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97 studies
Recruiting now, in plain language. Every study is watched over by an independent ethics board.
- Crohn's / ulcerative colitis (IBD)
Statin Therapy in Primary Sclerosing Cholangitis (PSC): a Multi-omics Study
Studying how statin treatment affects biology in people with primary sclerosing cholangitis
📍 Stanford University · Stanford, CA · Get directions →
PSC is a liver disease that has no medical cure. Patients with PSC are at a greatly increased risk of cancer and infection.
- 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.
- Crohn's / ulcerative colitis (IBD)
Clinical, Imaging, and Endoscopic Outcomes of Children Newly Diagnosed With Crohn's Disease
Looking at tests and scans in children newly diagnosed with Crohn’s disease
📍 Phoenix Children's Hospital · Phoenix, AZ · +21 more US sites · Get directions →
Crohn's disease (CD) is a condition that causes inflammation (swelling, redness) of the lining and wall of the small intestine, large intestine, or both. CD may be associated with abdominal cramps/pain, diarrhea, blood in the stool, weight loss, or delayed growth in children.
- 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
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.
- Crohn's / ulcerative colitis (IBD)
A Study of Vedolizumab With Tofacitinib in Adults With Ulcerative Colitis (UC)
Testing a medicine combination and then a single medicine for ulcerative colitis in adults
📍 Digestive Health Specialsits · Dothan, AL · +38 more US sites · Get directions →
The main aim of this study is to learn about the effect of treatment with vedolizumab IV (vedolizumab) together with tofacitinib in adults with moderate and severe ulcerative colitis (UC). Another aim is to learn about treatment with Vedolizumab alone after the double treatment.
- Dementia / Alzheimer'sParkinson's disease
PET Imaging Evaluation of [11C]SY08
Testing a PET scan tracer to detect alpha-synuclein protein clumps in dementia and Parkinson’s
📍 MGH · Charlestown, MA · Get directions →
The overall goal of the proposed research is to evaluate the use of \[11C\]SY08 as a PET radiotracer for aggregated alpha synuclein (αS) in individuals with Parkinson's disease (PD), Multiple system atrophy (MSA), Dementia with Lewy Bodies (DLB) and healthy controls. The purpose of this study is to evaluate the use of \[11C\]SY08 as a PET radiotracer for αS fibrils in individuals with PD, MSA, DLB and healthy controls.
- 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.
- Parkinson's disease
Evaluation of [¹⁸F]MODAG-009 PET Imaging in Synucleinopathies
Testing an injection for brain scans to study Parkinson’s disease and safety
📍 Institute for Neurodegenerative Disorders and XingImaging, LLC · New Haven, CT · Get directions →
This is a single-center, open-label clinical study designed to evaluate the imaging characteristics and safety of \[¹⁸F\]MODAG-009 in participants with Parkinson's disease (PD), Multiple system atrophy (MSA), and Healthy controls (HC). Approximately 13 participants will be enrolled in this study.
- Crohn's / ulcerative colitis (IBD)
An Early-Stage Study in Multiple Clinics of How Afimkibart May Affect the Body's Processing of Medicines That Rely on Cytochrome P450 Enzymes in Participants With Ulcerative Colitis
Testing how afimkibart in people with ulcerative colitis affects drug processing in the liver
📍 Erick H. Alayo Medical Corporation - Gastro SB Clinic · Chula Vista, CA · +1 more US site · Get directions →
The purpose of this study is to evaluate the disease-drug-drug interaction (DDDI) potential of afimkibart (also known as RO7790121). This will be assessed by the characterization of the pharmacokinetics (PK) of cytochrome P450 (CYP) enzyme substrates alone and after administration of afimkibart in participants with moderately to severely active ulcerative colitis (UC).
- 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).
- Crohn's / ulcerative colitis (IBD)
Effect of Statins on Crohn's Disease
Testing whether statins can reduce gut narrowing (strictures) in adults with Crohn’s disease
📍 Stanford University · Stanford, CA · Get directions →
The goal of this clinical trial is to learn if statins work to prevent strictures in adults with Crohn's disease. The main question it aims to answer is: * Can statins reduce the formation of strictures in participants with stricturing Crohn's disease?
- Crohn's / ulcerative colitis (IBD)
Autologous Stem Cell Transplantation for Crohn's Disease
Testing stem cell treatment for severe Crohn's disease in teens and adults
📍 UPMC Prebyterian- Adult Gastroenterology · Pittsburgh, PA · +1 more US site · Get directions →
The objective of this study is to evaluate the safety and effectiveness of administering high-dose chemotherapy followed by infusion of autologous CD34-selected peripheral blood stem cells (PBSC) in pediatric and adult patients with severe Crohn's disease.
- 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.
- Crohn's / ulcerative colitis (IBD)
An Open-Label, Proof of Consent Study of Vorinostat for the Treatment of Mdoerate-to-Severe Crohn s Disease and Maintenance Therapy With Ustekinumab
Testing vorinostat in people with moderate to severe Crohn’s disease and using ustekinumab to
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Crohn s disease (CD) is an inflammatory bowel disease. It causes inflammation of the gut.
- Parkinson's disease
Open-label Study to Evaluate Brain α-Synuclein Deposition Using PET and [18F]MK-0947 in Parkinson's Disease
Testing a new PET brain scan to measure nerve protein buildup in Parkinson’s
📍 Invicro (dba Perceptive) · New Haven, CT · +1 more US site · Get directions →
This clinical study is being conducted to learn more about a new imaging drug called \[18F\]MK-0947, which is designed to help doctors see changes in the brain related to Parkinson's disease (PD). PD is a condition that affects movement, balance, and thinking.
- Crohn's / ulcerative colitis (IBD)
Treatment of Moderate to Severe Refractory Crohn's Disease
Testing different doses of a new treatment for Crohn’s disease
📍 Mayo Clinic Arizona · Scottsdale, AZ · +12 more US sites · Get directions →
This research study is testing an investigational research product called TRX103 as a possible treatment for individuals suffering from Crohn's Disease (CD). The primary purpose of this study is to learn how safe and effective different doses of TRX103 are when administered to individuals with CD.
- 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.
- 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.
- Crohn's / ulcerative colitis (IBD)
Cranberry and Gut Health in Crohn's Disease
Testing whether a cranberry supplement helps gut health in people with Crohn’s disease
📍 UMASS medical center · Worcester, MA · Get directions →
This study is investigating whether a cranberry-based dietary supplement, rich in polyphenols and fiber, can enhance gut health in individuals with Crohn's disease. People with Crohn's disease often have an imbalance in their gut microbiome (the community of bacteria in the gut).
- 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).
- 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.
- 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.
- 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
- 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.
- Crohn's / ulcerative colitis (IBD)
VECTORS - A Study to Evaluate Transmural Healing as a Treatment Target in Crohn's Disease
Testing whether healing through the whole bowel wall is a good treatment goal in Crohn’s disease
📍 TLC Clinical Research Inc - Los Angeles · Los Angeles, CA · +2 more US sites · Get directions →
Transmural healing (TMH) is recognized as a potentially important measure of Crohn's disease (CD) activity but not a formal target. Observational studies suggest that TMH may be associated with better long-term outcomes.
- 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.
- Crohn's / ulcerative colitis (IBD)
Upadacitinib Combined With Corticosteroids vs Corticosteroid Monotherapy Induction for Inpatients and Outpatients With Acute Severe Ulcerative Colitis
Testing the best medication sequence for acute severe ulcerative colitis using a drug plus steroids
📍 University of Michigan · Ann Arbor, MI · Get directions →
This trial is being conducted to learn more about the optimal sequence of various medications in the management of acute severe ulcerative colitis (ASUC). This research is studying multiple drugs already approved by the Food and Drug Administration (FDA).
- 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.
- Crohn's / ulcerative colitis (IBD)💵 May compensate
Open-label Single-arm Study to Assess the Efficacy of Mirikizumab in Patients With Inflammatory Strictures Due to CD
Testing whether mirikizumab is safe and works for people with Crohn’s strictures
📍 Digestive & Liver Center of Florida · Orlando, FL · +1 more US site · Get directions →
This is an open-label, single-arm, phase 4 study to assess the safety and efficacy of mirikizumab in approximately 60 participants with stricturing CD.
- 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.
- Parkinson's disease
Longitudinal TSPO PET Imaging With [18F]DPA-714 in PPMI (PPMI DPA-714 PET Imaging)
Studying how a brain imaging tracer binds in early Parkinson’s over 24 months
📍 UAB · Birmingham, AL · Get directions →
The overall goal of this protocol is to investigate \[18F\]DPA-714 binding in prodromal and early manifest Parkinson's Disease (PD) and to determine the baseline and change from baseline in \[18F\]DPA-714 binding in PD participants during a 24-month interval. Primary Objectives * To compare \[18F\]DPA-714 binding in prodromal and manifest PD and healthy volunteers.
- Crohn's / ulcerative colitis (IBD)
Bowel Preparation for Colonoscopy Among Individuals With Crohn's and Ulcerative Colitis Disease.
Comparing two laxatives for bowel prep before colonoscopy in IBD
📍 University of Manitoba · Winnipeg, Manitoba · Get directions →
The purpose of this study is to compare how effective and how tolerable two different bowel preparation laxatives are for colonoscopy. The aim is to compare oral sulfate solution (OSS) to another laxative called 2L polyethylene glycol (PEG) solution to see which is more effective and more tolerable by individuals with IBD (Crohn's disease or Ulcerative colitis).
- Crohn's / ulcerative colitis (IBD)
A Study of Vedolizumab in Adults With Ulcerative Colitis or Crohn's Disease in the Community Setting
Testing whether treatment for ulcerative colitis or Crohn's helps symptoms go away after 14 weeks
📍 Gastro Health Research- St. Vincents East · Birmingham, AL · +39 more US sites · Get directions →
Ulcerative Colitis (UC) and Crohn's Disease (CD) are long-term conditions in the gut that can cause diarrhea, swelling (inflammation), bleeding from the anus, and belly pain. The main aim of this study is to check for how many participants with UC and CD signs and symptoms disappear after 3.5 months (14 weeks) of treatment with Vedolizumab (this is called remission).
- Crohn's / ulcerative colitis (IBD)
R-3750 in Patients With Mild to Moderate Ulcerative Colitis
Testing the safety of an oral probiotic in people with mild to moderate ulcerative colitis
📍 Amicis Research Center · Valencia, CA · +4 more US sites · Get directions →
The goal of this study is to determine the safety and tolerability of orally taken probiotic (R-3750) in patients with mild to moderate ulcerative colitis. Patients will take an oral dosage of probiotic (R-3750) and provide patient-reported and physician scored measures of their colitis.
- 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.
- 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.
- Sickle cell disease
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Measuring how long red blood cells last in sickle cell disease
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early.
- Crohn's / ulcerative colitis (IBD)
Fecal Microbiota Transplantation in an Expanded Ulcerative Colitis Population
Testing fecal transplant for ulcerative colitis in a feasibility study
📍 University of Calgary · Calgary, Alberta · Get directions →
This is a multi-centre, randomised controlled trial comparing fecal microbiota transplantation to placebo in an expanded ulcerative colitis population: a feasibility study (FRONTIER-UC) to determine whether a full-scale randomized controlled trial (RCT) to investigate fecal microbiota transplantation (FMT) in ulcerative colitis (UC) is feasible.
- 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.
- Crohn's / ulcerative colitis (IBD)
Beta-Hydroxybutyrate Feasibility Treating IBD
Testing whether taking a ketone supplement for 4 weeks is doable in people with Crohn’s disease
📍 University of Texas at Austin · Austin, TX · Get directions →
This clinical trial aims to understand the feasibility of patients taking ketone body supplement beta-hydroxybutyrate (BHB) for 4 weeks with a confirmed diagnosis of Crohn's disease and starting new therapy for active disease. The main questions it aims to answer are: * BHB supplementation will be feasible and acceptable to patients.
- Crohn's / ulcerative colitis (IBD)
Study of XmAb942 in Healthy Participants and Participants With Ulcerative Colitis
Testing the safety and effects of a drug called XmAb942 in ulcerative colitis
📍 Xencor Investigative Site · Scottsdale, AZ · +23 more US sites · Get directions →
Brief summary The Phase 1 study described herein will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of XmAb942 in healthy volunteers (Parts A and B). Part C of this study will be a Phase 2 study to evaluate XmAb942 in participants with ulcerative colitis (UC).
- 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.
- Crohn's / ulcerative colitis (IBD)
68Ga-FAPI-46 PET/CT for Assessing Small Bowel Fibrostenosis in Crohn's Disease
Testing a scan to see early scarring in the small bowel in Crohn’s
📍 Mayo Clinic · Rochester, MN · Get directions →
This study is a prospective, case-control study evaluating whether the PET radiotracer 68Ga-FAPI-46 can detect fibrostenosing Crohn's disease in the small bowel. The goal is to determine whether areas of early or developing fibrosis ("pre-stricture" changes) demonstrate uptake of the tracer, which binds to fibroblast activation protein (FAP).
- Crohn's / ulcerative colitis (IBD)
Feasibility Pilot Sequential Multiple Assignment Randomized Trial (SMART) for Acute Severe Ulcerative Colitis
Testing if personalized treatment plans for severe ulcerative colitis in hospital are workable
📍 University of Michigan · Ann Arbor, MI · Get directions →
The goal of this trial is to create personalized treatments for each patient admitted to the hospital with acute severe ulcerative colitis (ASUC). The study will test the feasibility and acceptability of these treatment strategies among patients and physicians so that the study team can later do a larger trial to test whether the medication treatment pathways help patients avoid colectomy while ensuring patient's are safe.
- 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.
- 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
- 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.
- Crohn's / ulcerative colitis (IBD)
De-escalation of Ustekinumab Therapy in Patients With Crohn's Disease and Ulcerative Colitis
Testing whether reducing injections of ustekinumab can keep Crohn’s or colitis in remission
📍 MUHC - Montreal General Hospital · Montreal, Quebec · Get directions →
The goal of this clinical trial is to evaluate whether disease remission can be maintained when biologic therapy is reduced in patients with Crohn"s disease (CD) and ulcerative colitis (UC) taking ustekinumab (UST). The main question it aims to answer is: Can we de-escalate UST subcutaneous dose either from every 4 weeks (Q4) to every 8 weeks (Q8) or every 8 weeks (Q8) to every 12 weeks (Q12) in CD or UC patients in deep remission without loosing their response?
- 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.
- 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.
- Parkinson's disease
Title: A Multimodal Exercise Program With Gamification for Parkinson Disease (MEP+G)
Testing an 8-week exercise program with boxing and computer games for people with Parkinson disease
📍 Equilibrium Physical Therapy · Ventura, CA · Get directions →
In this pilot study the research will explore whether exercises and the outcome measures would be acceptable to conduct a more detailed research study. This 8 week pilot study will utilize a comprehensive exercise program that includes non-contact boxing along with computerized games for individuals with Parkinson Disease.
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