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86 studies

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  • DepressionAnxiety

    Acceptance and Commitment Therapy for Adolescents and Young Adults With Sickle Cell Disease

    Testing whether therapy sessions can help teens and young adults with sickle cell cope with

    📍 Children's Hospital Los Angeles · Los Angeles, CA · Get directions →

    The current study seeks to build on previous research that demonstrates the efficacy of Acceptance and Commitment Therapy in combating stigma by investigating the feasibility and acceptability of a protocol to support Black adolescents and young adults in coping with race related stress. The study will consist of a small, purposeful, non-randomized sample (N = 30) of clients enrolled into a 10-session Acceptance and Commitment Therapy group.

    ✓ Study care freePhase 1Ages 14–21Learn more →
  • COPD (chronic lung disease)

    Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency

    Testing how a genetic change in alpha-1 antitrypsin affects immune and lung responses to a pneumonia

    📍 University of Alabama at Birmingham · Birmingham, AL · Get directions →

    The goal of this study is to learn whether patients who have a genetic mutation in the genes that cause alpha 1 antitrypsin deficiency also have genetic variation in nearby genes that can increase risk for reduced immune function and respiratory infections. To investigate this hypothesis, we will compare immune responses to the 20-valent pneumococcal conjugate vaccine (PCV20, Pfizer) between participants who have one abnormal copy of the SERPINA1 gene and either no COPD exacerbations, vs those with 2 or more COPD exacerbations in the past year.

    ✓ Study care freePhase 4Ages 19+Learn more →
  • 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 →
  • Anxiety

    Self-Adjusted Nitrous Oxide: A Feasibility Study in the Setting of Vasectomy

    Testing whether self-controlled nitrous oxide during vasectomy lowers pain and anxiety

    📍 Beth Israel Deaconess Medical Center · Boston, MA · Get directions →

    This study is being done to see if nitrous oxide during vasectomy decreases pain and anxiety, and also assess whether patients have better satisfaction when they control their own level of nitrous oxide during the procedure. If we find that patients experience less pain or anxiety with nitrous oxide, it could be suggested that self-adjusted nitrous oxide (SANO) may be a useful tool for improving experience of care during vasectomy.

    ✓ Study care freePhase 4Ages 21–85MenLearn more →
  • DepressionAnxiety

    ACT for Individuals With MNCD and Their Care Partners

    Testing acceptance commitment therapy for depression and anxiety in dementia

    📍 Texas Tech University Plaza Building · Lubbock, TX · Get directions →

    This project aims to implement and evaluate a dyadic intervention (i.e., acceptance commitment therapy) for persons with AD/ADRD and their care partners. We hypothesize the intervention will be feasible, acceptable, and show preliminary efficacy of the dyadic interveniotns.

    ✓ Study care freePhase 1Ages 18+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 →
  • 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 →
  • COPD (chronic lung disease)Asthma

    The Effects of Positive Airway Pressure on the Mucolytic Effects of NAC (TEAM)

    Testing whether positive airway pressure helps a lung medicine (NAC) reach mucus plugs in asthma or

    📍 UCSF Airway Clinical Research Center · San Francisco, CA · Get directions →

    The goal of this clinical trial is to determine if positive pressure during inspiration will improve penetration of aerosolized N-Acetylcysteine (NAC) into airway mucus plugs in the lungs of patients with asthma or Chronic Obstructive Pulmonary Disease (COPD). The main questions it aims to answer are: * Does delivery of aerosolized NAC with positive inspiratory pressure have a greater effect on mucus plug burden in the lungs than delivery of NAC without positive pressure.

    ✓ Study care freePhase 4Ages 18–85Learn 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

    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 →
  • 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 →
  • Anxiety

    Pharmacogenetically-guided Escitalopram Treatment for Pediatric Anxiety: Aiming to Improve Safety and Efficacy (PrEcISE)

    Testing whether genetic-guided dosing of a daily anxiety medicine helps kids

    📍 University of Cincinnati, Department of Psychiatry & Behavioral Neuroscience · Cincinnati, OH · Get directions →

    This double-blind, 12-week study will consist include132 anxious youth who are randomized (1:1) to standard or pharmacogenetically-guided escitalopram dosing. Block randomization (1:1) will be stratified by sex and metabolizer status.

    ✓ Study care freePhase 4Ages 12–17Learn 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 →
  • Anxiety

    Adjunctive Treatment With L-methylfolate for Treatment-Resistant Generalized Anxiety Disorder: a Pilot Study

    Testing whether adding L-methylfolate is safe and tolerated for hard-to-treat anxiety

    📍 Kingston Health Sciences Centre · Kingston, Ontario · Get directions →

    The goal of this feasibility study is to determine the tolerability and safety of add on treatment with L-methylfolate in patients with treatment-resistant generalized anxiety disorder (GAD). The primary objective is to monitor for side effects and other risks associated with the treatment.

    ✓ Study care freePhase 4Ages 18–65Learn 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 →
  • DepressionAnxiety

    Open-Label Psilocybin Study in Transdiagnostic Population

    Testing psilocybin for safety and symptom changes in people with depression or anxiety

    📍 Connecticut Mental Health Center - Yale School of Medicine · New Haven, CT · Get directions →

    The primary objective of this study is to investigate the safety, feasibility, and tolerability of psilocybin treatment in individuals with functional impairment due to psychiatric symptoms. The secondary objective of this study is to determine whether individuals with functional impairments due to psychiatric symptoms will experience statistically significant symptom reduction and functional improvement from baseline symptom measurements (Visit 3) to 1-week (Visit 7), 4-weeks (Visit 8), and 6-weeks (Visit 9) post dosing.

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

    Use of a Generative AI (Gen-AI) Chatbot for Anxiety and Depression Among Persons With Cannabis Use

    Testing a chatbot to support people using cannabis with anxiety or depression

    📍 46 Centerra Parkway, Suite 300, Office #333S · Lebanon, NH · Get directions →

    This study tests a chatbot called Therabot-CALM to help people who use cannabis with anxiety and/or low mood. Participants will do questionnaires, try the chatbot for 4 weeks, and join virtual interviews to give feedback on how usable and helpful it feels. The goal is to see if the chatbot is acceptable to users and can improve symptoms.

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

    The Impact of Focused Ultrasound Thalamotomy of the Anterior Nucleus for Focal-Onset Epilepsy on Anxiety

    Testing whether focused ultrasound thalamotomy can improve anxiety

    📍 The Ohio State University · Columbus, OH · Get directions →

    The purpose of this study is to evaluate the feasibility, safety, and effects on anxiety of high intensity focused ultrasound ablation (FUSA) in patients suffering from treatment-refractory focal epilepsy and anxiety. FUSA is a non-invasive neurosurgical procedure that uses ultrasound waves, sent directly through the scalp and skull, to precisely target small abnormal areas of the brain.

    ✓ Study care freePhase 1Ages 18–65Learn 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 →
  • COPD (chronic lung disease)

    📍 Equity Medical Bowling Green · Bowling Green, KY · +2 more US sites · Get directions →

    Randomized study of single and multiple doses of BBT002 in healthy volunteers and in adult patients with chronic obstructive pulmonary disease (COPD) or chronic rhinosinusitis with nasal polyps (CRSwNP).

    ✓ Study care freePhase 1Ages 18–80Learn 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 →
  • 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 →
  • 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 →
  • COPD (chronic lung disease)

    Effect of the Inhaled Triple Therapies Over the Small Airway in Biomass Exposure

    Comparing inhaled triple versus double medicines for COPD lung airways after wood smoke exposure

    💵 May compensate

    📍 Insituto Nacional de Enfermedades Respiratorias "Ismael Cosío Villegas" · Mexico City, Tlalpan · Get directions →

    This phase IV randomized controlled clinical trial intend to compare the effect of three close standard inhaled triple therapies and one close standard inhaled double therapy on the small airway in patients with Chronic Obstructive Pulmonary Disease (COPD-B) and chronic bronchitis without obstruction (BCNO) exposed to wood smoke. The treatment phase duration is of 3 months.

    ✓ Study care freePhase 4Ages 35–85WomenLearn more →
  • COPD (chronic lung disease)

    Compare the Effects of Nebulizer Versus Inhaler Based Therapy for COPD Using Long-acting Bronchodilators

    Comparing nebulizer and inhaler treatments for COPD symptoms

    📍 The University of Tennessee Graduate School of Medicine · Knoxville, TN · Get directions →

    The purpose of this study is to compare the effectiveness of inhaled bronchodilators delivered via nebulizers vs. dry powder inhalers (DPIs) in symptomatic participants with Chronic Obstructive Pulmonary Disease (COPD) who have airflow obstruction (FEV1/FVC ≤ 70%) and show significant air trapping (RV ≥ 120% of predicted).

    ✓ Study care freePhase 4Ages 40+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 →
  • COPD (chronic lung disease)

    A Study to Test the Safety and Effects of Inhaled GDC-6988 in Participants With Muco-obstructive Disease

    Testing the safety and effects of an inhaled medicine for COPD

    📍 University of Alabama at Birmingham · Birmingham, AL · +6 more US sites · Get directions →

    This study evaluates the safety, tolerability, and activity of inhaled GDC-6988 in participants with muco-obstructive disease.

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

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

    Affect of Duavive on Mood & Anxiety Symptoms

    Testing how Duavive affects mood and anxiety symptoms in early menopause women

    📍 St Joseph's Healthcare · Hamilton, Ontario · Get directions →

    This study evaluates the impact of conjugated estrogens/ bazedoxifene (CE/ BZA) on the mood (depression and anxiety) in peri- and early menopausal women.

    ✓ Study care freePhase 1Ages 45–60WomenLearn more →
  • Sickle cell disease

    Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease

    Testing an injectable birth control shot to reduce pain in women with sickle cell disease

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

    This research is being conducted to see if using an injectable contraception, Depot Medroxyprogesterone Acetate (Depo-Provera), can reduce the pain experienced by women with sickle cell disease. Participants in this study will be adult women with sickle cell disease who regularly experience sickle cell pain.

    ✓ Study care freePhase 4Ages 18–50WomenLearn more →
  • Parkinson's disease

    Treating Parkinson's Disease Through Transplantation of Autologous Stem Cell-Derived Dopaminergic Neurons

    Testing surgery to transplant dopamine brain cells made from a patient’s own skin

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

    The goal of this clinical trial is to assess the safety and tolerability of the surgical transplantation of dopaminergic progenitor cells into the brains of participants with Parkinson's disease. The transplanted dopaminergic cells will be derived from the participant's own skin cells.

    ✓ Study care freePhase 1Ages 45–80Learn more →
  • Sickle cell disease

    Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease

    Testing whether children with sickle cell disease can take daily ADHD medicine and monitoring its

    📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →

    The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork problems and if they have any side effects. The study will assess any thinking or attention problems participants may have both before taking this drug and after.

    ✓ Study care freePhase 1Ages 8–17Learn more →
  • Dementia / Alzheimer'sParkinson's diseaseMultiple sclerosis (MS)

    Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers

    Testing safety of a PET imaging tracer in people with ALS, AD, MS, PD

    📍 UCSF · San Francisco, CA · +2 more US sites · Get directions →

    This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs. 18F-OP-801 is intended as a biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation.

    ✓ Study care freePhase 1Ages 18–80Learn more →
  • Sickle cell disease

    Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease

    Testing if adding ruxolitinib lowers graft failure after transplant in children with sickle cell

    📍 Children's Hospital of Colorado · Aurora, CO · +3 more US sites · Get directions →

    This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants.

    ✓ Study care freePhase 1Ages 12–45Learn more →
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