritis in adults with systemic lupus erythematosus and active lupus nephritis. It will also learn about the safety of cenerimod.
- Multiple sclerosis (MS)
Targeting Agonists of Glucagon-like Peptide-1 Receptor for Multiple Sclerosis
Testing a medicine to reduce brain and eye tissue loss in people with multiple sclerosis
📍 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).
- Fatty liver disease (NASH/MASH)💵 May compensate
A Study of Efimosfermin Alfa in Participants With Biopsy-confirmed Cirrhosis (Compensated) Due to MASH
Testing efimosfermin in people with MASH-related compensated cirrhosis
📍 GSK Investigational Site · Chandler, AZ · +29 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, preliminary efficacy, and pharmacokinetics (PK) of efimosfermin in participants with metabolic dysfunction associated steatohepatitis (MASH) and compensated cirrhosis consistent with stage F4 fibrosis.
- Lupus (SLE)
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Obinutuzumab in Adolescents With Active Class III or IV Lupus Nephritis and the Safety and PK of Obinutuzumab in Pediatric Participants
Testing a medicine in children and teens with active lupus kidney disease
📍 Loma Linda University health · Loma Linda, CA · +10 more US sites · Get directions →
This phase II, randomized, double-blind, placebo-controlled study is designed to evaluate the safety, efficacy and pharmacokinetics (PK) of obinutuzumab in adolescent participants (AP) aged 12 to less than 18 with biopsy-confirmed proliferative lupus nephritis (LN). It will also evaluate open label safety and PK of obinutuzumab in pediatric participants (PP), aged 5 to \<12 with LN.
- 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.
- Multiple sclerosis (MS)
Hyperpolarized Carbon Metabolic Imaging in Multiple Sclerosis
Testing whether special MRI using hyperpolarized carbon can predict how relapsing-remitting MS
📍 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.
- Lupus (SLE)
Phase I/II Study of AD-PluReceptor Plus Tafasitamab-cxix and Lymphodepleting Chemotherapy in Patients With Autoimmune Disorders
Testing the safety and best dose of an immune cell treatment with lupus drugs and chemotherapy
📍 MD Anderson Cancer Center · Houston, TX · +1 more US site · Get directions →
The goal of Safety Lead-In is to confirm the safety of tafasitamab when given to patients with SSc, SLE, and LN. The goal of Phase 1 is to find the recommended dose of AD-PluReceptor-NK cells in combination with tafasitamab and lymphodepleting chemotherapy that can be given to patients with the disease.
- Multiple sclerosis (MS)
A Study to Evaluate the Efficacy and Safety of Remibrutinib in Secondary Progressive Multiple Sclerosis
Testing how well and how safely remibrutinib works in secondary progressive multiple sclerosis
📍 Alabama Neurology Associates PC · Birmingham, AL · +32 more US sites · Get directions →
The purpose of this study is to provide efficacy and safety data for remibrutinib in patients with secondary progressive multiple sclerosis (SPMS)
- Sickle cell disease
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
Testing early heart screening and iron overload treatment in people with sickle cell disease
📍 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.
- Sickle cell disease
Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial
Testing two daily doses of zinc in people with sickle cell disease to see changes in bone-related
📍 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?
- Sickle cell disease
Allo HSCT for High Risk Hemoglobinopathies
Testing donor stem cell transplant for people with high-risk sickle cell
📍 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.
- Sickle cell disease
Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease
Testing a single pneumococcal vaccine dose in children with sickle cell disease
📍 Site # 8400006 · Atlanta, GA · +4 more US sites · Get directions →
The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV (licensed pneumococcal vaccine) when given as a single dose to children aged 2 to 17 years with sickle cell disease who had received or not a previous vaccination with pneumococcal conjugate or pneumococcal polysaccharide vaccine.
- Heart / cardiovascular diseaseLupus (SLE)
The Role of Anifrolumab in Improving Markers of Vascular Risk in Patients With Systemic Lupus Erythematosus (SLE) - IFN-CVD
Testing whether anifrolumab improves blood vessel risk markers in lupus
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: People with systemic lupus erythematosus (SLE) are at risk of developing complications in their blood vessels. This can increase the risk of heart attacks or stroke.
- Fatty liver disease (NASH/MASH)💵 May compensate
Phase 2a Study of Safety, Tolerability, and Efficacy of TLC-2716 in Subjects With Hypertriglyceridemia and NAFLD
Testing a medicine at two doses for safety and effects on fats in people with high triglycerides and
📍 OrsoBio Research Site · Guadalajara, Jalisco · Get directions →
This is a Phase 2a, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, and efficacy of 2 dose levels of TLC-2716 in subjects with hypertriglyceridemia and nonalcoholic fatty liver disease as assessed by changes in fasting triglycerides, liver steatosis by MRI, and other biomarkers.
- Lupus (SLE)
A Study of Rapcabtagene Autoleucel in Active, Refractory Systemic Lupus Erythematosus (SLE) or Lupus Nephritis (LN) Patients (AUTOGRAPH - SLE/LN)
Testing a one-time cell treatment plus chemotherapy to treat severe lupus
📍 University Of Alabama · Birmingham, AL · +18 more US sites · Get directions →
The purpose of this study is to evaluate the efficacy and safety of rapcabtagene autoleucel (administered once following lymphodepletion) in patients with active, refractory systemic lupus erythematosus (SLE) or active, refractory lupus nephritis (LN).
- Lupus (SLE)
A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease
Testing a treatment called enpatoran for skin lupus symptoms over 24 weeks
📍 Dermatology Research Associates - 300204025 · Los Angeles, CA · +10 more US sites · Get directions →
The purpose of this global, multicenter, Phase 3 study is to evaluate the efficacy and safety of enpatoran over 24 weeks in participants with active cutaneous manifestations of lupus erythematosus with or without systemic disease. Study details include: Study Duration: Up to 35 weeks.
- Multiple sclerosis (MS)
Metformin as an add-on or Monotherapy in Treatment of Aging People With Multiple Sclerosis (MS)
Testing metformin alone or with other treatment in older people with multiple sclerosis
📍 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.
- Lupus (SLE)
Obe-cel in Severe, Refractory Systemic Lupus Erythematosus (SLE) With Active Lupus Nephritis (LN)
Testing a one-time cell therapy approach for severe lupus with kidney involvement
📍 State University of New York Upstate Medical Center (SUNY) · Syracuse, NY · +1 more US site · Get directions →
The purpose of this trial is to evaluate the efficacy and safety of obecabtagene autoleucel (obe-cel) administered once following lymphodepletion in participants with severe, refractory systemic lupus erythematosus (SLE) and active lupus nephritis (LN).
- Lupus (SLE)Rheumatoid arthritisCrohn's / ulcerative colitis (IBD)
COVID-19 Booster and IIV Schedule in Immunocompromised Hosts
Testing COVID-19 booster and flu vaccine schedules in people with lupus, rheumatoid arthritis, or
📍 Maisonneuve -Rosemont Installation · Montreal, Quebec · Get directions →
The goal of this pragmatic embedded open-label, 2 x 2 factorial phase II randomized controlled trial is to evaluate strategies to improve COVID-19 booster and influenza vaccine immunogenicity in people living with immunocompromising conditions (PLIC). The main questions it aims to answer are: 1.
- Sickle cell disease
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Testing immune-suppressing treatment before stem cell transplant for people with sickle cell disease
📍 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.
- Sickle cell disease
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Testing long-term treatment with a new medicine for sickle cell or thalassaemia
📍 Univ of Alabama Birmingham · Birmingham, AL · +38 more US sites · Get directions →
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
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