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78 studies
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- 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).
- Eczema / atopic dermatitis
A Study to Learn About Study Medicine Called PF-08049820 in People With Eczema
Testing whether an experimental medicine is safe and works for eczema
📍 Northridge Clinical Trials · Northridge, CA · +3 more US sites · Get directions →
The purpose of this study is to learn if the study medicine (PF-08049820) is safe and effective for the treatment of atopic dermatitis (AD), also known as eczema, or atopic eczema. People with this condition may have severe itching and rashes on the skin.
- High blood pressureKidney disease
A Phase IIb Study to Evaluate the Effect of Dapagliflozin in Combination With Baxdrostat Compared With Baxdrostat on Albuminuria in Participants With Chronic Kidney Disease and High Blood Pressure.
Testing how adding a diabetes medicine to baxdrostat affects urine protein in people with chronic
📍 Research Site · Surprise, AZ · +17 more US sites · Get directions →
International, Multicenter and Double-Blind study. The purpose is to measure the effect of baxdrostat in combination with dapagliflozin compared with baxdrostat/placebo on albuminuria, as well as safety, in participants with chronic kidney disease and high blood pressure.
- Fatty liver disease (NASH/MASH)
A Study of a Thyroid Hormone Receptor Beta Isoform (THRβ) Agonist and an Semicarbazide Sensitive Amine Oxidase (SSAO) Inhibitor, Alone and in Combination, in Adults With Presumed Metabolic Dysfunction-associated Steatohepatitis (MASH)
Testing drugs together or alone to lower liver fat in adults with MASH
📍 Arizona Liver Health · Chandler, AZ · +39 more US sites · Get directions →
The primary objective of this trial is to evaluate the dose-dependent and comparative effects of ECC4703 (low and high dose), ECC0509 (low and high dose), and their combination on hepatic fat reduction as assessed by change in magnetic resonance imaging proton density fat fraction (MRI-PDFF) at Week 12.
- Eczema / atopic dermatitis
Cardamom and Topical Roseomonas in Atopic Dermatitis
Testing cardamom and a skin-applied germ to treat eczema
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Atopic dermatitis (AD), also called eczema, is a chronic skin condition. AD can make skin dry and itchy, and sometimes it can lead to serious health problems, such as asthma, food allergies, eye infections, and sleep problems.
- Type 2 diabetesHigh blood pressure
Polypill for Prevention of Cardiomyopathy
Testing a pill combination to prevent heart muscle disease in people with type 2 diabetes and high
📍 UT Southwestern Medical Center · Dallas, TX · Get directions →
This study will investigate the utility of a polypill-based strategy for patients with type 2 diabetes mellitus and high risk of heart failure (HF), as assessed via the WATCH-DM risk score. Polypill therapy will consist of empagliflozin 12.5 mg, losartan 25, 50 or 100 mg, and finerenone 10 mg daily.
- 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.
- Sickle cell disease
Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults
Testing whether a low dose of ketamine helps treat sickle cell pain in children and young adults
📍 Harbor UCLA Medical Center · Torrance, CA · Get directions →
The purpose of this research is to see if ketamine is effective and safe in treating children and young adults with sickle cell disease experiencing sickle cell related pain. In this study, we will compare the outcomes (such as pain scores) in persons who receive standard of care pain medicine (an opioid such as morphine) plus a low dose (amount) of ketamine to those who receive only standard of care pain medicine.
- Eczema / atopic dermatitis
A Long-term Safety and Efficacy Study Evaluating APG777 in Atopic Dermatitis
Testing the long-term safety and effects of a drug in people with eczema
📍 Investigational Site · Fountain Valley, CA · +21 more US sites · Get directions →
This is a multicenter, double-blind, Long-Term Extension (LTE) study to evaluate the long-term safety and efficacy of APG777 in patients with moderate-to-severe AD who have completed treatment in an APG777 Parent Study (NCT06395948). The LTE study will consist of 3 periods: 1) Screening Visit will coincide with the last visit of the Maintenance Period in the Parent Study 2) Extended Treatment Period 3) Post-treatment Follow-up Period.
- Fatty liver disease (NASH/MASH)
Impact of Soymilk on Liver Disease Severity of Children With Non-alcoholic Fatty Liver Disease (NAFLD)
Testing whether daily soy milk affects liver disease in children with fatty liver
📍 Cincinnati Children's Hospital Medical Center · Cincinnati, OH · Get directions →
A randomized, controlled study of standard soy milk consumption compared to 2% fat cow's milk consumption in children with Non-alcoholic Fatty Liver Disease (NAFLD). The investigators hypothesize that the daily consumption of soy isoflavones found in the soy milk will be beneficial in reducing NAFLD and other obesity-related comorbidities.
- 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.
- Eczema / atopic dermatitis
A Study Evaluating BFB759 in Moderate to Severe Atopic Dermatitis
Testing a medicine for moderate to severe eczema, compared with a placebo
📍 Mountain West Derm - Blackhart PLLC dba Skin Cancer and Dermatology Institute · Phoenix, AZ · +39 more US sites · Get directions →
This is a double-blind, placebo-controlled study where subjects are participating for approximately 36 to 40 weeks. The study compares how well BFB759 works and how safe it is compared with a placebo.
- Sickle cell disease
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Testing a once-daily mouth medicine in teens with sickle cell disease
📍 The Hospital for Sick Children · Toronto, Ontario · Get directions →
This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat.
- Fatty liver disease (NASH/MASH)💵 May compensate
Non-Alcoholic Fatty Liver Disease, the HEpatic Response to Oral Glucose, and the Effect of Semaglutide (NAFLD HEROES)
Testing how the liver responds to glucose in fatty liver disease, and semaglutide’s effect
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: In non-alcoholic fatty liver disease (NAFLD), fat accumulates in the liver and can cause damage. Researchers want to learn what causes the damage NAFLD, and to see if a medication can help.
- Fatty liver disease (NASH/MASH)
A Trial to Investigate Safety, Exposure, and Efficacy of HU6 Compared With Placebo in Adult Participants With Metabolic Dysfunction-associated Steatohepatitis (MASH)
Testing how safe and effective HU6 is for adults with MASH
📍 Arizona Liver Health - Chandler · Chandler, AZ · +30 more US sites · Get directions →
Rivus Pharmaceuticals. Inc. is sponsoring this research study to assess the safety and tolerability of HU6 as a possible treatment for patients diagnosed with metabolic dysfunction-associated steatohepatitis (MASH). The study will also asse
- Eczema / atopic dermatitis
A Study to Evaluate the Efficacy and Safety of IMG-007 in Adult Participants With Moderate-to-Severe Atopic Dermatitis
Testing different doses of a medicine versus placebo for eczema
📍 Inmagene Site 1 · Lancaster, CA · +14 more US sites · Get directions →
The purpose of this study is to evaluate the efficacy and safety of different dose regimens of IMG-007, compared to placebo.
- Fatty liver disease (NASH/MASH)💵 May compensate
Mechanisms of SGLT2 Inhibition in Pediatric Steatotic Liver Disease
Testing how SGLT2 inhibitors work for fatty liver disease in obese teens
📍 Ann & Robert H Lurie Children's Hospital of Chicago · Chicago, IL · Get directions →
This study is a randomized, double-blind, placebo-controlled trial specifically designed to evaluate the preliminary feasibility, initial efficacy and safety of SGLT2 inhibitors for treating NAFLD in adolescents with obesity.
- Eczema / atopic dermatitis
Study to Evaluate Soquelitinib in Participants With Moderate to Severe AD
Testing how different doses of soquelitinib work for moderate to severe atopic dermatitis
📍 Corvus Clinical Trials · South San Francisco, CA · Get directions →
The main purpose of this study is to see how well different doses of soquelitinib work in participants with atopic dermatitis (AD) as compared to a placebo (pill with no medicine in it). To check this, the study doctors will examine participants' skin regularly to track how their AD changes during the study.
- 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.
- High blood pressureCOPD (chronic lung disease)
Breathe Easier With Tadalafil Therapy for Dyspnea in COPD-PH
Testing tadalafil to see if it improves shortness of breath in COPD with high blood pressure in the
📍 Rocky Mountain Regional VA Medical Center, Aurora, CO · Aurora, CO · +4 more US sites · Get directions →
The investigators will study whether the drug tadalafil improves shortness of breath in 126 Veterans with Chronic Obstructive Pulmonary Disease (COPD) and high blood pressure in the lungs. The investigators will also assess whether tadalafil improves quality of life, home daily physical activity, exercise endurance, the frequency of acute flares of COPD, blood pressure in the lungs, and lung function.
- Eczema / atopic dermatitis
Treatment of Atopic Dermatitis and Alopecia Areata With Abrocitinib in Individuals With Down Syndrome
Testing a daily medicine for eczema and hair loss in people with Down syndrome
📍 Icahn School of Medicine at Mount Sinai · New York, NY · Get directions →
This is a single-center, open-label, basket phase 2b trial that will enroll Down Syndrome (DS) participants with at least one inflammatory skin condition (Atopic Dermatitis (AD) and/or Alopecia Areata (AA)). Patients will receive Abrocitinib 100 mg daily for 12 weeks.
- Eczema / atopic dermatitis
A Long Term Extension Study to Evaluate the Safety and Efficacy of Afimkibart (RO7790121) in Participants With Atopic Dermatitis
Checking the long-term safety and effects of an eczema medicine in people who previously studied it
📍 Hamilton Research, LLC · Alpharetta, GA · +2 more US sites · Get directions →
This study will assess long-term safety and efficacy of Afimkibart (also known as RO7790121) in participants with Atopic Dermatitis (AD) who participated in previous afimkibart clinical trials.
- 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).
- Fatty liver disease (NASH/MASH)💵 May compensate
A Study of Efimosfermin Alfa in Participants With Biopsy-confirmed Cirrhosis (Compensated) Due to MASH
Testing efimosfermin in people with MASH-related compensated cirrhosis
📍 GSK Investigational Site · Chandler, AZ · +29 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, preliminary efficacy, and pharmacokinetics (PK) of efimosfermin in participants with metabolic dysfunction associated steatohepatitis (MASH) and compensated cirrhosis consistent with stage F4 fibrosis.
- Sickle cell disease
Cannabidiol in Sickle Cell Disease
Testing twice-daily cannabidiol doses for people with sickle cell disease
📍 Icahn School of Medicine at Mount Sinai · Manhattan, NY · Get directions →
Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.
- Sickle cell disease
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
Testing early heart screening and iron overload treatment in people with sickle cell disease
📍 Inova Schar Cancer · Fairfax, VA · +1 more US site · Get directions →
This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.
- Sickle cell disease
Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial
Testing two daily doses of zinc in people with sickle cell disease to see changes in bone-related
📍 UCSF Benioff Children's Hospital Oakland · Oakland, CA · +5 more US sites · Get directions →
The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover?
- Sickle cell disease
Allo HSCT for High Risk Hemoglobinopathies
Testing donor stem cell transplant for people with high-risk sickle cell
📍 Masonic Cancer Center · Minneapolis, MN · Get directions →
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
- High blood pressureDementia / Alzheimer's
Impact of Intensive Treatment of SBP on Brain Perfusion, Amyloid, and Tau (IPAT Study)
Testing whether tightly lowering high blood pressure slows brain changes tied to Alzheimer’s
📍 University of Texas Southwestern Medical Center · Dallas, TX · Get directions →
The purpose of this study is to determine if intensive lowering of systolic blood pressure (SBP), using FDA approved medications (antihypertensive), reduces Alzheimer's Disease pathology (i.e., excessive brain amyloid and tau protein deposition) in older adults at high risk for memory decline or dementia.
- Fatty liver disease (NASH/MASH)💵 May compensate
Phase 2a Study of Safety, Tolerability, and Efficacy of TLC-2716 in Subjects With Hypertriglyceridemia and NAFLD
Testing a medicine at two doses for safety and effects on fats in people with high triglycerides and
📍 OrsoBio Research Site · Guadalajara, Jalisco · Get directions →
This is a Phase 2a, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, and efficacy of 2 dose levels of TLC-2716 in subjects with hypertriglyceridemia and nonalcoholic fatty liver disease as assessed by changes in fasting triglycerides, liver steatosis by MRI, and other biomarkers.
- Sickle cell disease
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Testing immune-suppressing treatment before stem cell transplant for people with sickle cell disease
📍 Memorial Sloan Kettering at Basking Ridge (Consent only) · Basking Ridge, NJ · +5 more US sites · Get directions →
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD.
- 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 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
Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin
Testing empagliflozin to treat kidney disease related to sickle cell
📍 University of Illinois Chicago, Sickle Cell Center · Chicago, IL · Get directions →
Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA.
- Eczema / atopic dermatitis
A Phase 2b Study of the Effects of Camoteskimab in Adults With Moderate-to-Severe Atopic Dermatitis
Testing how camoteskimab works in adults with moderate to severe eczema
📍 AllerVie Clinical Research · Birmingham, AL · +32 more US sites · Get directions →
This is a phase 2b, multicenter, randomized, double-blind, placebo-controlled study.
- Eczema / atopic dermatitis
A Study to Assess Molecular Changes in Adult Participants With Moderate to Severe Hidradenitis Suppurativa or With Moderate to Severe Atopic Dermatitis Receiving Subcutaneous Injections of Lutikizumab
Testing how a skin injection changes molecules in adults with eczema
📍 Physioseq, LLC /ID# 267266 · Sacramento, CA · +1 more US site · Get directions →
Hidradenitis suppurativa (HS) and atopic dermatitis (AD) are chronic inflammatory skin diseases that lead to the development of skin lesions and symptoms such as pain and discomfort. The purpose of this study is to assess molecular changes in adult participants with moderate to severe HS or with moderate to severe AD.
- Sickle cell disease
Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody
Testing a transplant plan with daratumumab to reduce blood cell failure in sickle cell disease
📍 Children's National Hospital · Washington D.C., DC · Get directions →
This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies.
- Eczema / atopic dermatitis
Evaluate Efficacy and Safety of Repeat Subcutaneous Doses of FB825 in Adults With Moderate-to-Severe Atopic Dermatitis
Testing repeat under-the-skin doses of FB825 for adult eczema
📍 First OC Dermatology · Fountain Valley, CA · +5 more US sites · Get directions →
This is a randomized, placebo-controlled and double-blind study to evaluate the efficacy, pharmacokinetics, and Safety of repeat subcutaneous doses of FB825 in adults with moderate-to-severe atopic dermatitis.
- Eczema / atopic dermatitis
A Double-Blind, Randomized, Vehicle-Controlled Phase 2 Study to Assess the Efficacy and Safety of GX-03 in Adult Subjects With Moderate to Severe Atopic Dermatitis (Eczema)
Testing a topical ointment to see if it helps adult eczema and is safe
📍 ALS Global · Irving, TX · Get directions →
This is a Phase 2, randomized, double-blind, vehicle-controlled clinical study evaluating the efficacy and safety of GX-03 topical ointment in adult subjects with moderate to severe atopic dermatitis (eczema). The study plans to enroll up to 120 eligible patients with a target of at least 100 completing the study and an ability to expand enrollment up to 200 subjects based on a pre-specified interim assessment conducted by an Independent Data Monitoring Committee (IDMC).
- Fatty liver disease (NASH/MASH)💵 May compensate
Milk Thistle Clinical Trial in Pediatric NAFLD
Testing milk thistle for children with fatty liver disease
📍 University Hospitals Cleveland Medical Center · Cleveland, OH · Get directions →
Pediatric Fatty Liver disease is a growing problem in the United States and is expected to be the leading cause of Liver Transplantation in Adults in 20 years. Following lifestyle changes such as diet restrictions and exercise may be difficult to consistently maintain.
- Fatty liver disease (NASH/MASH)
Digoxin In NASH (CODIN)
Testing digoxin for people with metabolic fatty liver inflammation
📍 Yale New Haven Health · New Haven, CT · +1 more US site · Get directions →
Nonalcoholic steatohepatitis (NASH) is a severe subtype of nonalcoholic fatty liver disease (NAFLD) which affects 1 in 3 Americans. The mainstay of treatment for NASH, which was recently renamed metabolic associated steatohepatitis (MASH), involves lifestyle interventions to promote weight loss and to treat comorbidities such as hypertension, hyperlipidemia, and diabetes mellitus.
- High blood pressureKidney disease
Equol and Vascular Function in Women With Chronic Kidney Disease
Testing whether equol affects blood vessel function in women with chronic kidney disease
📍 University of Colorado Anschutz Medical Campus · Aurora, CO · Get directions →
The risk of cardiovascular disease (CVD) is significantly elevated in patients with chronic kidney disease (CKD). Notably, women with CKD commonly experience menstrual disturbances induced by CKD, which may contribute to impaired vascular function and elevated CVD risk.
- 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
Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
Testing a stem cell transplant plan for children with sickle cell disease using a mismatched blood
📍 Alberta Children's Hospital · Calgary, Alberta · Get directions →
The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression.
- Sickle cell disease
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Testing stem cell transplant with specific immune cell removal for sickle cell
📍 New York Medical College · Valhalla, NY · Get directions →
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion. All other treatment is standard of care.
- Sickle cell disease
Intravenous L-Citrulline for Vaso-occlusive Pain Episode in Sickle Cell Disease
Testing whether an IV dose of citrulline can treat sudden pain in hospitalized sickle cell patients
📍 Children's National Hospital · Washington D.C., DC · Get directions →
The goal of this clinical trial is to learn if intravenous citrulline works to treat acute pain in hospitalized patients with sickle cell disease. It will also learn about the safety of intravenous citrulline.
- Sickle cell disease
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
Testing the safety of a medicine (given under the skin) in adults with sickle cell disease
📍 UAMS Medical Center · Little Rock, AR · +4 more US sites · Get directions →
This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. The primary aim of the study is to assess the safety of anumigilimab in participants with SCD.
- Sickle cell disease
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
Testing a vein drug (hemopexin) to treat pain crises from sickle cell
📍 Univ. of California, San Francisco Health Care · Oakland, CA · +15 more US sites · Get directions →
This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.
- Sickle cell diseaseDementia / Alzheimer's
Biomarker-based Trial of NPC-1 for Alzheimer's Pathology
Testing an early treatment to change Alzheimer’s blood markers in adults with memory decline
📍 Massachusetts General Hospital · Boston, MA · Get directions →
This early phase, open label, single arm clinical trial will determine the intraindividual safety, tolerability and effects of NPC1 (parthenolide and ipriflavone) on blood-based biomarkers of Alzheimer's disease (AD) pathology among adults with subjective cognitive decline, mild cognitive impairment, or Alzheimer's disease and objective indicators of seeding AD pathology
- Sickle cell disease
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Testing if adding ruxolitinib lowers graft failure after transplant in children with sickle cell
📍 Children's Hospital of Colorado · Aurora, CO · +3 more US sites · Get directions →
This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants.
- Eczema / atopic dermatitis
A Dose-ranging Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Galvokimig in Adult Study Participants With Atopic Dermatitis
Testing different doses of galvokimig for adults with eczema
📍 Atd002 52021 · Oceanside, CA · +19 more US sites · Get directions →
The purpose of the study is to evaluate the dose-response relationship of galvokimig compared with placebo in study participants with moderate-to-severe atopic dermatitis (AtD).
- Eczema / atopic dermatitis
Study of ENV-294 in Adults With Moderate-to-Severe Atopic Dermatitis
Testing a daily medicine for eczema severity in adults over 12 weeks
📍 Enveda Investigative Site · Birmingham, AL · +22 more US sites · Get directions →
The goal of this clinical trial is to learn about the safety and effectiveness of ENV-294 in adults with moderate to severe atopic dermatitis. The main questions it will answer are: * Is there an impact on the severity and area of atopic dermatitis when participants take ENV-294 * What medical problems do participants have when taking ENV-294 Participants will: * Take drug ENV-294 or a placebo once every day for 12 weeks * Visit the clinic every 2 to 4 weeks for checkups and tests * Keep a diary of their symptoms and when they took their study drug * Return to the clinic for the final study visit at approximately week 16
- Fatty liver disease (NASH/MASH)💵 May compensate
Study to Evaluate Resmetirom in Post-Liver Transplant Patients With MASH
Testing resmetirom in people with MASH-related liver scarring after liver transplant
📍 University of California San Diego · La Jolla, CA · +15 more US sites · Get directions →
A Phase 2 double-blind, randomized, placebo-controlled study to evaluate resmetirom in 2 cohorts of subjects with moderate to advanced fibrosis, consistent with stage F2 and F3 fibrosis, who have undergone liver transplant. Cohort 1 will consist of patients who have undergone liver transplant for MASH cirrhosis who developed recurrent MASH.
- Eczema / atopic dermatitis
A Study To Learn About Two Study Medicines (PF-07275315 And PF-07264660) In People Who Have Moderate To Severe Atopic Dermatitis
Testing two medicines for safety and effects in people with eczema
📍 Allervie Clinical Research · Birmingham, AL · +39 more US sites · Get directions →
The purpose of this study is to learn about the safety and effects of 2 study medicines (PF-07275315 and PF-07264660) for the treatment of atopic dermatitis (AD). AD is a long- lasting itchy red rash, caused by a skin reaction.
- Sickle cell disease
Alendronate for Osteonecrosis in Adults With Sickle Cell Disease
Testing oral alendronate in adults with sickle cell disease and bone damage
📍 UC Davis Comprehensive Cancer Center · Sacramento, CA · Get directions →
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
- Eczema / atopic dermatitis
Effects of Treatments on Atopic Dermatitis
Testing treatments for eczema (atopic dermatitis)
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: \- Atopic dermatitis, or eczema, is a chronic skin disorder. Patients sometimes have infections with S.
- Sickle cell disease
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
Testing defibrotide to prevent liver blood vessel problems in sickle cell patients getting a stem
📍 University of California Los Angeles · Los Angeles, CA · +3 more US sites · Get directions →
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS.
- Sickle cell disease
Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
Testing a stem cell transplant plan to treat aggressive sickle cell disease
📍 University of Illinois at Chicago · Chicago, IL · Get directions →
The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v.
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