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119 studies
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- 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.
- Lupus (SLE)
Safety and Efficacy of Combination Belimumab and Voclosporin in the Treatment of Proliferative Forms of Lupus Glomerulopathy: Synergy Trial
Testing whether adding two lupus medicines to standard care improves kidney results
📍 AARA Clinical Research · Glendale, AZ · +6 more US sites · Get directions →
This is a Phase IV, open-label, randomized trial to determine whether the combination of Belimumab (BEL) and Voclosporin (VCS), plus background therapy with Mycophenolate Mofetil (MMF), improves the proportion of patients with proliferative lupus nephritis achieving complete renal response (CRR) compared to proportion of patients achieving CRR from recent clinical trials. This protocol will additionally determine whether combination therapy using Belimumab (BEL) and Voclosporin (VCS) facilitates rapid discontinuation of MMF.
- Heart / cardiovascular diseaseKidney disease
A Study of Orforglipron (LY3502970) on Cardiovascular Outcomes in Adults With Atherosclerotic Cardiovascular Disease and/or Chronic Kidney Disease (ATTAIN-Outcomes)
Testing whether orforglipron changes heart outcomes in adults with heart disease and/or kidney
📍 Instituto de Investigaciones Clínicas Bahia Blanca · Bahía Blanca, · Get directions →
The purpose of this study is to measure cardiovascular outcomes with orforglipron compared with placebo in participants with atherosclerotic cardiovascular disease (ASCVD) and/or chronic kidney disease (CKD). Participation in the study will last about 5 years.
- 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.
- Lupus (SLE)Eczema / atopic dermatitis
Evaluating the Safety and Tolerability of Baricitinib in Patients With Job Syndrome With Lupus-Like Disease and/or Atopic Dermatitis
Testing whether baricitinib is safe in people with Job syndrome and lupus or eczema
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system. It can cause skin and lung infections and problems with blood vessels, connective tissues, and bones.
- 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).
- Lupus (SLE)
US Zamto-cel Autoimmune Diseases
Testing the safety of an experimental cell therapy for lupus
📍 Froedtert Hospital and the Medical College of Wisconsin · Milwaukee, WI · Get directions →
AID is a phase I multi-cohort study to assess the safety and tolerability of zamtocabtagene autoleucel (zamto-cel) in patients with refractory autoimmune diseases (SLE-Non renal, SLE-LN, SSc/dcSSc) after receiving standard therapy.
- Lupus (SLE)
Dapagliflozin in Active Lupus Nephritis
Testing dapagliflozin for lupus-related kidney inflammation (kidney disease)
📍 Massachusetts General Hospital · Boston, MA · +1 more US site · Get directions →
Lupus nephritis is a chronic and life-threatening autoimmune cause of kidney disease that predominately impacts young people and can lead to kidney failure. Sodium-glucose co-transporter-2 inhibitors, including dapagliflozin, are known to improve outcomes for people with other causes of chronic kidney disease.
- Type 2 diabetesHigh blood pressure
Polypill for Prevention of Cardiomyopathy
Testing a pill combination to prevent heart muscle disease in people with type 2 diabetes and high
📍 UT Southwestern Medical Center · Dallas, TX · Get directions →
This study will investigate the utility of a polypill-based strategy for patients with type 2 diabetes mellitus and high risk of heart failure (HF), as assessed via the WATCH-DM risk score. Polypill therapy will consist of empagliflozin 12.5 mg, losartan 25, 50 or 100 mg, and finerenone 10 mg daily.
- 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.
- High blood pressure💵 May compensate
Arterial Ablation for the Treatment of Type 2 Diabetes Mellitus and Its Comorbidities
Testing a device used through a blood-vessel procedure for type 2 diabetes and high blood pressure
📍 Hospital Paitilla Clinicas y Hospitales · Panama City, · Get directions →
This study is assess the safety and performance of the Neurotronic Infusion catheter for treatment of patients with Type 2 Diabetes (T2DM) and hypertension.
- 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.
- Obesity / overweightHigh blood pressure
A Study of LY3971297 in Healthy Participants
Testing side effects and how the body processes an injection in healthy and obese people with high
📍 CenExel ACT · Anaheim, CA · +2 more US sites · Get directions →
The purpose of this study is to measure side effects of LY3971297 injection administered under the skin in healthy participants and obese participants with high blood pressure (BP). Blood tests will be performed to check how much LY3971297 gets into the bloodstream and how long it takes the body to eliminate it.
- Lupus (SLE)
A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease
Testing the safety and early effects of a new treatment for lupus in adults
📍 Research Site 4 · Redwood City, CA · +6 more US sites · Get directions →
This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).
- 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).
- Lupus (SLE)
A Phase 1b Study of Budoprutug in Systemic Lupus Erythematosus (SLE)
Testing the safety and tolerance of budoprutug in adults with lupus
📍 Climb Bio Investigative Site #100104 · Allen, TX · +1 more US site · Get directions →
The main objective is to assess the safety and tolerability of budoprutug in adults with SLE. Pharmacokinetics, pharmacodynamics, and preliminary clinical efficacy will also be assessed.
- Lupus (SLE)Rheumatoid arthritis
A Study of CC-97540, CD-19-Targeted Nex-T CAR T Cells, in Participants With Severe, Refractory Autoimmune Diseases (Breakfree-1)
Testing a CD19-targeted CAR T cell treatment in people with severe lupus or rheumatoid arthritis
📍 University of Colorado Anschutz Medical Campus · Aurora, CO · +30 more US sites · Get directions →
The purpose of this study is to establish the tolerability, preliminary efficacy, and pharmacokinetics of CC-97540 in participants with severe, refractory autoimmune diseases (Breakfree-1).
- Obesity / overweightHigh blood pressure
Impact GLP-1 Agonists Following Bariatric
Testing whether GLP-1 medicines after bariatric surgery affect weight and blood sugar
📍 University of Missouri Hospital · Columbia, MO · Get directions →
Glucagon-like peptide 1 (GLP-1) is a hormone that helps regulate blood glucose levels through improved insulin sensitivity and release of insulin from the pancreas, control hunger, induce satiety and plays a role in the metabolic health of a person. GLP-1 receptor agonists (GLP1-RAs) have been shown to be effective in achieving weight loss in patients with type 2 diabetes while improving blood glucose control.
- 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.
- Lupus (SLE)
Trial of Belimumab in Early Lupus
Testing belimumab in people with lupus diagnosed within 2 years
📍 Feinstein Institute · Manhasset, NY · Get directions →
This two year study will evaluate the effects of giving belimumab (Benlysta) to patients with Early Lupus. Early lupus is a diagnosis of lupus within 2 years.
- Lupus (SLE)
A Study of GSK5926371 in Participants With B-cell Driven Autoimmune Rheumatic Diseases (ARD)
Testing different doses of an experimental medicine for lupus
📍 GSK Investigational Site · Buenos Aires, · Get directions →
This is a 2-part study of GSK5926371 in participants with autoimmune rheumatic diseases (ARD). In part 1, participants will receive different doses of GSK5926371 to find a suitable priming dose.
- Lupus (SLE)
A Phase 1 Study of FT819 in B-cell Mediated Autoimmune Disease
Testing an immune treatment for safety and effects on B-cells in lupus
📍 Wallace Rheumatic Center · Beverly Hills, CA · +15 more US sites · Get directions →
This is a phase 1 study designed to evaluate the safety, pharmacokinetics (PK), and anti-B-cell activity of FT819 following treatment with or without auxiliary medicinal product (AMP) in participants with moderate-to-severe active systemic lupus erythematosus (SLE) with or without nephritis, antineutrophilic cytoplasmic antibody (ANCA)-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). The study will consist of a dose-escalation stage, followed by an expansion stage to further evaluate the safety and activity of FT819.
- 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
- 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).
- Lupus (SLE)
BAFF CAR-T Cells (LMY-920) for Systemic Lupus Erythematosus
Testing safety and best dose for CAR-T cell treatment in adults with lupus
📍 Nationwide Children's · Columbus, OH · Get directions →
This phase 1 study seeks to examine the safety and recommended phase 2 dose (RP2D) of BAFF-ligand CAR-T cells (LMY-920) in adult patients with refractory systemic lupus erythematosus (SLE). It is hypothesized that BAFF CAR-T cells will be safe and will improve SLE disease activity scores.
- Lupus (SLE)
PRESERVE: LUPKYNIS in Combination With Belimumab, Obinutuzumab or Anifrolumab in Patients With Lupus Nephritis
Testing LUPKYNIS with other lupus medicines for kidney response in lupus nephritis
📍 Purushotham & Akther Kotha MD, Inc. · La Mesa, CA · +14 more US sites · Get directions →
The goal of this clinical study is to assess the efficacy and safety of LUPKYNIS® in combination with belimumab, obinutuzumab or anifrolumab at inducing rapid renal response in patients with lupus nephritis (LN).
- 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.
- Heart / cardiovascular diseaseHigh blood pressure
EASi-PROTKT™ - A Study to Test Vicadrostat (BI 690517) Taken Together With Empagliflozin in People With Type 2 Diabetes, High Blood Pressure, and Cardiovascular Disease
Testing a drug taken with empagliflozin in adults with type 2 diabetes and heart disease
📍 Pinnacle Research Group, LLC · Anniston, AL · +39 more US sites · Get directions →
This study is open to adults with type 2 diabetes, high blood pressure, and cardiovascular disease. People can join the study if they have these conditions and do not have a history of heart failure.
- 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.
- Lupus (SLE)
A Study Describing the Efficacy and Safety of Belimumab Administered in Adult Participants With Early Systemic Lupus Erythematosus (SLE)
Testing the safety and effects of belimumab in early lupus adults
📍 GSK Investigational Site · Anniston, AL · +39 more US sites · Get directions →
This is a prospective, open-label, single arm 3-year clinical study to describe the short-term and long-term efficacy and safety of belimumab in participants with autoantibody positive early SLE with ongoing disease activity despite stable first-line SLE therapy.
- 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.
- Lupus (SLE)
A Study to Assess Adverse Events, How the Drug Moves Through the Body and Effectiveness of Intravenous Infusions of ABBV-319 in Adult Participants With Systemic Lupus Erythematosus (SLE) and Sjogren's Disease (SjD)
Testing side effects and how an intravenous drug works in lupus and Sjogren’s
📍 Life Clinical Trials - Colonial Drive - Margate /ID# 276050 · Margate, FL · +3 more US sites · Get directions →
Systemic lupus erythematosus (SLE) is a chronic, systemic autoimmune disease characterized by B cell hyperactivity and Sjorgren's disease (SjD) is a chronic, multisystem autoimmune disease characterized by lacrimal and salivary gland inflammation, with resultant dryness of the eyes and mouth and occasional glandular enlargement. ABBV-319 exhibits potential B cell depletion in SLE and SjD which are characterized by B cell hyperactivity.
- 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.
- Heart / cardiovascular diseaseKidney disease💵 May compensate
Sotagliflozin to Slow Kidney Function Decline in Persons With Type 1 Diabetes and Diabetic Kidney Disease
Testing whether sotagliflozin can slow kidney damage in type 1 diabetes
📍 Stanford University Medical Center · Stanford, CA · +12 more US sites · Get directions →
Powerful new drugs that can prevent or delay end stage kidney disease (ESKD) - so called sodium-glucose cotransporter-2 inhibitors (SGLT2i) - are now available for patients with type 2 diabetes. Whether these drugs have similar effects in patients with type 1 diabetes (T1D) remains unknown because of the few studies in this population, due to concerns about the increase in risk of diabetic ketoacidosis (DKA, a serious, potentially fatal acute complication of diabetes due to the accumulation of substances called ketone bodies) observed with SGLT2i therapy in T1D.
- Obesity / overweightHigh blood pressure
Mitigating the Pro-inflammatory Phenotype of Obesity
Testing whether 4 weeks of a blood pressure medicine can reduce inflammation in people with obesity
📍 University of Kansas Medical Center · Kansas City, KS · Get directions →
The purpose of this study is to examine the hypothesize that 4 weeks of sympathetic nerve activity (SNA) inhibition (oral clonidine) will cause a significant reduction in circulating blood concentrations and endothelial cell expression of inflammatory markers (e.g., TNF-α, IL-6). Our study is a prospective study using a randomized, double-blinded design to test 4 weeks of SNA blockade (oral clonidine) compared with a BP-lowering control condition (diuretic, hydrochlorothiazide) or a placebo.
- Lupus (SLE)
REACT-01: Reversing Autoimmunity Through Cell Therapy
Testing a modified immune cell therapy for children and young adults with lupus
📍 Seattle Children's Hospital · Seattle, WA · Get directions →
This is a phase 1, open-label, non-randomized study enrolling pediatric and young adult research participants with treatment-refractory Systemic Lupus Erythematosus (SLE), to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to express CD19 specific chimeric antigen receptor (CAR) A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have their T cells collected. The T cells will then be bioengineered into a CAR T cell that targets circulating and tissue residing B cells.
- 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.
- Lupus (SLE)
Alnuctamab for Refractory SLE (LATTE Study)
Testing alnuctamab in people with moderate to severe lupus not helped
📍 Icahn School of Medicine at Mount Sinai · New York, NY · Get directions →
This study will assess the safety and preliminary efficacy of the bi-specific TCE, alnuctamab (known as BMS-986349, CC-93269, EM901), targeting BCMA in patients with moderate to severe SLE, refractory to standard-of-care treatments.
- 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.
- High blood pressure
A Study Comparing the Clinical Benefit of Finerenone Versus a Fixed Dose Combination (FDC) of Extended-Release Torsemide and Spironolactone in Patients With Hypertension and Chronic Kidney Disease
Comparing finerenone versus two-fluid pill combination for high blood pressure and chronic kidney
📍 Sarfez Pharmaceuticals, Inc. · Vienna, VA · Get directions →
A study Comparing the Clinical Benefit of Finerenone Versus a Fixed-Dose Combination (FDC) of Extended-Release Torsemide and Spironolactone in Patients with Hypertension and Chronic Kidney Disease.
- Sickle cell disease
Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease
Testing whether routine red blood cell transfusions in pregnancy reduce sickle cell flare-ups or
📍 Grady Health System · Atlanta, GA · Get directions →
The goal of this study is to determine if there is a positive effect of prophylactic red blood cell (RBC) transfusion of leukoreduced, ABO, Rh (D/Cc/Ee) and Kell matched blood compared to standard of care on the number of episodes of acute sickle cell disease (SCD) manifestations or pregnancy-related complications requiring acute health care encounters (acute care/ER/Hospital visits) or resulting in death over the entirety of pregnancy until 2 months post-partum in women with SCD. RBC transfusion is the only disease-modifying therapy for pregnant women with SCD, and it is considered a standard treatment option however, there exists no consensus on the role of transfusion therapy in preventing SCD-related pregnancy complications.
- Sickle cell disease
Gene Editing For Sickle Cell Disease
Testing whether gene editing is safe for people with sickle cell disease
📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.
- 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.
- Obesity / overweightHeart / cardiovascular disease💵 May compensate
Prevention of Progression of Prediabetes, Obesity and CV Risk
Testing ways to stop prediabetes and obesity from getting worse
📍 University of Texas Health Science Center at San Antonio · San Antonio, TX · Get directions →
The investigators are studying how to help people with prediabetes (Pre-DM) and obesity. The goal is to use new and affordable treatments to bring blood sugar levels back to normal and help participants to lose weight.
- Lupus (SLE)
Safety of the Herpes Zoster Subunit Vaccine in Lupus
Testing the safety of a shingles vaccine in people with lupus
📍 NYU Langone Health · New York, NY · +1 more US site · Get directions →
This randomized, double-blind, placebo-controlled, non-inferiority crossover study will evaluate the Herpes Zoster Sunbit (HZ/su) vaccine in SLE patients in order to evaluate safety and immunogenicity in patients with variable baseline clinical activities, ages and immunosuppressant exposures. The investigators hypothesize that HZ/su administration will be non-inferior to placebo with respect to the risk of moderate or severe SLE flare(s) occurring within 24 weeks of receiving the first dose of the assigned treatment.
- Lupus (SLE)
A Study of GSK4527363 in Healthy Participants, Systemic Lupus Erythematosus (SLE) Participants, Healthy Chinese, and Japanese Participants and CTD-ILD Participants
Testing the safety and immune effects of a drug in people with lupus and related lung disease
📍 GSK Investigational Site · Scottsdale, AZ · +5 more US sites · Get directions →
This study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of GSK4527363 in healthy participants (Part A), participants with active SLE (Part B), healthy participants of Chinese and Japanese descent (Part C), and participants with interstitial lung disease associated with connective tissue disease (Part D)
- Obesity / overweightHeart / cardiovascular disease
Evaluating the Impact of Maridebart Cafraglutide on Cardiovascular Outcomes in Participants With Atherosclerotic Cardiovascular Disease and Overweight or Obesity
Testing whether maridebart cafraglutide lowers heart risks in people with heart disease and obesity
📍 Alliance For Multispecialty Research - Daphne · Daphne, AL · +39 more US sites · Get directions →
The primary objective of this trial is to demonstrate that maridebart cafraglutide is superior to placebo when given as an adjunct to standard of care with respect to reducing cardiovascular (CV) morbidity and mortality.
- Obesity / overweightHeart / cardiovascular disease💵 May compensate
Maridebart Cafraglutide in Heart Failure With Preserved or Mildly Reduced Ejection Fraction and Obesity
Testing maridebart cafraglutide with standard care to reduce heart failure events in obese people
📍 University of Alabama Saint Vincent's Birmingham · Birmingham, AL · +39 more US sites · Get directions →
This trial will examine if maridebart cafraglutide as an adjunct to standard of care will lead to a reduction in heart failure (HF) events such as HF hospitalizations and urgent HF visits, cardiovascular (CV) deaths and improvement in HF symptoms in participants with HF with preserved ejection fraction (HFpEF) and HF with mildly reduced ejection fraction (HFmrEF) who are obese. This is a phase 3, global, multicenter, 2-part trial with a double-blind period and an open-label extension (OLE).
- 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.
- Obesity / overweightHeart / cardiovascular disease
A Research Study to Look at How Well NNC0487-0111 Works Compared to Placebo in People With Heart Failure and Obesity
Testing how well NNC0487-0111 works versus placebo in heart failure with excess body weight
📍 Advanced Cardiovascular, LLC · Alexander City, AL · +39 more US sites · Get directions →
This study is being done to look at the safety and effect of NNC0487-0111 in people with Heart Failure with preserved Ejection Fraction (HFpEF) or Heart Failure with mildly reduced Ejection Fraction (HFmrEF) and excess body weight when compared to placebo. The purpose of this clinical study is to find out if NNC0487-0111 is safe and effective for treating people who have HFpEF or HFmrEF and excess body weight.
- Obesity / overweightHigh blood pressure💵 May compensate
Optimizing Portal Hypertension With TIPS and Interval Metabolic Surgery for Advanced Liver Disease
Testing treatments to lower high pressure in liver veins in people with advanced liver disease and
📍 Cleveland Clinic Main Campus · Cleveland, OH · Get directions →
Cirrhosis is a form of advanced liver disease that can lead to serious complications, especially when combined with severe obesity. Many patients with cirrhosis also develop a condition called clinically significant portal hypertension (CSPH), which is increased pressure in the veins of the liver.
- 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 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.
- 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.
- Lupus (SLE)
A Study to Investigate the Safety and Preliminary Efficacy of ALLO-329, an Allogeneic CAR T-cell Therapy, in Adults With Autoimmune Disease
Testing safety and early effects of a CAR T-cell treatment in adults with lupus
📍 Mayo Clinic · Phoenix, AZ · +12 more US sites · Get directions →
This is a first-in-human, single-arm, open-label study evaluating the safety, tolerability, and preliminary efficacy of ALLO-329 in adults with autoimmune diseases: systemic lupus erythematosus (SLE) with and without renal involvement, idiopathic inflammatory myopathy (IIM), and systemic sclerosis (SSc).The purpose of this trial is to evaluate the safety and tolerability of ALLO-329, an allogeneic anti-CD19, anti-CD70 dual chimeric antigen receptor (CAR) T cell therapy, in adults with autoimmune disorders, provide initial evidence of biological activity and clinical response to the treatment and determine the recommended Phase 2 regimen (RP2R).
- Lupus (SLE)
Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders
Testing the safety and effects of Descartes-08 in young people with lupus
📍 H01- Children's National Hospital · Washington D.C., DC · +1 more US site · Get directions →
Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis
- Lupus (SLE)
An Efficacy and Safety Study of Intravenous Anifrolumab to Treat Systemic Lupus Erythematosus in Pediatric Participants
Testing an IV medicine to study effects and safety in children with lupus
📍 Research Site · Phoenix, AZ · +21 more US sites · Get directions →
A Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Efficacy, and Safety of Anifrolumab in Children with Moderate to Severe Active Systemic Lupus Erythematosus (SLE)
- 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.
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