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120 studies

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  • Dementia / Alzheimer'sParkinson's disease

    Phase 2 Trial of Tributyrin in People With Parkinson's Disease and Cognitive Impairments

    Testing whether a tributyrin supplement helps memory, thinking, and balance in adults with Parkinson

    📍 Domino's Farms · Ann Arbor, MI · Get directions →

    The goal of this clinical trial is to learn if taking a tributyrin supplement works to improve memory and thinking and walking and balance in adults with Parkinson disease Parkinson disease dementia. It will also learn about the safety of tributyrin supplementation.

    ✓ Study care freePhase 1Ages 45+Learn more →
  • Parkinson's disease

    Virtual tES Device Treatment for Parkinson's Disease Motor Symptoms

    Testing a non-invasive brain stimulation device to improve Parkinson’s movement problems

    📍 U LLC Headquarters · Cleveland, OH · Get directions →

    This study tests a new, non-invasive brain stimulation device called Sphere V3.0 to help improve movement problems in people with Parkinson's disease. The device delivers gentle electrical signals to the scalp using small electrodes placed on the head.

    ✓ Study care freePhase 1Ages 18–80Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Parkinson's disease

    First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)

    Testing an experimental treatment for early Parkinson’s disease in adults

    📍 Montreal Neurological Institute and Hospital · Montreal, Quebec · Get directions →

    This study is researching an experimental drug called ALN-SNCA (called "study drug"). The study is focused on people with early Parkinson's disease, a disorder of the nervous system that affects movement.

    ✓ Study care freePhase 1Ages 50–80Learn more →
  • Parkinson's disease

    📍 University of Kentucky · Lexington, KY · Get directions →

    The investigators propose a Phase I single surgical-center, double-blinded randomized parallel clinical trial involving bilateral autologous peripheral nerve tissue (PNT) delivery into the NBM or the alternate target also affecting cognition in this population, the substantia nigra (SN), to address "repair cell" support of these areas. Twenty-four participants with idiopathic Parkinson's Disease (PD) who have selected, qualified and agreed to receive as standard of care deep brain stimulation (DBS) will be enrolled and randomly allocated to receive bilateral PNT deployment to either the NBM or SN at the time of DBS surgery.

    ✓ Study care freePhase 1Ages 45–75Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 50+Learn more →
  • Sickle cell disease

    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

    💵 May compensate

    📍 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.

    ✓ Study care freePhase 1Ages 18–90Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 12–120Learn more →
  • Parkinson's disease

    A Study of Buntanetap in Participants With PD

    Testing the long-term safety of buntanetap in people with Parkinson’s

    📍 University of Alabama at Birmingham · Birmingham, AL · +26 more US sites · Get directions →

    This study will examine the long-term safety of buntanetap in participants with PD. This will be a 36-month open-label safety study.

    ✓ Study care freePhase 2Ages 40–85Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 12–29Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–55Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 18–75Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 18–70Learn more →
  • Parkinson's disease

    A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease

    Testing a Parkinson’s surgery approach to see if it is safe and works

    📍 Banner Sun Health Research Institute · Sun City, AZ · +32 more US sites · Get directions →

    Study BRT-DA01-301 is a Phase 3 multicenter, randomized, sham surgery-controlled, double-blind study to assess efficacy and safety of bemdaneprocel in approximately 102 adults with Parkinson's Disease (PD).

    ✓ Study care freePhase 3Ages 45–75Learn more →
  • 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).

    ✓ Study care freePhase 3Ages 10–65Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 7–21Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–70Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 12–70Learn more →
  • 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

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Lupus (SLE)

    📍 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).

    ✓ Study care freePhase 3Ages 12–100Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 1+Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 18+Learn more →
  • Parkinson's disease

    A Study of LY4006896 in Healthy Participants and Participants With Parkinson's Disease

    Testing an IV medicine in healthy people and people with Parkinson’s

    📍 Collaborative Neuroscience Network - CNS · Los Alamitos, CA · +11 more US sites · Get directions →

    The purpose of this study is to generate evidence of the safety, tolerability, and pharmacokinetics/pharmacodynamics of IV LY4006896 compared with placebo in healthy participants and participants with Parkinson's disease.

    ✓ Study care freePhase 1Ages 30–85Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 60+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–70Learn more →
  • Lupus (SLE)

    📍 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.

    ✓ Study care freePhase 1Ages 18–75Learn more →
  • Parkinson's disease

    SAD Study in Patients With Parkinson's Disease and Motor Fluctuations

    Testing a single dose of SER-252 in people with Parkinson’s disease

    💵 May compensate

    📍 Rocky Mountain Clinical Research · Englewood, CO · +3 more US sites · Get directions →

    This is a randomized, placebo-controlled, single ascending dose (SAD) study of SER-252 in participants with Parkinson's Disease (PD) and motor fluctuations.

    ✓ Study care freePhase 1Ages 40–80Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 12–35Learn more →
  • Parkinson's disease

    18F-DOPA II - PET Imaging Optimization

    Testing an imaging scan to study Parkinson’s disease using PET

    📍 WC Mackenzie Health Science Centre / University of Alberta Hospital · Edmonton, Alberta · Get directions →

    A single centre non-randomized, non-blinded phase III prospective cohort study of 18F-DOPA PET/CT imaging in specific patient populations: 1. Pediatric patients (less than 18 years old) with congenital hyperinsulinism.

    ✓ Study care freePhase 3Learn more →
  • Lupus (SLE)

    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

    💵 May compensate

    📍 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.

    ✓ Study care freePhase 3Ages 18–75Learn more →
  • Heart / cardiovascular diseaseKidney disease

    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

    💵 May compensate

    📍 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.

    ✓ Study care freePhase 3Ages 18–75Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 2–30Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–60Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Parkinson's disease

    Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell Exosomes for Parkinson's Disease

    Testing the safety and effectiveness of nose drops made from donor stem cell exosomes for

    📍 Medical Surgical Associates Center · St John's, · Get directions →

    This trial will study the safety and efficacy of intranasal instillation of cultured allogeneic adult umbilical cord derived mesenchymal stem cell exosomes for the treatment of Parkinson's Disease

    ✓ Study care freePhase 1Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–24Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Parkinson's disease

    A Pilot Study of In-lab Dynamic Standing in Parkinson's Disease

    Testing a new adjustable standing desk for people with Parkinson’s

    📍 Domino's Farms · Ann Arbor, MI · Get directions →

    This research is studying the use of a new type of standing desk in a small number of people to learn about the user experience for people with Parkinson's disease. 12 4-hour sessions will be performed to test the primary hypothesis that dynamic standing improves gait function compared to static standing and control sitting.

    ✓ Study care freePhase 1Ages 50+Learn more →
  • Parkinson's disease

    First-in-Human Single and Multiple Dose of HL-400

    Testing an NLRP3 inhibitor taken by mouth for safety in healthy people

    📍 Pharmaron CPC, Inc. · Baltimore, MD · Get directions →

    This is a randomized, double-blind, placebo-controlled, dose-escalation study in healthy subjects to evaluate the safety, tolerability, pharmacokinetics of HL-400 (a NLRP3 inhibitor) following oral single and multiple ascending dose administration.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Obesity / overweightHeart / cardiovascular disease

    Prevention of Progression of Prediabetes, Obesity and CV Risk

    Testing ways to stop prediabetes and obesity from getting worse

    💵 May compensate

    📍 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Parkinson's disease

    Using taVNS to Modulate Cardiovascular Function in Individuals With Neurologic Disease

    Testing gentle ear nerve stimulation to see if it affects heart rate and blood pressure in people

    📍 Wellness, Health and Research Facility at UAB · Birmingham, AL · Get directions →

    The purpose of this study is to find out whether a type of gentle nerve stimulation, called transcutaneous auricular Vagus Nerve Stimulation (taVNS), can help improve how the body regulates heart rate and blood pressure in people with Parkinson's Disease (PD). Problems with heart rate and blood pressure control are common and can make it harder for people to exercise or do daily activities.

    ✓ Study care freePhase 1Ages 18–80Learn more →
  • 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)

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 45–99Learn more →
  • Obesity / overweightHeart / cardiovascular disease

    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

    💵 May compensate

    📍 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).

    ✓ Study care freePhase 3Ages 18–99Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 18–75Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 18+Learn more →
  • Parkinson's disease

    Enrichment of Glutathione Using Gamma-glutamylcysteine Supplementation in Parkinson's Disease Patients.

    Testing whether taking glutathione-building supplements helps early Parkinson’s patients

    📍 UPMC Presbyterian Hospital · Pittsburgh, PA · Get directions →

    This study is designed l to evaluate the effects of GGC oral supplementation in early Parkinson's disease (PD) patients. The main objectives of the study are to evaluate: 1.

    ✓ Study care freePhase 1Ages 50–80Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 2+Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • 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).

    ✓ Study care freePhase 1Ages 18–69Learn more →
  • 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

    ✓ Study care freePhase 1Ages 12+Learn more →
  • 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)

    ✓ Study care freePhase 3Ages 5–17Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 4–65Learn more →
  • 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.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Sickle cell disease

    Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

    Testing gene editing in stem cells for severe sickle cell disease

    📍 Boston Children's Hospital · Boston, MA · Get directions →

    A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.

    ✓ Study care freePhase 1Ages 13–40Learn more →
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