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📍 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.

📍 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.

📍 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).

📍 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.

📍 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.

📍 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).

📍 Mayo Clinic in Arizona · Scottsdale, AZ · +4 more US sites · Get directions →

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and therapeutic activity of GI-102 as a single agent and in combination with conventional anti-cancer drugs, pembrolizumab or trastuzumab deruxtecan(T-DXd) over a range of advanced and/or metastatic solid tumors.

📍 Research Site · Glendale, CA · +4 more US sites · Get directions →

This study will evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single ascending doses (SAD) and multiple ascending doses (MAD) of AZD4248 administered as an oral solution and intravenous (IV) infusion. Additionally, the study investigates the non-interventional feasibility of home measurement of serum creatinine in participants with diabetic kidney disease (DKD).

📍 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.

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

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

📍 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.

📍 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.

📍 Stanford · Palo Alto, CA · +2 more US sites · Get directions →

A phase I/II, open-label, study to determine the safety and preliminary efficacy of orellanine in patients with metastatic clear-cell or papillary renal carcinoma who have failed standard-of-care therapy. All participants must have end-stage kidney disease and be receiving stable chronic hemodialysis.

📍 Quotient Sciences Sea View · Miami, FL · Get directions →

The purpose of this first-in-human (FIH) study is to evaluate safety, tolerability, pharmacokinetic (PK) of OJR520.

📍 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.

📍 Northwestern Memorial Hospital · Chicago, IL · +1 more US site · Get directions →

The purpose of this study is to evaluate the safety and efficacy of the 10 GE Xenokidney in patients with ESRD who are either not eligible for conventional allogeneic kidney transplantation (Group 1) or are on an Organ Procurement and Transplantation Network (OPTN) kidney transplant waitlist, but are more likely to die or go untransplanted within 5 years than receive a kidney transplant (Group 2). The study consists of xenotransplantation followed by a 24-week Post-transplant Follow up Period (Part A) to evaluate the efficacy and safety objectives followed by a Long-term Follow-up Period (Part B) to evaluate participant survival, 10 GE Xenokidney survival, and screening for zoonotic infections.

📍 Northwestern University · Evanston, IL · +1 more US site · Get directions →

People spend approximately one-third of their lives asleep, yet sleep is often underused as an opportunity to support psychological well-being. Contemplative traditions, including Tibetan Dream Yoga, have developed practices that use waking imagination and lucid dreaming to explore perception, awareness, and habitual patterns of thinking.

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

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

📍 University of Cincinnati · Cincinnati, OH · Get directions →

This study will evaluate the safety, PK, and efficacy of AT 1501 in patients undergoing kidney transplantation.

📍 Panax Clinical Research ( Site 0003) · Miami Lakes, FL · +2 more US sites · Get directions →

The goal of this trial is to measure what happens to 1 or 2 doses of MK-2828 in a person's body over time (pharmacokinetic or PK trial). Researchers want to learn if the PK of people with certain types of kidney disease is similar to the PK of healthy people.

📍 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.

📍 Floridian Clinical Research, LLC · Miami Lakes, FL · +1 more US site · Get directions →

The purpose of the study is to assess the amount of Eloralintide (LY3841136) that reaches the bloodstream and the time it takes for the body to get rid of it when given to participants with renal (kidney) impairment and to healthy participants. The study drug will be administered subcutaneously (SC) (under the skin).

📍 SouthCoast Research Center, Inc. · Miami, FL · +1 more US site · Get directions →

The goal of this clinical trial is to learn about the safety (good or bad) of giving two AION-301 intravenous (IV) infusions, in adults with Stage 3 Chronic Kidney Disease (CKD). It will also help to learn if AION-301 reduces the symptoms of CKD and/or progression.

📍 Mayo Clinic · Rochester, MN · +1 more US site · Get directions →

The purpose of this study is to establish if concomitant renal and vascularized urinary bladder allograft transplantation is feasible.

📍 Floridian Clinical Research · Miami Lakes, FL · +1 more US site · Get directions →

This study will be conducted to assess the safety and pharmacokinetics of INCB123667 when administered orally to adult participants with severe renal impairment or end stage renal disease.

📍 UPMC Presbyterian · Pittsburgh, PA · +1 more US site · Get directions →

The purpose of this study is to determine whether a lung transplantation prior to bone marrow transplantation (BMT) would allow for restoration of pulmonary function prior to BMT, allowing to proceed to BMT, to restore hematologic function.

📍 Brigham and Women's Hospital · Boston, MA · Get directions →

The aim of this protocol is to assess the presence and severity of primary aldosteronism pathophysiology in patients with type 2 diabetes who have, or are at-risk for developing, chronic kidney disease.

📍 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.

📍 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).

📍 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.

📍 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).

📍 The University of Chicago · Chicago, IL · Get directions →

Single arm- subject treated with Tegoprubart and everolimus. The purpose of this research is to gather information on the safety and effectiveness of investigational regimen containing 2 experimental components: * An investigational drug called Tegoprubart and * Human pancreatic islet cells Both Tegoprubart and human pancreatic islet cells are considered investigational because they are not approved for use in the United States by the Food and Drug Administration (FDA).

📍 Mayo Clinic in Florida · Jacksonville, FL · Get directions →

Researchers are evaluating the safety of allogeneic Adipose Derived Mesenchymal Stem Cells (AMSC) use during hemodialysis arteriovenous fistula and arterial bypass creation and its efficacy on improving access maturation and primary anastomotic patency.

📍 New York University Langone Health · New York, NY · Get directions →

The purpose of this study is to evaluate the safety and efficacy of the GGTA1 KO Thymokidney in patients with end-stage renal disease (ESRD) who are either not eligible for conventional allogeneic kidney transplantation (Group 1) or are on an Organ Procurement and Transplantation Network (OPTN) kidney transplant waitlist, but are more likely to die or go untransplanted within 5 years than receive a kidney transplant (Group 2). The study consists of xenotransplantation followed by a 24-week Post-transplant Follow-up Period (Part A) to evaluate the efficacy and safety objectives followed by a Long-term Follow-up Period (Part B) to evaluate participant survival, GGTA1 KO Thymokidney survival, and screening for zoonotic infections.

📍 Atlanta VA Medical Center · Decatur, GA · Get directions →

The purpose of this study is to find out why patients with chronic kidney disease (CKD) have poor exercise capacity and what causes an increase in blood pressure during exercise (i.e. increased adrenaline levels, or decreased ability of blood vessels to dilate).

📍 The University of Texas Health Science Center at San Antonio · San Antonio, TX · Get directions →

The study will test and compare the efficacy of a single essential amino acid valine with a combination of essential amino acids (EAA) supplement on fatigue, frailty, and cognitive function in end-stage kidney disease (ESKD) patients undergoing hemodialysis (HD) treatment.

📍 Adia Med Of Winter Park · Winter Park, FL · Get directions →

The goal of this clinical trial is to learn whether a new regenerative treatment called AdiaVita, made from umbilical cord blood-derived stem cells and exosomes combined with glutathione, is safe and can help improve kidney function in adults with chronic kidney disease (CKD). In this condition, the kidneys gradually lose their ability to filter blood as well as they should.

📍 Ann & Robert H. Lurie Children's Hospital of Chicago · Chicago, IL · Get directions →

The goal of this study is to learn if a clinical trial of sodium-glucose co-transporter 2 inhibitors (SGLT2i) is possible in youth with chronic kidney disease (CKD). The investigators also plan to explore whether treatment with SGLT2i (Empagliflozin) helps improve risk factors for worsening kidney and heart disease.

📍 UCLA · Los Angeles, CA · Get directions →

The study seeks to determine if patients with a pre-existing, well-functioning kidney transplant from a HLA-identical living donor can be withdrawn from immunosuppressive medications without compromising allograft function through hematopoietic stem cell (HPSC) infusion from the same donor. HPSC infusion will be preceded by a conditioning regimen of total lymphoid irradiation (TLI) and rabbit anti-thymocyte globulin (rATG).

📍 Northwestern University · Chicago, IL · Get directions →

The goal of this study is to better understand the effects of a sodium-glucose transport protein 2 inhibitor, dapagliflozin, added on to standard of care on heart and lung function and circulating metabolites (substances created when our bodies break down food, drugs, or its own tissues) in patients with chronic kidney disease.

📍 Ohio State University Comprehensive Cancer Center · Columbus, OH · Get directions →

This phase 1B trial tests the safety, side effects and best dose of rh-Hsc70- BZLF1 peptide complex (OSU-2131) with Stimulon (Trademark) QS-21 and evaluates how well it works in preventing Epstein-Barr virus (EBV) infection and related cancers in healthy volunteers and patients awaiting a solid organ transplant. Currently, patients who receive an organ transplant receive immune suppression therapy which can make it harder for the body to fight infections.

📍 George E Wahlen VA Medical Center · Salt Lake City, UT · Get directions →

Many control mechanisms exist which successfully match the supply of blood with the metabolic demand of various tissues under wide-ranging conditions. One primary regulator of vasomotion and thus perfusion to the muscle tissue is the host of chemical factors originating from the vascular endothelium and the muscle tissue, which collectively sets the level of vascular tone.

📍 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.

📍 University of Virginia Health System · Charlottesville, VA · Get directions →

Hyper polarized Xenon-129 MRI will be directly compared to a radioactive Xe-133 scintigraphy to detect defects in lung ventilation from airflow limitation. This study is conducted as a pilot study with intention to conduct a larger clinical trial.

📍 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).

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

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

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

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

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

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

📍 Cedars-Sinai Medical Center · Los Angeles, CA · Get directions →

This is a prospective, single center, double blind, randomized, crossover feasibility study of oral ketamine versus placebo for the treatment of anxiety in patients with pancreatic cancer currently receiving or within 12 weeks of receiving cancer targeted therapy. The primary objective is to determine the feasibility of enrolling subjects and treatment adherence.

📍 University of Connecticut · Storrs, CT · Get directions →

To examine the extent to which Cannabidiol (CBD) enhances fear conditioning extinction in college undergraduates who show elevated social anxiety. Undergraduates who display elevated social anxiety on standard assessments will be recruited at the University of Connecticut.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.

📍 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.