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53 studies
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- Asthma
The Safety, Feasibility, and Repeatability of Inhaled ATP Cough Challenges
Testing whether inhaling ATP to trigger cough is safe and repeatable in people with asthma
📍 Respiratory Research Lab · Hamilton, Ontario · Get directions →
The inhalation of adenosine 5'-triphosphate (ATP) to evoke cough (ATP cough challenge) is becoming increasingly used as a tool to measure cough hypersensitivity in patients with chronic cough. However, the safety, feasibility, and repeatability of this procedure is not widely known.
- 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.
- Fatty liver disease (NASH/MASH)
A Study to Evaluate ALN-CIDEB in Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease or With Metabolic Dysfunction-Associated Steatohepatitis (MASLD/MASH)
Testing an experimental medicine for MASLD or MASH in adults
📍 Arizona Liver Health · Chandler, AZ · +1 more US site · Get directions →
This study is researching an experimental drug called ALN-CIDEB, also referred to as "study drug". The study is focused on participants with metabolic dysfunction-associated steatotic liver disease (MASLD) (Part A) and metabolic dysfunction-associated steatohepatitis (MASH) (Part B).
- Fatty liver disease (NASH/MASH)
A Research Study of a Potential New Medicine (NNC4005-0001) for Liver Disease in Adult Participants With Increased Body Weight and Liver Fat
Testing a new medicine for liver disease in adults with extra body fat
📍 Altasciences Clinical Company, Inc · Montreal, Quebec · Get directions →
The purpose of this clinical study is to find out if NNC4005-0001 is well-tolerated and safe for people who have increased body weight and increased liver fat. Participants will receive either NNC4005-0001, which is the treatment being tested, or a placebo, which is a treatment that contains no active medicine.
- Type 2 diabetesFatty liver disease (NASH/MASH)
A Phase 1b Study of QX1206 in T2DM Patients With NAFLD
Testing QX1206 in people with type 2 diabetes and fatty liver
📍 Centricity Research Toronto LMC. · Toronto, · Get directions →
This is an open label phase 1b trial of QX1206 in patients with T2DM and with NAFLD. Laboratory tests and other measurements will be assessed prior to the first dose of study treatment and throughout the study to determine the recommended phase 2 dose.
- 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.
- Asthma
A Study of CDX-622 in Participants With Mild to Moderate Asthma
Testing the safety and how a medicine affects the body in adults with mild to moderate asthma
📍 University of California, San Francisco · San Francisco, CA · +3 more US sites · Get directions →
This is a study to determine the safety, pharmacodynamics (PD), and pharmacokinetics (PK) of CDX-622 in adults with mild to moderate asthma.
- 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.
- Asthma
A Study to Learn How Different Amounts of the Study Medicine Called PF-08103402 Are Tolerated and Act in the Body in Healthy Adults or Adults With Mild To-moderate Asthma
Testing how different doses of a new medicine affect safety and the body in people with mild to
📍 Pfizer Clinical Research Unit - New Haven · New Haven, CT · Get directions →
The purpose of this study is to learn about the safety of a new study medicine called PF-08103402 in healthy adults (do not have disease) and or in adults with mild-to-moderate asthma. This is the first time the study medicine is being given to people.
- 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).
- 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.
- Obesity / overweightFatty liver disease (NASH/MASH)💵 May compensate
Human Models of Selective Insulin Resistance: Alpelisib, Part I
Testing how insulin works in healthy people versus people at risk for type 2 diabetes
📍 Columbia University Irving Medical Center · New York, NY · Get directions →
The goal of this clinical trial is to understand how the blood sugar-lowering hormone insulin works in healthy adults versus those who are at risk for type 2 diabetes. The study will use a drug called alpelisib, which interferes with insulin's actions in the body, to answer the study's main question: does the liver continue to respond to insulin's stimulation of fat production even when it loses the ability to stop making glucose (sugar) in response to insulin.
- 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.
- 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.
- 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.
- Fatty liver disease (NASH/MASH)💵 May compensate
Evaluating the Pharmacokinetics and Safety of Miricorilant
Testing how a drug works in the body and its safety in adults with MASH
📍 Site# 433 · San Antonio, TX · Get directions →
A Phase 1b, Open-Label Study Evaluating the Pharmacokinetics and Safety of Miricorilant in Adult Patients With Presumed Metabolic Dysfunction-Associated Steatohepatitis (MASH)
- Fatty liver disease (NASH/MASH)
Bicalutamide Therapy in Young Women With NAFLD and PCOS
Testing bicalutamide in young women with fatty liver inflammation (NASH) and PCOS
📍 University of California San Francisco · San Francisco, CA · Get directions →
Nonalcoholic steatohepatitis (NASH), or fat-related liver inflammation and scarring is projected to be the leading cause of cirrhosis in the United States (U.S.) within the next few years. Women are at disproportionate risk for NASH, with approximately 15 million U.S.
- 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.
- 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.
- 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.
- 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).
- 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.
- Asthma
Phase 1 Study Evaluating Safety and Pharmacokinetics of ASY202 in Adults With Asthma
Testing safety and how a single inhaled dose works in adults with asthma
📍 Sun City Clinical Research · Glendale, AZ · +1 more US site · Get directions →
This is a phase 1 study, randomized, double-blind, placebo-controlled, 2 treatment, 2 period crossover study to evaluate the pharmacokinetics, safety, and tolerability of a single inhaled dose of ASY202 in adults with stable asthma. Following screening, eligible participants will be enrolled and randomized to one of two treatment sequences.
- 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.
- 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).
- 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.
- Crohn's / ulcerative colitis (IBD)
Low Dose IL-2 for the Treatment of Crohn's Disease
Testing low-dose interleukin-2 to find a safe dose for Crohn's disease
📍 Boston Children's Hospital · Boston, MA · +2 more US sites · Get directions →
The purpose of this study is to determine the safety and maximum effective dose (MED) of Interleukin-2 in subjects with moderate-to-severe crohn's disease.
- 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.
- 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.
- Fatty liver disease (NASH/MASH)
A Study of Efimosfermin Alfa in Adults With Hepatic Impairment
Testing how a single dose affects drug levels and safety in adults with liver impairment and
📍 GSK Investigational Site · Rialto, CA · +2 more US sites · Get directions →
This study is designed to study the pharmacokinetic (PK) and safety profiles of a single dose of efimosfermin alfa in participants with varying degrees of Hepatic Impairment (HI) (assessed by Child-Pugh score) due to steatotic liver disease, with and without significant alcohol consumption.
- Obesity / overweightFatty liver disease (NASH/MASH)💵 May compensate
Effect of Insulin Lowering on Lipogenesis
Testing whether lowering insulin for one week changes liver fat in people with obesity and MASLD
📍 Columbia University Irving Medical Center · New York, NY · Get directions →
The goal of this clinical trial is to compare a one-week course of diazoxide (2 mg/kg per dose x 14 doses) and placebo in people with obesity and insulin resistance (IR) with metabolic dysfunction-associated steatotic liver disease (MASLD). The main question it aims to answer are how mitigation of compensatory hyperinsulinemia with diazoxide affects hepatic de novo lipogenesis, a major contributor to MASLD pathophysiology.
- Crohn's / ulcerative colitis (IBD)
Study to Evaluate the Fecal Microbiota Transplantation (FMT) in the Treatment of Ulcerative Colitis
Testing frozen stool transplant methods for people with active ulcerative colitis
📍 The University of Texas Health Science Center at Houston · Houston, TX · Get directions →
The study is to evaluate the safety, feasibility, and preliminary efficacy of frozen FMT delivery via retention enema compared to lyophilized powder given in oral capsules as induction FMT in subjects with active UC. This study will also determine changes in microbiome (diversity and genera) and proportion of antibody-coated microbiota from baseline to after completion of FMT.
- Asthma
Pilot Study to Identify the Mediators and Inflammatory Cell Surface Receptors Involved in Allergic Airway Inflammation
Studying what body signals and immune cells drive asthma-related airway inflammation
📍 Massachusetts General Hospital · Boston, MA · Get directions →
Asthma is a heterogeneous disorder in which multiple potential inflammatory pathways contribute to airway obstruction. The biological basis for airway inflammation is the subject of intensive investigation.
- Sickle cell disease
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
Testing a new transplant plan using immune-suppressing treatment before a blood stem cell transplant
📍 City of Hope Medical Center · Duarte, CA · Get directions →
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.
- Sickle cell disease
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Testing whether using mismatched donor stem cells works for severe sickle cell disease and other
📍 Children's Hospital of Pittsburgh of UPMC · Pittsburgh, PA · Get directions →
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease.
- Lupus (SLE)Rheumatoid arthritisMultiple sclerosis (MS)
Testing an Immunotherapy Anti-cancer Drug, Nivolumab, for Advanced Cancers in Patients With Autoimmune Disorders, AIM-NIVO
Testing nivolumab for side effects and cancer response in people with advanced cancer and autoimmune
📍 University of Alabama at Birmingham Cancer Center · Birmingham, AL · +39 more US sites · Get directions →
This phase Ib trial studies the side effects of nivolumab and to see how well it works alone and in combination with other treatments, such as ipilimumab, cabozantinib, platinum containing therapy, and fluoropyrimidine, in treating patients with autoimmune disorders and cancer that has spread from where it first started (primary site) to nearby tissue, lymph nodes, or distant parts of the body (advanced), to other places in the body (metastatic) or cannot removed by surgery (unresectable). Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
- Crohn's / ulcerative colitis (IBD)
Pilot and Feasibility Study of 2'-FL as a Dietary Supplement in IBD Patients Receiving Stable Maintenance Anti-TNF Therapy
Testing whether a supplement called 2'-FL is safe and well tolerated in IBD
📍 Connecticut Children's Medical Center · Hartford, CT · +2 more US sites · Get directions →
Randomized, placebo-controlled dose-ranging study of 2'-FL in IBD, Crohn's Disease (CD) and ulcerative colitis (UC). The overarching hypothesis is that 2'-FL supplementation in IBD will be safe and well tolerated, while increasing fecal Bifidobacterium abundance and butyrate in a dose dependent manner.
- Sickle cell disease
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Testing how oral medicines may help people with high-risk sickle cell disease
📍 University of Illinois at Chicago College of Medicine · Chicago, IL · Get directions →
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
- Crohn's / ulcerative colitis (IBD)
Stem Cell Transplantation in Crohn's Disease
Testing stem cell transplant after high-dose immune treatment for severe Crohn's disease
📍 Cedars-Sinai Medical Center · Los Angeles, CA · Get directions →
Unfortunately, some patients with Crohn's disease (CD) fail to respond to the best clinical treatments and some only experience temporary benefit. For severe Crohn's disease, there is an experimental treatment called "high dose immunoablation" followed by autologous hematopoietic stem cell transplantation (HSCT).
- High blood pressureCOPD (chronic lung disease)Asthma
Hyperpolarized 129Xe MR Imaging of Lung Function in Healthy Volunteers and Subjects With Pulmonary Disease
Testing a lung scan method using hyperpolarized oxygen gas MRI in healthy and people with COPD or
📍 University of Kansas Medical Center · Kansas City, KS · Get directions →
The purpose of this study is to develop and evaluate the usefulness of hyperpolarized (HP) 129Xe gas MRI for regional assessment of pulmonary function.
- Sickle cell disease
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Testing gene correction in a person’s own blood-forming stem cells for severe sickle cell disease
📍 Children's Hospital Los Angeles · Los Angeles, CA · +5 more US sites · Get directions →
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
- Sickle cell disease
PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD
Using PET scans to find blood vessel blockage markers in sickle cell disease
📍 UPMC · Pittsburgh, PA · Get directions →
The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET).
- Crohn's / ulcerative colitis (IBD)
Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell Intravenous Infusion for IBD
Testing safety of an IV infusion of lab-grown donor stem cells for IBD
📍 Medical Surgical Associates Center · St John's, · Get directions →
This trial will study the safety and efficacy of intravenous infusion of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of inflammatory bowel disease
- COPD (chronic lung disease)Asthma
Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cells for Pulmonary Diseases
Testing the safety of an IV stem cell treatment for lung diseases like COPD and asthma
📍 Medical Surgical Associates Center · St John's, · Get directions →
This trial will study the safety and efficacy of intravenous infusion of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of Pulmonary Diseases
- Fatty liver disease (NASH/MASH)💵 May compensate
Evaluation of Miricorilant on Liver Fat in Patients With MASLD
Testing miricorilant’s effect on liver fat in people with MASH
📍 University of Missouri · Columbia, MO · Get directions →
A Phase 1, Open-Label Study Evaluating the Effect of Miricorilant on Hepatic Lipids in Patients with Presumed Metabolic Dysfunction-Associated Steatohepatitis (MASH)