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79 studies

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  • Kidney disease

    Oral Intradialytic Amino Acid Supplementation to Vitalize End-stage Kidney Disease Patients on Hemodialysis

    Testing amino acid supplements during hemodialysis for fatigue and thinking in end-stage kidney

    📍 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.

    ✓ Study care freeEarly Phase 1Ages 18–64Learn more →
  • Kidney disease

    📍 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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Dementia / Alzheimer'sParkinson's disease

    Phase 2 Trial of Tributyrin in People With Parkinson's Disease and Cognitive Impairments

    Testing whether a tributyrin supplement helps memory, thinking, and balance in adults with Parkinson

    📍 Domino's Farms · Ann Arbor, MI · Get directions →

    The goal of this clinical trial is to learn if taking a tributyrin supplement works to improve memory and thinking and walking and balance in adults with Parkinson disease Parkinson disease dementia. It will also learn about the safety of tributyrin supplementation.

    ✓ Study care freePhase 1Ages 45+Learn more →
  • Parkinson's disease

    Virtual tES Device Treatment for Parkinson's Disease Motor Symptoms

    Testing a non-invasive brain stimulation device to improve Parkinson’s movement problems

    📍 U LLC Headquarters · Cleveland, OH · Get directions →

    This study tests a new, non-invasive brain stimulation device called Sphere V3.0 to help improve movement problems in people with Parkinson's disease. The device delivers gentle electrical signals to the scalp using small electrodes placed on the head.

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

    A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)

    Testing different doses of DISC-3405 to check safety in sickle cell disease

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

    This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Parkinson's disease

    First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)

    Testing an experimental treatment for early Parkinson’s disease in adults

    📍 Montreal Neurological Institute and Hospital · Montreal, Quebec · Get directions →

    This study is researching an experimental drug called ALN-SNCA (called "study drug"). The study is focused on people with early Parkinson's disease, a disorder of the nervous system that affects movement.

    ✓ Study care freePhase 1Ages 50–80Learn more →
  • Parkinson's disease

    📍 University of Kentucky · Lexington, KY · Get directions →

    The investigators propose a Phase I single surgical-center, double-blinded randomized parallel clinical trial involving bilateral autologous peripheral nerve tissue (PNT) delivery into the NBM or the alternate target also affecting cognition in this population, the substantia nigra (SN), to address "repair cell" support of these areas. Twenty-four participants with idiopathic Parkinson's Disease (PD) who have selected, qualified and agreed to receive as standard of care deep brain stimulation (DBS) will be enrolled and randomly allocated to receive bilateral PNT deployment to either the NBM or SN at the time of DBS surgery.

    ✓ Study care freePhase 1Ages 45–75Learn more →
  • Sickle cell disease

    Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease

    Testing higher doses of VAS-101 in people with stable sickle cell disease

    💵 May compensate

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.

    ✓ Study care freePhase 1Ages 18–90Learn more →
  • 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.

    ✓ Study care freeEarly Phase 1Ages 50–80Learn more →
  • Kidney disease

    📍 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.

    ✓ Study care freePhase 1Ages 35–75Learn more →
  • Sickle cell disease

    A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)

    Testing the safety and how an oral carbon monoxide liquid works in sickle cell disease

    📍 Cevaxin - The Panama Clinic · Panama City, · Get directions →

    This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days.

    ✓ Study care freePhase 1Ages 18–55Learn more →
  • 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.

    ✓ Study care freeEarly Phase 1Ages 50–80Learn more →
  • Kidney disease

    Intradialytic Exercise Combined With Creatine Monohydrate in Patients With Chronic Kidney Disease Undergoing Hemodialysis.

    Testing whether exercise during dialysis and creatine help people with chronic kidney disease

    📍 IMSS-Bienestar State Haemodialysis Centre · Colima, Coliima · Get directions →

    This study aims to evaluate the effects of creatine monohydrate supplementation and an intradialytic exercise program on body composition, biochemical parameters, and functional capacity in patients with chronic kidney disease (CKD) undergoing maintenance hemodialysis. The study will compare the independent and combined effects of these interventions over an 8-week period to determine their potential benefits in this patient population.

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

    Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

    Testing whether extra arginine lowers pain in children with sickle cell

    📍 Children's Healthcare fo Atlanta at Hughes Spalding · Atlanta, GA · +1 more US site · Get directions →

    The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit.

    ✓ Study care freePhase 1Ages 7–21Learn more →
  • 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.

    ✓ Study care freeEarly Phase 1Ages 18–80Learn more →
  • Kidney disease

    A Study of Eloralintide (LY3841136) in Participants With Renal Impairment and in Participants With Normal Renal Function

    Testing how a diabetes medicine in the bloodstream changes in kidney disease

    📍 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).

    ✓ Study care freePhase 1Ages 18–85Learn more →
  • Sickle cell disease

    RH Genotype Matched RBC Transfusions

    Testing whether matching donor red blood cells by RH type helps people with sickle cell who need

    📍 Children's Hospital of Philadelphia · Philadelphia, PA · Get directions →

    To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.

    ✓ Study care freePhase 1Ages 1+Learn more →
  • Parkinson's disease

    A Study of LY4006896 in Healthy Participants and Participants With Parkinson's Disease

    Testing an IV medicine in healthy people and people with Parkinson’s

    📍 Collaborative Neuroscience Network - CNS · Los Alamitos, CA · +11 more US sites · Get directions →

    The purpose of this study is to generate evidence of the safety, tolerability, and pharmacokinetics/pharmacodynamics of IV LY4006896 compared with placebo in healthy participants and participants with Parkinson's disease.

    ✓ Study care freePhase 1Ages 30–85Learn more →
  • Kidney disease

    Concomitant Renal and Urinary Bladder Allograft Transplantation

    Testing whether transplanting a new kidney and bladder at the same time is possible for people with

    📍 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.

    ✓ Study care freeEarly Phase 1Ages 1–60Learn more →
  • Parkinson's disease

    SAD Study in Patients With Parkinson's Disease and Motor Fluctuations

    Testing a single dose of SER-252 in people with Parkinson’s disease

    💵 May compensate

    📍 Rocky Mountain Clinical Research · Englewood, CO · +3 more US sites · Get directions →

    This is a randomized, placebo-controlled, single ascending dose (SAD) study of SER-252 in participants with Parkinson's Disease (PD) and motor fluctuations.

    ✓ Study care freePhase 1Ages 40–80Learn more →
  • Sickle cell disease

    Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

    Testing a one-time blood stem cell transplant using gene editing for severe sickle cell disease

    📍 University of California, Los Angeles · Los Angeles, CA · +1 more US site · Get directions →

    This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).

    ✓ Study care freePhase 1Ages 12–35Learn more →
  • Kidney disease

    Inflammation REduction to Prevent cArdiovascular Injury in Renal Disease (REPAIR)

    Testing a daily anti-inflammatory medicine to reduce heart and blood vessel injury in people with

    📍 University of Alberta Hospital · Edmonton, Alberta · Get directions →

    Phase 1 basket trial including 2 open-label single-arm cohorts: REPAIR CKD cohort and REPAIR Dialysis cohort. Open label colchicine 0.3 mg daily for 8 weeks followed, in patients who tolerated the 0.3 mg dose, by forced titration to 0.6 mg daily for 8 weeks.

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

    Feasibility Trial of Sodium-GLucose coTransporter 2 INhibitors in Pediatric Chronic KIDney DiSease

    Testing whether a trial of SGLT2 inhibitors in youth with chronic kidney disease is feasible

    📍 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.

    ✓ Study care freeEarly Phase 1Ages 12–25Learn more →
  • Kidney disease

    Study to Evaluate the Safety and Efficacy of the 10 GE Xenokidney in Patients With ESRD

    Testing a GE xenokidney transplant in people with ESRD for safety and effectiveness

    📍 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.

    ✓ Study care freePhase 1Ages 40–70Learn more →
  • Sickle cell disease

    Cannabidiol in Sickle Cell Disease

    Testing twice-daily cannabidiol doses for people with sickle cell disease

    📍 Icahn School of Medicine at Mount Sinai · Manhattan, NY · Get directions →

    Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Parkinson's disease

    Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell Exosomes for Parkinson's Disease

    Testing the safety and effectiveness of nose drops made from donor stem cell exosomes for

    📍 Medical Surgical Associates Center · St John's, · Get directions →

    This trial will study the safety and efficacy of intranasal instillation of cultured allogeneic adult umbilical cord derived mesenchymal stem cell exosomes for the treatment of Parkinson's Disease

    ✓ Study care freePhase 1Learn more →
  • Type 2 diabetesKidney disease

    Subclinical Primary Aldosteronism in Diabetes At-Risk for Kidney Disease

    Testing whether people with type 2 diabetes have early hormone-related kidney risk

    📍 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.

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

    Gene Editing For Sickle Cell Disease

    Testing whether gene editing is safe for people with sickle cell disease

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

    This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.

    ✓ Study care freePhase 1Ages 18–24Learn more →
  • Kidney disease

    Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of OJR520 in Healthy Volunteers and Participants With Chronic Kidney Disease

    Testing the safety and side effects of OJR520 and how the body handles it in people with chronic

    📍 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.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Parkinson's disease

    A Pilot Study of In-lab Dynamic Standing in Parkinson's Disease

    Testing a new adjustable standing desk for people with Parkinson’s

    📍 Domino's Farms · Ann Arbor, MI · Get directions →

    This research is studying the use of a new type of standing desk in a small number of people to learn about the user experience for people with Parkinson's disease. 12 4-hour sessions will be performed to test the primary hypothesis that dynamic standing improves gait function compared to static standing and control sitting.

    ✓ Study care freePhase 1Ages 50+Learn more →
  • Parkinson's disease

    First-in-Human Single and Multiple Dose of HL-400

    Testing an NLRP3 inhibitor taken by mouth for safety in healthy people

    📍 Pharmaron CPC, Inc. · Baltimore, MD · Get directions →

    This is a randomized, double-blind, placebo-controlled, dose-escalation study in healthy subjects to evaluate the safety, tolerability, pharmacokinetics of HL-400 (a NLRP3 inhibitor) following oral single and multiple ascending dose administration.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Parkinson's disease

    Using taVNS to Modulate Cardiovascular Function in Individuals With Neurologic Disease

    Testing gentle ear nerve stimulation to see if it affects heart rate and blood pressure in people

    📍 Wellness, Health and Research Facility at UAB · Birmingham, AL · Get directions →

    The purpose of this study is to find out whether a type of gentle nerve stimulation, called transcutaneous auricular Vagus Nerve Stimulation (taVNS), can help improve how the body regulates heart rate and blood pressure in people with Parkinson's Disease (PD). Problems with heart rate and blood pressure control are common and can make it harder for people to exercise or do daily activities.

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

    Study to Evaluate the Safety and Efficacy of the GGTA1 KO Thymokidney in Patients With ESRD

    Testing a new kidney tissue transplant to treat end-stage kidney disease

    📍 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.

    ✓ Study care freePhase 1Ages 40–70Learn more →
  • Kidney disease

    📍 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).

    ✓ Study care freePhase 1Ages 18–75Learn more →
  • 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.

    ✓ Study care freeEarly Phase 1Ages 18–99Learn more →
  • Kidney disease

    BZLF1 Peptide Vaccine (OSU-2131) With QS-21 for the Prevention of Epstein-Barr Virus Related Cancer in Patients Awaiting Solid Organ Transplants

    Testing a vaccine to prevent Epstein-Barr virus cancer in transplant patients

    📍 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.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Kidney disease

    A Study to Assess the Safety and Pharmacokinetics of INCB123667 When Administered Orally to Adult Participants With Severe Renal Impairment or End Stage Renal Disease

    Testing the safety and how an oral medicine moves through the body in adults with severe kidney

    📍 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.

    ✓ Study care freePhase 1Ages 18–85Learn more →
  • Parkinson's disease

    Enrichment of Glutathione Using Gamma-glutamylcysteine Supplementation in Parkinson's Disease Patients.

    Testing whether taking glutathione-building supplements helps early Parkinson’s patients

    📍 UPMC Presbyterian Hospital · Pittsburgh, PA · Get directions →

    This study is designed l to evaluate the effects of GGC oral supplementation in early Parkinson's disease (PD) patients. The main objectives of the study are to evaluate: 1.

    ✓ Study care freePhase 1Ages 50–80Learn more →
  • Sickle cell disease

    A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

    Testing how BMS-986470 affects the body and safety in sickle cell disease

    📍 University of Alabama at Birmingham · Birmingham, AL · +18 more US sites · Get directions →

    The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.

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

    Breakthrough - T1DM and Chronic Kidney Disease

    Testing an experimental treatment of Tegoprubart plus islet cells for diabetes and chronic kidney

    📍 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).

    ✓ Study care freePhase 1Ages 18–70Learn more →
  • 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.

    ✓ Study care freeEarly Phase 1Ages 18–100Learn more →
  • Sickle cell disease

    Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

    Testing a partial stem cell transplant from matched donors for severe sickle cell disease or

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens.

    ✓ Study care freePhase 1Ages 4–65Learn more →
  • Sickle cell disease

    Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

    Testing how safe escalating doses of fostamatinib are in people with stable sickle cell disease

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Sickle cell disease

    Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

    Testing gene editing in stem cells for severe sickle cell disease

    📍 Boston Children's Hospital · Boston, MA · Get directions →

    A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.

    ✓ Study care freePhase 1Ages 13–40Learn more →
  • Parkinson's disease

    📍 Cornell University Weill Medical College · New York, NY · Get directions →

    This is a phase 1/2 clinical study to evaluate the safety, tolerability, feasibility, and preliminary efficacy of NouvNeu001 in patients with advanced Parkinson's Disease.

    ✓ Study care freePhase 1Ages 30–75Learn more →
  • Sickle cell disease

    SCD Stem Cell Mobilization and Apheresis Using Motixafortide

    Testing whether motixafortide is safe and can raise stem cells in people with sickle cell disease

    📍 University of Alabama at Birmingham · Birmingham, AL · +2 more US sites · Get directions →

    This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Parkinson's disease

    Transplantation of Human iPS Cell-derived Dopaminergic Progenitors (CT1-DAP001) for Parkinson's Disease (Phase I/II)

    Testing whether transplanting lab-made dopamine cells is safe for Parkinson’s

    📍 University of California, San Diego · La Jolla, CA · Get directions →

    To evaluate the safety and efficacy of transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors, CT1-DAP001, into the corpus striatum in patients with Parkinson's disease

    ✓ Study care freePhase 1Ages 40–75Learn more →
  • Parkinson's disease

    Autologous suraL nervE Grafting to the Substantia nigrA in Patients With Synuclienopathies

    Testing whether a nerve tissue graft can be safely placed in the brain in Parkinson’s or MSA

    📍 University of Kentucky · Lexington, KY · Get directions →

    This phase I double-blind study focuses on the safety and feasibility of implanting autologous peripheral nerve tissue (PNT) into the substantia nigra area of the brain in persons who have been diagnosed with either Parkinson's disease (PD) or Multiple System Atrophy (MSA). 7 participants will be enrolled, with 4 participants receiving the graft and 3 receiving a sham surgery.

    ✓ Study care freePhase 1Ages 40–75Learn more →
  • Sickle cell disease

    Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

    Testing the safety and effects of panobinostat in adults with sickle cell disease

    📍 Augusta University · Augusta, GA · Get directions →

    The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor.

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

    ✓ Study care freeEarly Phase 1Ages 40–80Learn more →
  • Parkinson's disease

    Noise-augmented Automatic Speech Recognition for Speech Treatment in Parkinson's Disease

    Testing whether automatic speech recognition feedback improves speech clarity and social

    📍 Teachers College, Columbia University · New York, NY · Get directions →

    The goal of this clinical trial is to learn whether noise-augmented feedback using automatic speech recognition improves intelligibility and social participation in individuals with Parkinson's disease. The main questions the investigators will address are: Does noise-augmented feedback lead to increased intelligibility, as measured by transcription accuracy scores?

    ✓ Study care freeEarly Phase 1Ages 50–80Learn more →
  • Sickle cell disease

    A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

    Testing how a new medicine works in healthy people and sickle cell patients

    📍 University of Alabama Birmingham · Birmingham, AL · +5 more US sites · Get directions →

    The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).

    ✓ Study care freePhase 1Ages 12–55Learn more →
  • Kidney disease

    Sympatholysis in Chronic Kidney Disease

    Studying why people with chronic kidney disease struggle to exercise and see blood pressure rise

    📍 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).

    ✓ Study care freePhase 1Ages 18–75Learn more →
  • Sickle cell disease

    Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

    Testing a bone marrow transplant with lower-intensity treatment for kids with sickle cell and

    📍 Yale School of Medicine · New Haven, CT · +3 more US sites · Get directions →

    This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.

    ✓ Study care freePhase 1Learn more →
  • Parkinson's disease

    A Clinical Trial of LY3962681 in Healthy Volunteers and in Patients With Parkinson's Disease

    Testing how a Parkinson’s drug candidate affects safety and body handling in healthy people and

    📍 Austin Clinic PPD · Austin, TX · Get directions →

    The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics/pharmacodynamics (PK/PD) of LY3962681 in healthy volunteers and patients with Parkinson's disease. The study consists of two parts, the Single Ascending Dose (SAD) study and the Multiple Ascending Dose (MAD) study.

    ✓ Study care freePhase 1Ages 30–80Learn more →
  • Parkinson's disease

    rTMS to Improve Cognition in Parkinson's

    Testing whether noninvasive brain stimulation can improve thinking in people with Parkinson’s

    📍 Jesse Brown VA Medical Center, Chicago, IL · Chicago, IL · +1 more US site · Get directions →

    The purpose of this study is to examine safety, feasibility, and the behavioral and brain effects of a non-invasive treatment, repetitive transcranial magnetic stimulation (rTMS), for Veterans with Parkinson's disease or atypical parkinsonism and mild impairments in their thinking. The hypothesis is that rTMS can improve thinking for people with Parkinson's disease or atypical parkinsonism who are experiencing mild problems with their thinking ability.

    ✓ Study care freePhase 1Ages 50+Learn more →
  • Dementia / Alzheimer'sParkinson's disease

    [18F]F-DOPA Imaging in Patients With Autonomic Failure

    Testing [18F]F-DOPA brain scans in people with autonomic failure

    📍 Vanderbilt University Medical Center · Nashville, TN · Get directions →

    Alpha-synucleinopathies refer to age-related neurodegenerative and dementing disorders, characterized by the accumulation of alpha-synuclein in neurons and/or glia. The anatomical location of alpha-synuclein inclusions (Lewy Bodies) and the pattern of progressive neuronal death (e.g.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Parkinson's disease

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

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

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

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

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

    Study of Orellanine in Metastatic Clear-Cell or Papillary Renal Cell Carcinoma

    Testing orellanine in kidney cancer patients on chronic hemodialysis to see if it is safe and may

    📍 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.

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

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

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

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

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

    ✓ Study care freePhase 1Ages 8–17Learn more →
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