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92 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 →
  • Fatty liver disease (NASH/MASH)

    📍 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).

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Fatty liver disease (NASH/MASH)

    A Research Study of a Potential New Medicine (NNC4005-0001) for Liver Disease in Adult Participants With Increased Body Weight and Liver Fat

    Testing a new medicine for liver disease in adults with extra body fat

    📍 Altasciences Clinical Company, Inc · Montreal, Quebec · Get directions →

    The purpose of this clinical study is to find out if NNC4005-0001 is well-tolerated and safe for people who have increased body weight and increased liver fat. Participants will receive either NNC4005-0001, which is the treatment being tested, or a placebo, which is a treatment that contains no active medicine.

    ✓ Study care freePhase 1Ages 18–69Learn 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 →
  • Fatty liver disease (NASH/MASH)

    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

    💵 May compensate

    📍 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.

    ✓ Study care freePhase 3Ages 18–75Learn 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 →
  • Type 2 diabetesFatty liver disease (NASH/MASH)

    A Phase 1b Study of QX1206 in T2DM Patients With NAFLD

    Testing QX1206 in people with type 2 diabetes and fatty liver

    📍 Centricity Research Toronto LMC. · Toronto, · Get directions →

    This is an open label phase 1b trial of QX1206 in patients with T2DM and with NAFLD. Laboratory tests and other measurements will be assessed prior to the first dose of study treatment and throughout the study to determine the recommended phase 2 dose.

    ✓ Study care freePhase 1Ages 18–64Learn 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 →
  • 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 →
  • 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 →
  • 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 →
  • Parkinson's disease

    A Study of CREXONT (Carbidopa and Levodopa) Extended-Release Capsules in Participants With Parkinson's Disease

    Testing a carbidopa and levodopa extended-release capsule in people with Parkinson’s

    📍 Barrow Neurological Institute · Phoenix, AZ · +26 more US sites · Get directions →

    The primary purpose of this study is to evaluate efficacy and safety of CREXONT under real world conditions in participants with Parkinson disease (PD).

    ✓ Study care freePhase 4Ages 18+Learn 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 →
  • 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 →
  • 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.

    ✓ Study care freePhase 3Ages 18–80Learn more →
  • 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.

    ✓ Study care freePhase 3Ages 18–80Learn more →
  • Obesity / overweightFatty liver disease (NASH/MASH)

    Human Models of Selective Insulin Resistance: Alpelisib, Part I

    Testing how insulin works in healthy people versus people at risk for type 2 diabetes

    💵 May compensate

    📍 Columbia University Irving Medical Center · New York, NY · Get directions →

    The goal of this clinical trial is to understand how the blood sugar-lowering hormone insulin works in healthy adults versus those who are at risk for type 2 diabetes. The study will use a drug called alpelisib, which interferes with insulin's actions in the body, to answer the study's main question: does the liver continue to respond to insulin's stimulation of fat production even when it loses the ability to stop making glucose (sugar) in response to insulin.

    ✓ Study care freePhase 1Ages 18–70Learn 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 →
  • Obesity / overweightFatty liver disease (NASH/MASH)

    Fibrosis Lessens After Metabolic Surgery

    Testing whether metabolic surgery can reduce liver scarring in obesity

    💵 May compensate

    📍 Banner Health Center · Phoenix, AZ · +3 more US sites · Get directions →

    Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), a major global public health concern, is commonly associated with obesity, diabetes, and dyslipidemia. MASLD is currently the most common cause of chronic liver disease affecting about 80% of people with obesity, ranging from simple fat deposits in the liver to Metabolic Dysfunction-Associated Steatohepatitis (MASH), cellular injury, advanced fibrosis, cirrhosis, or hepatocellular carcinoma.

    ✓ Study care freePhase 4Ages 18–75Learn 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 →
  • Fatty liver disease (NASH/MASH)

    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

    💵 May compensate

    📍 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 \[previously known as nonalcoholic steatohepatitis, NASH\]).

    ✓ Study care freePhase 3Ages 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 →
  • 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 →
  • Parkinson's disease

    Cortical Electrophysiology of Response Inhibition in Parkinson's Disease

    Studying how deep brain stimulation in Parkinson’s affects brain control of stopping actions

    📍 Emory University Hospital · Atlanta, GA · +1 more US site · Get directions →

    Patients with Parkinson's Disease will be studied before, during, and after a deep brain stimulation implantation procedure to see if the stimulation location and the size of the electrical field produced by subthalamic nucleus (STN) DBS determine the degree to which DBS engages circuits that involve prefrontal cortex executive functions, and therefore have a direct impact on the patient's ability to inhibit actions.

    ✓ Study care freePhase 4Ages 45–75Learn 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

    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.

    ✓ Study care freePhase 4Ages 18+WomenLearn 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 →
  • Fatty liver disease (NASH/MASH)

    Evaluating the Pharmacokinetics and Safety of Miricorilant

    Testing how a drug works in the body and its safety in adults with MASH

    💵 May compensate

    📍 Site# 433 · San Antonio, TX · Get directions →

    A Phase 1b, Open-Label Study Evaluating the Pharmacokinetics and Safety of Miricorilant in Adult Patients With Presumed Metabolic Dysfunction-Associated Steatohepatitis (MASH)

    ✓ Study care freePhase 1Ages 18–75Learn 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 →
  • Fatty liver disease (NASH/MASH)

    Bicalutamide Therapy in Young Women With NAFLD and PCOS

    Testing bicalutamide in young women with fatty liver inflammation (NASH) and PCOS

    📍 University of California San Francisco · San Francisco, CA · Get directions →

    Nonalcoholic steatohepatitis (NASH), or fat-related liver inflammation and scarring is projected to be the leading cause of cirrhosis in the United States (U.S.) within the next few years. Women are at disproportionate risk for NASH, with approximately 15 million U.S.

    ✓ Study care freePhase 1Ages 18–42WomenLearn 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 →
  • 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 →
  • Parkinson's disease

    📍 Cornell University Weill Medical College · New York, NY · Get directions →

    This is a phase 1/2 clinical study to evaluate the safety, tolerability, feasibility, and preliminary efficacy of NouvNeu001 in patients with advanced Parkinson's Disease.

    ✓ Study care freePhase 1Ages 30–75Learn more →
  • Parkinson's disease

    BOOST-PD A Naturalistic Study on IPX-203 for Parkinson's Disease

    Testing a new long-acting levodopa dose to improve “on” time in Parkinson’s

    📍 Cleveland Clinic · Cleveland, OH · Get directions →

    The purpose of this study is to evaluate the effect of IPX203 (Crexont®) - the newest extended-release levodopa formulation - on the duration and quality of good on time, using a wearable device to monitor symptoms. 'Good on time' refers to a period (minutes to hours) when a patient experiences optimal symptom control due to effective medication and has better overall functioning without troublesome dyskinesias.

    ✓ Study care freePhase 4Ages 40+Learn more →
  • Sickle cell disease

    SCD Stem Cell Mobilization and Apheresis Using Motixafortide

    Testing whether motixafortide is safe and can raise stem cells in people with sickle cell disease

    📍 University of Alabama at Birmingham · Birmingham, AL · +2 more US sites · Get directions →

    This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use.

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

    📍 University Of Alabama · Birmingham, AL · +13 more US sites · Get directions →

    A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

    ✓ Study care freePhase 3Ages 12–100Learn more →
  • Parkinson's disease

    A Study to Evaluate the Efficacy and Safety of Intravenous (IV) Prasinezumab in Participants With Early-Stage Parkinson's Disease

    Testing an IV drug in people with early-stage Parkinson’s disease

    📍 University of Alabama at Birmingham · Birmingham, AL · +39 more US sites · Get directions →

    The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of prasinezumab compared with placebo in participants with early-stage Parkinson's disease (PD) on stable symptomatic monotherapy with levodopa.

    ✓ Study care freePhase 3Ages 50–85Learn more →
  • Parkinson's disease

    Transplantation of Human iPS Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II)

    Testing whether transplanting lab-made dopamine cells is safe for Parkinson’s

    📍 University of California, San Diego · La Jolla, CA · Get directions →

    To evaluate the safety and efficacy of transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors, CT1-DAP001, into the corpus striatum in patients with Parkinson's disease

    ✓ Study care freePhase 1Ages 40–75Learn more →
  • Sickle cell disease

    Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)

    Testing different ways to use hydroxyurea for treating sickle cell anemia

    📍 Cincinnati Children's Hospital Medical Center · Cincinnati, OH · Get directions →

    The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for people with SCA.

    ✓ Study care freePhase 4Ages 1+Learn more →
  • Parkinson's disease

    Autologous suraL nervE Grafting to the Substantia nigrA in Patients With Synuclienopathies

    Testing whether a nerve tissue graft can be safely placed in the brain in Parkinson’s or MSA

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

    This phase I double-blind study focuses on the safety and feasibility of implanting autologous peripheral nerve tissue (PNT) into the substantia nigra area of the brain in persons who have been diagnosed with either Parkinson's disease (PD) or Multiple System Atrophy (MSA). 7 participants will be enrolled, with 4 participants receiving the graft and 3 receiving a sham surgery.

    ✓ Study care freePhase 1Ages 40–75Learn more →
  • Fatty liver disease (NASH/MASH)

    A Study Evaluating Efruxifermin in Subjects With Compensated Cirrhosis Due to NASH/MASH

    Testing a medicine called efruxifermin in people with compensated liver scarring from NASH/MASH

    💵 May compensate

    📍 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 compensated cirrhosis due to NASH/MASH.

    ✓ Study care freePhase 3Ages 18–80Learn more →
  • Sickle cell disease

    Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

    Testing the safety and effects of panobinostat in adults with sickle cell disease

    📍 Augusta University · Augusta, GA · Get directions →

    The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor.

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

    A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

    Testing how a new medicine works in healthy people and sickle cell patients

    📍 University of Alabama Birmingham · Birmingham, AL · +5 more US sites · Get directions →

    The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).

    ✓ Study care freePhase 1Ages 12–55Learn more →
  • Sickle cell disease

    Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

    Testing a bone marrow transplant with lower-intensity treatment for kids with sickle cell and

    📍 Yale School of Medicine · New Haven, CT · +3 more US sites · Get directions →

    This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.

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

    A Clinical Trial of LY3962681 in Healthy Volunteers and in Patients With Parkinson's Disease

    Testing how a Parkinson’s drug candidate affects safety and body handling in healthy people and

    📍 Austin Clinic PPD · Austin, TX · Get directions →

    The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics/pharmacodynamics (PK/PD) of LY3962681 in healthy volunteers and patients with Parkinson's disease. The study consists of two parts, the Single Ascending Dose (SAD) study and the Multiple Ascending Dose (MAD) study.

    ✓ Study care freePhase 1Ages 30–80Learn more →
  • Parkinson's disease

    rTMS to Improve Cognition in Parkinson's

    Testing whether noninvasive brain stimulation can improve thinking in people with Parkinson’s

    📍 Jesse Brown VA Medical Center, Chicago, IL · Chicago, IL · +1 more US site · Get directions →

    The purpose of this study is to examine safety, feasibility, and the behavioral and brain effects of a non-invasive treatment, repetitive transcranial magnetic stimulation (rTMS), for Veterans with Parkinson's disease or atypical parkinsonism and mild impairments in their thinking. The hypothesis is that rTMS can improve thinking for people with Parkinson's disease or atypical parkinsonism who are experiencing mild problems with their thinking ability.

    ✓ Study care freePhase 1Ages 50+Learn more →
  • Dementia / Alzheimer'sParkinson's disease

    [18F]F-DOPA Imaging in Patients With Autonomic Failure

    Testing [18F]F-DOPA brain scans in people with autonomic failure

    📍 Vanderbilt University Medical Center · Nashville, TN · Get directions →

    Alpha-synucleinopathies refer to age-related neurodegenerative and dementing disorders, characterized by the accumulation of alpha-synuclein in neurons and/or glia. The anatomical location of alpha-synuclein inclusions (Lewy Bodies) and the pattern of progressive neuronal death (e.g.

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

    A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

    Testing how well etavopivat lowers painful sickle cell crises and organ damage

    📍 Uni of Alabama at Birmingham · Birmingham, AL · +39 more US sites · Get directions →

    This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo.

    ✓ Study care freePhase 3Ages 12+Learn more →
  • Parkinson's disease

    Pimavanserin vs. Quetiapine for Treatment of Parkinson's Psychosis

    Comparing treatments to see which works better for hallucinations in Parkinson’s disease

    📍 Southern Arizona VA Health Care System, Tucson, AZ · Tucson, AZ · +23 more US sites · Get directions →

    Patients with Parkinson's disease (PD) sometimes experience symptoms affecting their movement, such as slowness, tremor, stiffness, and balance or walking problems. Many patients also have other symptoms not related to movement, called non-motor symptoms, which may affect one's mood or emotions, memory or thinking, or cause one to see or hear things that aren't real (hallucinations) or believe things that aren't true (delusions).

    ✓ Study care freePhase 4Ages 40+Learn more →
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