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87 studies
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- 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.
- COPD (chronic lung disease)
Efficacy, Safety, and Tolerability Study of Lunsekimig Compared With Placebo in Adult Participants With Inadequately Controlled Chronic Obstructive Pulmonary Disease (COPD), Characterized by an Eosinophilic Phenotype
Testing a shot versus placebo for COPD symptoms in adults with high eosinophils
📍 University of Alabama at Birmingham- Site Number : 8400003 · Birmingham, AL · +39 more US sites · Get directions →
This is a parallel, Phase 2b/Phase 3, 3-arm study to investigate the efficacy, safety, and tolerability of subcutaneous (SC) treatment with lunsekimig compared with placebo in adult participants (aged 40 to 80 years, inclusive) with inadequately controlled Chronic obstructive pulmonary disease (COPD) characterized by an eosinophilic phenotype. Participation to the study consists of 3 periods: * Screening period of up to 4 weeks * Randomized intervention period of approximately 48 weeks * Follow-up period: Approximately 8 weeks The study duration will be up to 60 weeks.
- 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.
- Fatty liver disease (NASH/MASH)💵 May compensate
A Pivotal Clinical Study to Investigate Efimosfermin Alfa in Participants With Biopsy-confirmed F2- or F3-stage MASH
Testing how efimosfermin alfa compares with placebo for MASH with scarred liver tissue
📍 GSK Investigational Site · Arcadia, CA · +39 more US sites · Get directions →
The purpose of this study is to assess the safety and efficacy of efimosfermin alfa in the resolution of steatohepatitis and improvement of liver-related clinical outcome compared to placebo in individuals with MASH and biopsy-confirmed F2- or F3-stage fibrosis.
- COPD (chronic lung disease)
A Study to Evaluate the Long-Term Safety of Astegolimab in Participants With Chronic Obstructive Pulmonary Disease (COPD)
Testing the long-term safety of astegolimab in people with COPD
📍 UAB Lung Health Center · Birmingham, AL · +39 more US sites · Get directions →
The purpose of this study is to assess the long-term safety and to explore the efficacy of astegolimab in participants with chronic obstructive pulmonary disease (COPD) who have completed the 52-week placebo-controlled treatment period in parent studies GB43311 or GB44332.
- Heart / cardiovascular diseaseKidney disease
A Study of Orforglipron (LY3502970) on Cardiovascular Outcomes in Adults With Atherosclerotic Cardiovascular Disease and/or Chronic Kidney Disease (ATTAIN-Outcomes)
Testing whether orforglipron changes heart outcomes in adults with heart disease and/or kidney
📍 Instituto de Investigaciones Clínicas Bahia Blanca · Bahía Blanca, · Get directions →
The purpose of this study is to measure cardiovascular outcomes with orforglipron compared with placebo in participants with atherosclerotic cardiovascular disease (ASCVD) and/or chronic kidney disease (CKD). Participation in the study will last about 5 years.
- 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.
- COPD (chronic lung disease)
A Study to Investigate the Efficacy and Safety of Tezepelumab in Adult Participants With Moderate to Very Severe COPD (D5241C00006)
Testing whether tezepelumab is safe and works for adults with COPD
📍 Research Site · Foley, AL · +39 more US sites · Get directions →
A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group, Phase 3 Study to Evaluate the Efficacy and Safety of Tezepelumab in Adults with Moderate to Very Severe Chronic Obstructive Pulmonary Disease (COPD)
- 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.
- COPD (chronic lung disease)
Depemokimab as an Extended treatmeNt Duration Biologic in Adults With Chronic Obstructive Pulmonary Disease (COPD) and Type 2 Inflammation (ENDURA-2)
Testing a medicine added to standard care for moderate to severe COPD
📍 GSK Investigational Site · Sheffield, AL · +39 more US sites · Get directions →
Depemokimab is being developed as a treatment for individuals with moderate to severe COPD. The aim of this study is to assess the efficacy and safety of depemokimab compared as an add-on medicine in participants with uncontrolled moderate to severe COPD with type 2 inflammation.
- Sickle cell disease
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Testing rilzabrutinib for safety and effectiveness in sickle-cell disease
📍 University of Alabama at Birmingham- Site Number : 8400003 · Birmingham, AL · +15 more US sites · Get directions →
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B).
- 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.
- Fatty liver disease (NASH/MASH)
A Study Evaluating Efruxifermin in Subjects With Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis
Testing a drug to treat fat buildup liver disease in people with stage 2 or 3 fibrosis
📍 Akero Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →
This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with non-cirrhotic NASH/MASH and fibrosis stage 2 or 3 (F2 or F3). The study will enroll subjects in two cohorts for a total samples size of 1650 subjects.
- Fatty liver disease (NASH/MASH)
A Study Evaluating the Efficacy and Safety of Pegozafermin in Participants With MASH and Fibrosis (ENLIGHTEN-Fibrosis)
Testing whether two doses of pegozafermin are safe and work for liver scarring in adults with MASH
📍 89bio Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →
The study will assess the efficacy and safety of 2 dose regimens of pegozafermin compared to placebo for the treatment of liver fibrosis stage F2 or F3 in adult participants with MASH.
- 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.
- 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.
- Heart / cardiovascular diseaseHigh blood pressure
EASi-PROTKT™ - A Study to Test Vicadrostat (BI 690517) Taken Together With Empagliflozin in People With Type 2 Diabetes, High Blood Pressure, and Cardiovascular Disease
Testing a drug taken with empagliflozin in adults with type 2 diabetes and heart disease
📍 Pinnacle Research Group, LLC · Anniston, AL · +39 more US sites · Get directions →
This study is open to adults with type 2 diabetes, high blood pressure, and cardiovascular disease. People can join the study if they have these conditions and do not have a history of heart failure.
- COPD (chronic lung disease)
Multi-Center Clean Air Randomized Controlled Trial in COPD
Testing whether using an air cleaner improves COPD symptoms and quality of life
📍 University of Alabama, Lung Health Center · Birmingham, AL · +10 more US sites · Get directions →
This is a multi-center randomized, sham-controlled clinical trial to determine the effectiveness of an air cleaner intervention aimed at improving indoor air quality on reducing COPD exacerbation risk and improving quality of life, functional status, rescue medication use.
- Fatty liver disease (NASH/MASH)💵 May compensate
A Study to Evaluate the Efficacy and Safety of Pegozafermin in Participants With Compensated Cirrhosis Due to MASH
Testing whether pegozafermin is safe and works for MASH-related liver cirrhosis
📍 89bio Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →
The study will assess the efficacy and safety of pegozafermin administered in participants with compensated cirrhosis due to MASH (biopsy-confirmed fibrosis stage F4 MASH \[previously known as nonalcoholic steatohepatitis, NASH\]).
- 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).
- COPD (chronic lung disease)
Efficacy, Safety, and Tolerability Study of Lunsekimig Compared With Placebo in Adult Participants With Inadequately Controlled Chronic Obstructive Pulmonary Disease (COPD) Characterized by an Eosinophilic Phenotype
Testing a skin injection to treat long-term COPD in adults with high eosinophils
📍 Chandler Clinical Research Trials- Site Number : 8400025 · Chandler, AZ · +39 more US sites · Get directions →
This is a parallel, Phase 2b/Phase 3, 3-arm study to investigate the efficacy, safety, and tolerability of subcutaneous (SC) treatment with lunsekimig compared with placebo in adult participants (aged 40 to 80 years, inclusive) with inadequately controlled Chronic obstructive pulmonary disease (COPD) characterized by an eosinophilic phenotype. Participation to the study consists of 3 periods: * Screening period of up to 4 weeks * Randomized intervention period of approximately 48 weeks * Follow-up period: Approximately 8 weeks The study duration will be up to 60 weeks.
- Heart / cardiovascular diseaseKidney disease💵 May compensate
Sotagliflozin to Slow Kidney Function Decline in Persons With Type 1 Diabetes and Diabetic Kidney Disease
Testing whether sotagliflozin can slow kidney damage in type 1 diabetes
📍 Stanford University Medical Center · Stanford, CA · +12 more US sites · Get directions →
Powerful new drugs that can prevent or delay end stage kidney disease (ESKD) - so called sodium-glucose cotransporter-2 inhibitors (SGLT2i) - are now available for patients with type 2 diabetes. Whether these drugs have similar effects in patients with type 1 diabetes (T1D) remains unknown because of the few studies in this population, due to concerns about the increase in risk of diabetic ketoacidosis (DKA, a serious, potentially fatal acute complication of diabetes due to the accumulation of substances called ketone bodies) observed with SGLT2i therapy in T1D.
- Sickle cell disease
Cannabidiol in Sickle Cell Disease
Testing twice-daily cannabidiol doses for people with sickle cell disease
📍 Icahn School of Medicine at Mount Sinai · Manhattan, NY · Get directions →
Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.
- COPD (chronic lung disease)
A Study of BBT002 in Healthy Volunteers (HVs) and in Adult Patients With Chronic Obstructive Pulmonary Disease (COPD) or Chronic Rhiosininusitis With Nasal Polyps (CRSwNP)
Testing different doses of BBT002 in people with COPD and in healthy adults
📍 Equity Medical Bowling Green · Bowling Green, KY · +2 more US sites · Get directions →
Randomized study of single and multiple doses of BBT002 in healthy volunteers and in adult patients with chronic obstructive pulmonary disease (COPD) or chronic rhinosinusitis with nasal polyps (CRSwNP).
- COPD (chronic lung disease)
Depemokimab as an Extended treatmeNt Duration Biologic in Adults With Chronic Obstructive Pulmonary Disease (COPD) and Type 2 Inflammation (ENDURA -1)
Testing an add-on medicine to treat moderate to severe COPD with type 2 inflammation
📍 GSK Investigational Site · Loxahatchee Groves, FL · +3 more US sites · Get directions →
Depemokimab is being developed as a treatment for individuals with moderate to severe COPD. The aim of this study is to assess the efficacy and safety of depemokimab as an add-on medicine in participants with uncontrolled moderate to severe COPD with type 2 inflammation.
- Sickle cell disease
Gene Editing For Sickle Cell Disease
Testing whether gene editing is safe for people with sickle cell disease
📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.
- Sickle cell disease
Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease
Testing a single pneumococcal vaccine dose in children with sickle cell disease
📍 Site # 8400006 · Atlanta, GA · +4 more US sites · Get directions →
The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV (licensed pneumococcal vaccine) when given as a single dose to children aged 2 to 17 years with sickle cell disease who had received or not a previous vaccination with pneumococcal conjugate or pneumococcal polysaccharide vaccine.
- COPD (chronic lung disease)
A Study Evaluating the Efficacy of Budesonide, Glycopyrronium and Formoterol Fumarate Metered Dosed Inhaler on Cardiopulmonary Outcomes in Chronic Obstructive Pulmonary Disease
Comparing inhalers to see how triple breathing medicine affects heart and lung outcomes in COPD
📍 Research Site · Athens, AL · +39 more US sites · Get directions →
This study will evaluate the effect of triple ICS/LAMA/LABA therapy with BGF MDI 320/14.4/9.6 μg on cardiopulmonary outcomes relative to LAMA/LABA therapy with GFF MDI 14.4/9.6 μg in a population with COPD and elevated cardiopulmonary risk.
- Obesity / overweightHeart / cardiovascular disease💵 May compensate
Prevention of Progression of Prediabetes, Obesity and CV Risk
Testing ways to stop prediabetes and obesity from getting worse
📍 University of Texas Health Science Center at San Antonio · San Antonio, TX · Get directions →
The investigators are studying how to help people with prediabetes (Pre-DM) and obesity. The goal is to use new and affordable treatments to bring blood sugar levels back to normal and help participants to lose weight.
- 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.
- Obesity / overweightHeart / cardiovascular disease
Evaluating the Impact of Maridebart Cafraglutide on Cardiovascular Outcomes in Participants With Atherosclerotic Cardiovascular Disease and Overweight or Obesity
Testing whether maridebart cafraglutide lowers heart risks in people with heart disease and obesity
📍 Alliance For Multispecialty Research - Daphne · Daphne, AL · +39 more US sites · Get directions →
The primary objective of this trial is to demonstrate that maridebart cafraglutide is superior to placebo when given as an adjunct to standard of care with respect to reducing cardiovascular (CV) morbidity and mortality.
- Obesity / overweightHeart / cardiovascular disease💵 May compensate
Maridebart Cafraglutide in Heart Failure With Preserved or Mildly Reduced Ejection Fraction and Obesity
Testing maridebart cafraglutide with standard care to reduce heart failure events in obese people
📍 University of Alabama Saint Vincent's Birmingham · Birmingham, AL · +39 more US sites · Get directions →
This trial will examine if maridebart cafraglutide as an adjunct to standard of care will lead to a reduction in heart failure (HF) events such as HF hospitalizations and urgent HF visits, cardiovascular (CV) deaths and improvement in HF symptoms in participants with HF with preserved ejection fraction (HFpEF) and HF with mildly reduced ejection fraction (HFmrEF) who are obese. This is a phase 3, global, multicenter, 2-part trial with a double-blind period and an open-label extension (OLE).
- Obesity / overweightHeart / cardiovascular disease
A Research Study to Look at How Well NNC0487-0111 Works Compared to Placebo in People With Heart Failure and Obesity
Testing how well NNC0487-0111 works versus placebo in heart failure with excess body weight
📍 Advanced Cardiovascular, LLC · Alexander City, AL · +39 more US sites · Get directions →
This study is being done to look at the safety and effect of NNC0487-0111 in people with Heart Failure with preserved Ejection Fraction (HFpEF) or Heart Failure with mildly reduced Ejection Fraction (HFmrEF) and excess body weight when compared to placebo. The purpose of this clinical study is to find out if NNC0487-0111 is safe and effective for treating people who have HFpEF or HFmrEF and excess body weight.
- COPD (chronic lung disease)
A Study to Learn About the Study Medicine Called PF-07275315 in People With Moderate to Severe Chronic Obstructive Pulmonary Disease
Testing how a study medicine affects safety and symptoms in people with COPD
📍 University of Alabama at Birmingham Lung Health Center · Birmingham, AL · +39 more US sites · Get directions →
The purpose of this clinical trial is to learn about the effects and safety of the study medicine PF-07275315 for the potential treatment of COPD. COPD is a condition that makes it difficult to breathe, which negatively impacts the quality of life and functioning of people who are affected.
- COPD (chronic lung disease)
A Study to Test the Safety and Effects of Inhaled GDC-6988 in Participants With Muco-obstructive Disease
Testing the safety and effects of an inhaled medicine for COPD
📍 University of Alabama at Birmingham · Birmingham, AL · +6 more US sites · Get directions →
This study evaluates the safety, tolerability, and activity of inhaled GDC-6988 in participants with muco-obstructive disease.
- Obesity / overweightHigh blood pressureCOPD (chronic lung disease)
Feasibility of Semaglutide in Advanced Lung Disease
Testing whether semaglutide can be given safely to people with advanced lung disease
📍 University of Pennsylvania Perelman School of Medicine · Philadelphia, PA · Get directions →
The goal of this clinical trial is to learn whether semaglutide, an FDA-approved treatment for diabetes and obesity, is feasible and tolerable in patients with advanced lung disease. The main question\[s\] it aims to answer are: 1.
- Sickle cell disease
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Testing long-term treatment with a new medicine for sickle cell or thalassaemia
📍 Univ of Alabama Birmingham · Birmingham, AL · +38 more US sites · Get directions →
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- 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
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.
- Heart / cardiovascular disease
COLchicine and Non-enteric Coated Aspirin in the Cardiovascular Outcomes Trial of Patients With Type 2 Diabetes
Testing colchicine and plain aspirin, alone or together, for heart outcomes in high-risk type 2
📍 Curtin University · Bentley, Western Australia · Get directions →
To evaluate the efficacy and safety of colchicine and non-enteric coated aspirin, combined or alone, to improve cardiovascular outcomes in high-risk patients with type 2 diabetes.
- 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.
- Sickle cell disease
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
Testing a drug infusion versus placebo to treat frequent pain crises in sickle cell disease
📍 University Of Alabama · Birmingham, AL · +13 more US sites · Get directions →
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.
- Type 2 diabetesHeart / cardiovascular disease
FES Cycling With BFR in the Spinal Cord Injury Population
Testing a cycling exercise program with added arm-leg pressure for people with spinal cord injury
📍 Lyndhurst Centre · Toronto, Ontario · Get directions →
Cardiometabolic Disease (CMD) is the silent killer within the spinal cord injury/disease (SCI/D) population. Many SCI/D patients' initial CMD presentation is with a heart attack, stroke, or sudden cardiac death, highlighting the need for interventions to reduce CMD risk.
- COPD (chronic lung disease)
eValuating the Efficacy and Safety of InitiatinG depemokImab earLy therApy iN Chronic Obstructive Pulmonary Disorder (COPD) With Type 2 Inflammation
Testing an add-on medicine started early to treat COPD with type 2 inflammation
📍 GSK Investigational Site · Doral, FL · Get directions →
Depemokimab is being developed as a treatment for individuals with moderate to severe Chronic Obstructive Pulmonary Disorder (COPD). The aim of this study is to assess the efficacy and safety of early initiation of depemokimab as an add-on medicine in participants with moderate to severe COPD with type 2 inflammation.
- Fatty liver disease (NASH/MASH)💵 May compensate
A Study Evaluating Efruxifermin in Subjects With Compensated Cirrhosis Due to NASH/MASH
Testing a medicine called efruxifermin in people with compensated liver scarring from NASH/MASH
📍 Akero Clinical Study Site · Birmingham, AL · +39 more US sites · Get directions →
This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with compensated cirrhosis due to NASH/MASH.
- 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.
- Obesity / overweightHeart / cardiovascular disease
Mechanisms of Semaglutide Therapy in Heart Failure Patients
Testing how semaglutide works in people with heart failure and obesity
📍 Greenstone Biosciences · Palo Alto, CA · +1 more US site · Get directions →
Semaglutide is a glucagon-like peptide-1 (GLP-1) receptor agonist, primarily used for treatment of type-2 diabetes mellitus. GLP-1 receptors are present on pancreatic islet β-cells, δ-cells and α-cells.
- 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.
- Sickle cell disease
A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
Testing how well etavopivat lowers painful sickle cell crises and organ damage
📍 Uni of Alabama at Birmingham · Birmingham, AL · +39 more US sites · Get directions →
This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo.
- Fatty liver disease (NASH/MASH)💵 May compensate
LIVERAGE™: A Study to Test Whether Survodutide Helps People With a Liver Disease Called NASH/MASH Who Have Moderate or Advanced Liver Fibrosis
Testing whether survodutide helps adults with MASH and moderate to advanced liver scarring
📍 University of Alabama at Birmingham · Birmingham, AL · +39 more US sites · Get directions →
This study is open to adults who are at least 18 years old living with obesity and have: * a confirmed liver disease called non-alcoholic steatohepatitis (NASH)/metabolic associated steatohepatitis (MASH) and * moderate or advanced liver fibrosis People with a history of acute or chronic liver diseases other than MASH or chronic alcohol intake cannot take part in this study. The purpose of this study is to find out whether a medicine called survodutide helps people with MASH and moderate or advanced liver fibrosis improve their liver function.
- Fatty liver disease (NASH/MASH)💵 May compensate
LIVERAGE™ - Cirrhosis: A Study to Test Whether Survodutide Helps People With a Liver Disease Called NASH/MASH Who Have Cirrhosis
Testing whether survodutide helps adults with NASH/MASH cirrhosis
📍 The Institute for Liver Health II DBA Arizona Clinical Trials · Peoria, AZ · +39 more US sites · Get directions →
This study is open to adults who are at least 18 years old and have: * A confirmed liver disease called non-alcoholic steatohepatitis (NASH) or * A confirmed liver disease called metabolic-associated steatohepatitis (MASH) * BMI of 27 kg/m2 or more or * 25 kg/m2 or more if the participant is Asian. People with a history of other chronic liver diseases or high alcohol intake cannot take part in this study.
- Fatty liver disease (NASH/MASH)💵 May compensate
A Clinical Study to Investigate the Safety and Tolerability of Efimosfermin Alfa Injection in Participants With Known or Suspected F2- or F3-stage MASH
Testing the safety of an injected medicine in people with MASH and F2 or F3 scarring
📍 GSK Investigational Site · Arcadia, CA · +39 more US sites · Get directions →
This study will evaluate the safety and tolerability of Efimosfermin Alfa for participants with known or suspected MASH with fibrosis consistent with stage F2 or F3.
- 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.
- Heart / cardiovascular disease💵 May compensate
Canagliflozin Targeting Vascular Inflammation
Testing canagliflozin to reduce blood vessel inflammation in cardiovascular disease
📍 University of Ottawa Heart Institute · Ottawa, Ontario · Get directions →
CANTOR SING is a pilot single center double blinded randomized study. The investigators will compare the effect of canagliflozin (300 mg daily - intervention arm) vs.
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