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111 studies
Recruiting now, in plain language. Every study is watched over by an independent ethics board.
- COPD (chronic lung disease)
A Study to Investigate the Effect of AZD6793 in Participants With Moderate to Very Severe Chronic Obstructive Pulmonary Disease
Testing two doses of a medicine versus placebo for COPD symptoms
📍 Research Site · Birmingham, AL · +39 more US sites · Get directions →
This Phase IIb dose-ranging study will evaluate the efficacy and safety of 2 different doses of AZD6793 compared with placebo tablets in participants with moderate to very severe chronic obstructive pulmonary disease.
- 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.
- 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.
- 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.
- 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.
- Parkinson's disease
Clinical Trial Evaluating the Efficacy and Safety of AGB101 for Treatment of Parkinson's Disease Related Psychosis
Testing whether a low-dose seizure medicine can help Parkinson’s psychosis
📍 Johns Hopkins · Baltimore, MD · Get directions →
This clinical trial will test whether AGB101 (low-dose levetiracetam, 220 mg, extended release tablet) can improve symptoms of psychosis in Parkinson's disease. Participants will be asked to complete up to 5 in-person study visits over approximately 20 weeks.
- Parkinson's disease
A Phase 2 Study and Open-Label Extension of NEU-411 in Companion Diagnostic-Positive Participants With Early Parkinson's Disease
Testing a treatment in people with early Parkinson’s disease who have an LRRK2 gene test result
📍 Banner Sun Health Research Institute · Sun City, AZ · +39 more US sites · Get directions →
The goal of this Phase 2 clinical trial is to investigate the efficacy and safety of NEU-411 in men and women aged 40-80 years with early Parkinson's Disease (PD) who have predicted elevations in the activity of the "leucine-rich repeat kinase 2" ("LRRK2" for short) pathway based on their genetic profile. A DNA test will be used to identify the "LRRK2-driven" population with predicted elevation in the LRRK2 pathway.
- Parkinson's disease
A Study to Evaluate Feasibility, Safety, and Clinical Responses of Implanting Autologous Peripheral Nerve Tissue Into the Brain for Non-motor or Motor Symptoms in Patients With Parkinson's Disease Undergoing DBS Surgery
Testing whether implanting a patient’s own nerve tissue during DBS surgery is safe in Parkinson’s
📍 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.
- Sickle cell disease💵 May compensate
Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
Testing higher doses of VAS-101 in people with stable sickle cell disease
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.
- 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.
- 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.
- Sickle cell disease
Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults
Testing whether a low dose of ketamine helps treat sickle cell pain in children and young adults
📍 Harbor UCLA Medical Center · Torrance, CA · Get directions →
The purpose of this research is to see if ketamine is effective and safe in treating children and young adults with sickle cell disease experiencing sickle cell related pain. In this study, we will compare the outcomes (such as pain scores) in persons who receive standard of care pain medicine (an opioid such as morphine) plus a low dose (amount) of ketamine to those who receive only standard of care pain medicine.
- COPD (chronic lung disease)
To Evaluate the Effect of EXPD-101/FXS7553 Compared With Placebo in Patients With COPD Over 52 Weeks of Treatment
Testing two doses of a daily medicine versus placebo in people with COPD over 52 weeks
📍 DM Clinical Research - Phoenix · Phoenix, AZ · +11 more US sites · Get directions →
This is a Phase 2 study of the efficacy, safety, tolerability and pharmacokinetics of two doses of EXPD-101 in participants with COPD. Study participants will be randomized to receive either study drug or placebo administered once daily for 52 weeks.
- 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.
- Sickle cell disease
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Testing a once-daily mouth medicine in teens with sickle cell disease
📍 The Hospital for Sick Children · Toronto, Ontario · Get directions →
This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat.
- 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.
- DepressionParkinson's disease
Imaging Depression in Parkinson's Disease
Studying brain changes linked to depression in people with Parkinson’s
📍 Yale University · New Haven, CT · Get directions →
The goal of this observational study is to identify targetable neural substrates of depression in Parkinson's Disease for the first time in people with Parkinson's between the ages of 40 and 80, who are experiencing symptoms of depression.
- 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.
- High blood pressureCOPD (chronic lung disease)
Breathe Easier With Tadalafil Therapy for Dyspnea in COPD-PH
Testing tadalafil to see if it improves shortness of breath in COPD with high blood pressure in the
📍 Rocky Mountain Regional VA Medical Center, Aurora, CO · Aurora, CO · +4 more US sites · Get directions →
The investigators will study whether the drug tadalafil improves shortness of breath in 126 Veterans with Chronic Obstructive Pulmonary Disease (COPD) and high blood pressure in the lungs. The investigators will also assess whether tadalafil improves quality of life, home daily physical activity, exercise endurance, the frequency of acute flares of COPD, blood pressure in the lungs, and lung function.
- Parkinson's disease
Ketamine for Veterans With Parkinson's Disease
Testing ketamine for depression in veterans with Parkinson’s disease
📍 San Francisco VA Medical Center, San Francisco, CA · San Francisco, CA · Get directions →
Parkinson's disease (PD) is a devastating illness that has a growing impact on Veterans. One of the most disabling symptoms is depression, which is common in PD and linked to poor quality of life and higher risk of suicide.
- Parkinson's disease💵 May compensate
SAD Study in Patients With Parkinson's Disease and Motor Fluctuations
Testing a single dose of SER-252 in people with Parkinson’s disease
📍 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.
- 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).
- Parkinson's disease
Early Longitudinal Imaging in the Parkinson's Progressive Marker Initiative (PPMI) Using (18F)AV-133 (PPMI AV-133 Prodromal Imaging)
Tracking how brain scans change over time in people with early Parkinson’s
📍 Institute for Neurodegenerative Disorders · New Haven, CT · +1 more US site · Get directions →
The study is a longitudinal, multi-center study to assess progression of \[18F\] AV-133 imaging in Prodromal PD participants. Participants will be followed for up to 24 months.
- DepressionParkinson's disease
Psilocybin Therapy for Depression in Parkinson's Disease
Testing whether psilocybin therapy improves depression symptoms in people with Parkinson’s disease
📍 University of California, San Francisco · San Francisco, CA · Get directions →
The purpose of this study is to understand whether people with Parkinson's Disease and depression have improvement in their symptoms after psilocybin therapy.
- 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.
- COPD (chronic lung disease)
Rademikibart Add-on Treatment of an Acute COPD Exacerbation (Seabreeze STAT COPD)
Testing an add-on treatment for adults having a COPD flare with type 2 inflammation
📍 University of Alabama at Birmingham Lung Health Center · Birmingham, AL · +21 more US sites · Get directions →
This is a Phase 2, multicenter study in adult participants with an acute COPD exacerbation and type 2 inflammation
- 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
- COPD (chronic lung disease)
A Study of BBT002 in Healthy Volunteers (HVs) and in Adult Patients With Chronic Obstructive Pulmonary Disease (COPD) or Chronic Rhiosininusitis With Nasal Polyps (CRSwNP)
Testing different doses of BBT002 in people with COPD and in healthy adults
📍 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).
- Sickle cell disease
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
Testing early heart screening and iron overload treatment in people with sickle cell disease
📍 Inova Schar Cancer · Fairfax, VA · +1 more US site · Get directions →
This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.
- Sickle cell disease
Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial
Testing two daily doses of zinc in people with sickle cell disease to see changes in bone-related
📍 UCSF Benioff Children's Hospital Oakland · Oakland, CA · +5 more US sites · Get directions →
The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover?
- Sickle cell disease
Gene Editing For Sickle Cell Disease
Testing whether gene editing is safe for people with sickle cell disease
📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.
- Sickle cell disease
Allo HSCT for High Risk Hemoglobinopathies
Testing donor stem cell transplant for people with high-risk sickle cell
📍 Masonic Cancer Center · Minneapolis, MN · Get directions →
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
- 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.
- 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.
- Parkinson's disease
A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
Testing the safety and effect of a gene therapy put into the brain for moderate Parkinson’s disease
📍 Mayo Clinic · Phoenix, AZ · +33 more US sites · Get directions →
The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.
- Parkinson's disease
Low-dose Carbon Monoxide (HBI-002) Trial to Evaluate Safety, Tolerability, PK, and Biomarkers in Parkinson's Disease
Testing an oral low-dose carbon monoxide liquid in people with Parkinson’s
📍 Quest Research Institute · Farmington Hills, MI · Get directions →
A phase 2a multicenter, randomized, double-blind, placebo-controlled multiple dose study to evaluate the safety, tolerability, pharmacokinetics, of HBI-002, an oral low-dose carbon monoxide (CO) liquid drug product, administered daily over 14 days in subjects with Parkinson's disease (PD).
- 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.
- COPD (chronic lung disease)
A Phase IIb Ensifentrine-glycopyrrolate Fixed-dose Combination Dose Ranging Study in Subjects With COPD
Testing different doses of two inhaled medicines for COPD symptoms
📍 SEC Clinical Research, LLC · Dothan, AL · +39 more US sites · Get directions →
This study will assess the safety and efficacy of fixed dose combinations of ensifentrine with two different glycopyrrolate dose levels compared to placebo and to the individual components of the fixed dose combinations, each administered twice a day via standard jet nebulizer, in adult subjects with chronic obstructive pulmonary disease (COPD).
- Parkinson's disease
Pyridostigmine for the Treatment of Constipation in Parkinson Disease
Testing pyridostigmine as a treatment for constipation in Parkinson disease
📍 University of Vermont Medical Center · Burlington, VT · Get directions →
Constipation is a common and debilitating non-motor symptom of Parkinson disease (PD) that often precedes the onset of classic motor symptoms by decades. There is no standardized algorithm for managing constipation in this patient population, nor are there dedicated treatments.
- 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.
- Parkinson's disease
Rehabilitation of Airway Protection in Parkinson's Disease
Testing therapy to improve airway protection problems in Parkinson’s
📍 Purdue University · West Lafayette, IN · +1 more US site · Get directions →
Airway protective disorders are a prevalent and progressive consequence of Parkinson's Disease (PD), and often result in aspiration pneumonia which is the leading cause of death in PD. Despite this, a large number of patients with PD do not access specialized services to address these critical deficits.
- COPD (chronic lung disease)
A Study to Learn About the Study Medicine Called PF-07275315 in People With Moderate to Severe Chronic Obstructive Pulmonary Disease
Testing how a study medicine affects safety and symptoms in people with COPD
📍 University of Alabama at Birmingham Lung Health Center · Birmingham, AL · +39 more US sites · Get directions →
The purpose of this clinical trial is to learn about the effects and safety of the study medicine PF-07275315 for the potential treatment of COPD. COPD is a condition that makes it difficult to breathe, which negatively impacts the quality of life and functioning of people who are affected.
- 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.
- Sickle cell disease
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Testing immune-suppressing treatment before stem cell transplant for people with sickle cell disease
📍 Memorial Sloan Kettering at Basking Ridge (Consent only) · Basking Ridge, NJ · +5 more US sites · Get directions →
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD.
- Obesity / overweightHigh blood pressureCOPD (chronic lung disease)
Feasibility of Semaglutide in Advanced Lung Disease
Testing whether semaglutide can be given safely to people with advanced lung disease
📍 University of Pennsylvania Perelman School of Medicine · Philadelphia, PA · Get directions →
The goal of this clinical trial is to learn whether semaglutide, an FDA-approved treatment for diabetes and obesity, is feasible and tolerable in patients with advanced lung disease. The main question\[s\] it aims to answer are: 1.
- Sickle cell disease
A 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.
- COPD (chronic lung disease)
A Study to Evaluate Solrikitug in Participants With COPD (ZION)
Testing solrikitug in people with COPD to check safety and effects
📍 University of Alabama at Birmingham Hospital · Birmingham, AL · +39 more US sites · Get directions →
A Randomized, Double-blind, Placebo-controlled, Multiple Dose-Ranging Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Solrikitug in Participants with Chronic Obstructive Pulmonary Disease (COPD).
- Sickle cell disease
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
Testing a partial stem cell transplant from matched donors for severe sickle cell disease or
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens.
- Sickle cell disease
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Testing how safe escalating doses of fostamatinib are in people with stable sickle cell disease
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves.
- 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.
- Parkinson's disease
This Clinical Trial is Designed to Evaluate the Safety, Tolerability, Feasibility and Preliminary Efficacy of NouvNeu001 (Human Dopaminergic Progenitor Cells Injection) in Patients With Parkinson's Disease.
Testing an injected cell treatment for safety in people with advanced Parkinson’s
📍 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.
- Sickle cell disease
Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin
Testing empagliflozin to treat kidney disease related to sickle cell
📍 University of Illinois Chicago, Sickle Cell Center · Chicago, IL · Get directions →
Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA.
- Dementia / Alzheimer'sParkinson's disease
18F-mFBG Cardiac Uptake With Lewy Body Dementia
Testing a heart imaging tracer to see how Lewy body dementia affects nerve signals
📍 Houston Methodist Neurological Institute · Houston, TX · Get directions →
This is a Phase 2 study evaluating the positron-emitting radiopharmaceutical 18F-mFBG as an imaging agent for quantification of the effect of neurodegenerative diseases on myocardial sympathetic innervation. Effectiveness of 18F-mFBG imaging of the heart will be judged in terms of the quantitative difference between results for subjects with Lewy body and non-Lewy body neurologic disease as compared to historical data for healthy control subjects.
- Sickle cell disease
Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody
Testing a transplant plan with daratumumab to reduce blood cell failure in sickle cell disease
📍 Children's National Hospital · Washington D.C., DC · Get directions →
This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies.
- Parkinson's disease
Tenapanor in Synucleinopathy-Related Constipation
Testing tenapanor for constipation related to Parkinson’s disease
📍 Cedar Valley Digestive Health Center · Waterloo, IA · Get directions →
Investigation of tenapanor as a potential treatment for synucleinopathy-associated constipation
- Parkinson's disease
Evaluation of Tau-Pathology in Sporadic and LRRK2 Parkinson's Disease
Testing how much brain tau buildup is in people with Parkinson’s using PET scans
📍 Institute for Neurodegenerative Disorders / XingImaging, LLC · New Haven, CT · Get directions →
The study aims to evaluate the burden of tau pathology in people with Sporadic and LRRK2 PD via in vivo imaging using the tau tracer, \[18F\]PI-2620, and a high resolution PET camera, NeuroEXPLORER.
- Parkinson's disease
A Study to Evaluate the Effectiveness of Two Doses of AP-472 as Adjunctive Therapy to Levodopa in Parkinson's Disease (PD) Participants With Motor Fluctuations
Testing two doses of an add-on medicine to levodopa for Parkinson’s with motor fluctuations
📍 University of Arkansas for Medical Sciences · Little Rock, AR · +24 more US sites · Get directions →
This is a Phase 2 study in people with Parkinson's disease who experience motor fluctuations while taking levodopa. The study will evaluate how effective two different doses of the study drug AP-472 are when added to levodopa treatment, compared with a placebo.
- 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.
- COPD (chronic lung disease)
A Study of WIN378 in Participants With Moderate to Severe COPD (Sirius)
Testing the right dose of a long-acting medicine for moderate to severe COPD
📍 WB Contracted Clinical Research Site · Miami Lakes, FL · +3 more US sites · Get directions →
This study is trying to identify the right dose of a long-acting medicine called WIN378 for people with moderate - severe chronic obstructive pulmonary disease (COPD). WIN378 blocks the action of a protein called TSLP that causes inflammation in the lung and may contribute to COPD control and symptoms.
- 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
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