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50 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- Sickle cell disease
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Testing the safety and effects of panobinostat in adults with sickle cell disease
📍 Augusta University · Augusta, GA · Get directions →
The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor.
- Sickle cell disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Testing how a new medicine works in healthy people and sickle cell patients
📍 University of Alabama Birmingham · Birmingham, AL · +5 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).
- Sickle cell disease
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Testing a bone marrow transplant with lower-intensity treatment for kids with sickle cell and
📍 Yale School of Medicine · New Haven, CT · +3 more US sites · Get directions →
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
- 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.
- Sickle cell disease
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
Testing whether children with sickle cell disease can take daily ADHD medicine and monitoring its
📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →
The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork problems and if they have any side effects. The study will assess any thinking or attention problems participants may have both before taking this drug and after.
- Sickle cell disease
Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Testing if adding ruxolitinib lowers graft failure after transplant in children with sickle cell
📍 Children's Hospital of Colorado · Aurora, CO · +3 more US sites · Get directions →
This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants.
- Heart / cardiovascular diseaseHigh blood pressureCOPD (chronic lung disease)
: Vascular Function in Health and Disease
Studying how blood vessel function works in health and diseases like heart problems, high blood
📍 George E Wahlen VA Medical Center · Salt Lake City, UT · Get directions →
Many control mechanisms exist which successfully match the supply of blood with the metabolic demand of various tissues under wide-ranging conditions. One primary regulator of vasomotion and thus perfusion to the muscle tissue is the host of chemical factors originating from the vascular endothelium and the muscle tissue, which collectively sets the level of vascular tone.
- Sickle cell disease
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
Testing a new transplant plan using immune-suppressing treatment before a blood stem cell transplant
📍 City of Hope Medical Center · Duarte, CA · Get directions →
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.
- COPD (chronic lung disease)
Hyper Polarized Xenon-129 MRI vs Xenon-133 Scintigraphy
Comparing a lung scan that uses xenon MRI with a radioactive scan for COPD
📍 University of Virginia Health System · Charlottesville, VA · Get directions →
Hyper polarized Xenon-129 MRI will be directly compared to a radioactive Xe-133 scintigraphy to detect defects in lung ventilation from airflow limitation. This study is conducted as a pilot study with intention to conduct a larger clinical trial.
- COPD (chronic lung disease)
Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure
Testing whether lung transplant first can improve lung function before bone marrow transplant for
📍 UPMC Presbyterian · Pittsburgh, PA · +1 more US site · Get directions →
The purpose of this study is to determine whether a lung transplantation prior to bone marrow transplantation (BMT) would allow for restoration of pulmonary function prior to BMT, allowing to proceed to BMT, to restore hematologic function.
- Sickle cell disease
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
Testing whether using mismatched donor stem cells works for severe sickle cell disease and other
📍 Children's Hospital of Pittsburgh of UPMC · Pittsburgh, PA · Get directions →
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease.
- Sickle cell disease
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Testing how oral medicines may help people with high-risk sickle cell disease
📍 University of Illinois at Chicago College of Medicine · Chicago, IL · Get directions →
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
- High blood pressureCOPD (chronic lung disease)Asthma
Hyperpolarized 129Xe MR Imaging of Lung Function in Healthy Volunteers and Subjects With Pulmonary Disease
Testing a lung scan method using hyperpolarized oxygen gas MRI in healthy and people with COPD or
📍 University of Kansas Medical Center · Kansas City, KS · Get directions →
The purpose of this study is to develop and evaluate the usefulness of hyperpolarized (HP) 129Xe gas MRI for regional assessment of pulmonary function.
- Sickle cell disease
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Testing gene correction in a person’s own blood-forming stem cells for severe sickle cell disease
📍 Children's Hospital Los Angeles · Los Angeles, CA · +5 more US sites · Get directions →
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
- Sickle cell disease
PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD
Using PET scans to find blood vessel blockage markers in sickle cell disease
📍 UPMC · Pittsburgh, PA · Get directions →
The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET).
- COPD (chronic lung disease)Asthma
Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cells for Pulmonary Diseases
Testing the safety of an IV stem cell treatment for lung diseases like COPD and asthma
📍 Medical Surgical Associates Center · St John's, · Get directions →
This trial will study the safety and efficacy of intravenous infusion of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of Pulmonary Diseases
- COPD (chronic lung disease)
Trial to Determine Effective Aspirin Dose in COPD
Testing what aspirin dose works best for people with COPD
📍 Johns Hopkins Bayview Medical Center · Baltimore, MD · Get directions →
Chronic obstructive pulmonary disease (COPD) is the fourth leading cause of death in the United States. Current treatments for COPD focus on inhaler therapies that do not address manifestations of the disease on other organ systems.
- Sickle cell disease
A Phase 2/3 Study in Adult and Adolescent Participants With SCD
Testing safety and how well a treatment works for sickle cell in teens and adults
📍 Smilow Cancer Hospital · New Haven, CT · +23 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.
- Sickle cell disease
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Testing the safety and effectiveness of a one-time gene-edited cell treatment for severe sickle cell
📍 New York Presbyterian Hospital - Morgan Stanley Children's Hospital · New York, NY · +2 more US sites · Get directions →
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
- COPD (chronic lung disease)
A Study to Investigate the Efficacy and Safety of Tezepelumab in Adult Participants With Moderate to Very Severe COPD (D5241C00007)
Testing whether tezepelumab is safe and effective for adults with moderate to severe COPD
📍 Research Site · Cullman, 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)