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- Heart / cardiovascular diseaseHigh blood pressureKidney disease
📍 Phoenix Children's Hospital · Phoenix, AZ · +39 more US sites · Get directions →
The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to evaluate the PK of understudied drugs currently being administered to children per SOC as prescribed by their treating provider.
✓ Study care freeLearn more → - Multiple sclerosis (MS)
📍 TG Therapeutics Investigational Trial Site · Birmingham, AL · +34 more US sites · Get directions →
The primary purpose of this phase 3b study is to assess the efficacy of a modified regimen of ublituximab in participants with relapsing multiple sclerosis (RMS) as measured by T1 Gadolinium (Gd)-enhancing lesions in Part A; PK in Part B along with efficacy of ublituximab as measured by T1 Gd-enhancing lesions in participants who had a suboptimal experience on prior anti-CD20 therapy in Part C. The study consists of 3 parts: Part A is single-armed and open-label, Part B is randomized, double-blind, placebo-controlled, and Part C is single-armed and open-label.
- Sickle cell disease
📍 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.
- Multiple sclerosis (MS)
📍 Perseverance Research Center · Scottsdale, AZ · +39 more US sites · Get directions →
The purpose of this Phase 3b study is to assess the efficacy, safety and tolerability of remibrutinib after switching from ocrelizumab and compared to continuous ocrelizumab treatment, in patients living with relapsing multiple sclerosis (plwRMS).
- Sickle cell disease
📍 Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital · Phoenix, AZ · +37 more US sites · Get directions →
In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval.
✓ Study care freeLearn more → - Sickle cell disease
📍 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.
- COPD (chronic lung disease)
📍 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)
📍 SEC Clinical Research, LLC · Dothan, AL · +39 more US sites · Get directions →
This study will assess the safety and efficacy of fixed dose combinations of ensifentrine with two different glycopyrrolate dose levels compared to placebo and to the individual components of the fixed dose combinations, each administered twice a day via standard jet nebulizer, in adult subjects with chronic obstructive pulmonary disease (COPD).
- Heart / cardiovascular diseaseDementia / Alzheimer's
📍 University of Alabama at Birmingham · Birmingham, AL · +37 more US sites · Get directions →
PREVENTABLE is a multi-center, randomized, parallel group, placebo-controlled superiority study. Participants will be randomized 1:1 to atorvastatin 40 mg or placebo.
- Multiple sclerosis (MS)
📍 Alabama Neurology Associates PC · Birmingham, AL · +32 more US sites · Get directions →
The purpose of this study is to provide efficacy and safety data for remibrutinib in patients with secondary progressive multiple sclerosis (SPMS)
- COPD (chronic lung disease)
📍 University of Alabama · Birmingham, AL · +22 more US sites · Get directions →
This research is being done to test if a drug called omalizumab can help people with Chronic Obstructive Pulmonary Disease (COPD) and allergies. Each participant will be in the study for about 16 months, including 1-3 months of screening, 12 months of receiving the study drug at a clinic, and a follow-up call one month after your final clinic visit.
- COPD (chronic lung disease)
📍 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
📍 Children's of Alabama (MRD-HCT) · Birmingham, AL · +30 more US sites · Get directions →
The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention.
- COPD (chronic lung disease)
📍 University of Alabama at Birmingham Lung Health Center · Birmingham, AL · +21 more US sites · Get directions →
This is a Phase 2, multicenter study in adult participants with an acute COPD exacerbation and type 2 inflammation
- COPD (chronic lung disease)
📍 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)
📍 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.
- Multiple sclerosis (MS)
📍 TG Therapeutics Investigational Trial Site · Birmingham, AL · +39 more US sites · Get directions →
The purpose of this study is to evaluate safety, effiectiveness, and to gain insight into the treatment experience of participants prescribed BRIUMVI® (ublituximab-xiiy) in the real-world setting
- Multiple sclerosis (MS)
📍 Stanford Multiple Sclerosis Center · Palo Alto, CA · +21 more US sites · Get directions →
This is a multi-center prospective rater-masked (blinded) randomized controlled trial of 156 participants, comparing the treatment strategy of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) to the treatment strategy of Best Available Therapy (BAT) for treatment-resistant relapsing multiple sclerosis (MS). Participants will be randomized at a 1 to 1 (1:1) ratio.
- Sickle cell disease
📍 University of Alabama at Birmingham · Birmingham, AL · +16 more US sites · Get directions →
This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing Study BTX-AUT-001.
- COPD (chronic lung disease)
📍 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)
- COPD (chronic lung disease)
📍 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
📍 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).
- Lupus (SLE)Rheumatoid arthritisMultiple sclerosis (MS)
📍 University of Alabama at Birmingham Cancer Center · Birmingham, AL · +39 more US sites · Get directions →
This phase Ib trial studies the side effects of nivolumab and to see how well it works alone and in combination with other treatments, such as ipilimumab, cabozantinib, platinum containing therapy, and fluoropyrimidine, in treating patients with autoimmune disorders and cancer that has spread from where it first started (primary site) to nearby tissue, lymph nodes, or distant parts of the body (advanced), to other places in the body (metastatic) or cannot removed by surgery (unresectable). Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
- Multiple sclerosis (MS)
📍 Local Institution - 0011 · Birmingham, AL · +16 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG).
- 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.
- COPD (chronic lung disease)
📍 UAB School of Medicine/Lung Health Center · Birmingham, AL · +24 more US sites · Get directions →
Parallel-group, prospective, randomized, controlled phase III trial of home High flow Nasal Therapy (HFNT) via myAirvo 3 plus usual COPD medical care vs. usual COPD medical care, for at least 1 year and up to two years in 642 GOLD Grade D, Stages II-IV patients with moderate to very severe COPD at risk for moderate and severe exacerbations with a prior history of severe exacerbation requiring hospitalization within the past 6 weeks.
- COPD (chronic lung disease)
📍 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.
- Multiple sclerosis (MS)
📍 North Central Neurology Associates- Site Number : 8401100 · Cullman, AL · +21 more US sites · Get directions →
This is a randomized, open-label, parallel, Phase 3 study with 2-arms for treatment. The purpose of this study is to evaluate SC administration of frexalimab every 4 weeks (q4w) compared to IV administration of frexalimab q4w in male and female participants with RMS and nrSPMS (aged 18 to 60 years at the time of enrollment).
- COPD (chronic lung disease)
📍 University of Alabama at Birmingham · Birmingham, AL · +17 more US sites · Get directions →
The objective of this study is to assess the safety and efficacy of Apreo BREATHE system when used to support native airways and release trapped air in the treatment of adult COPD patients with emphysema suffering from dyspnea due to hyperinflation despite optimal medical treatment. The Apreo BREATHE Airway Scaffold is a permanent implant designed to tent open native airways.
- Multiple sclerosis (MS)
📍 Alabama Neurology Associates · Birmingham, AL · +14 more US sites · Get directions →
The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986368 in participants with Multiple Sclerosis Spasticity
- COPD (chronic lung disease)
📍 Banner University Medical Center · Phoenix, AZ · +17 more US sites · Get directions →
This is a prospective, open-label, multi-center, single-arm study planned to enroll 200 subjects with heterogeneous emphysema and collateral ventilation (CV) in the target lobe. Subjects will undergo instillation of AeriSeal Foam in the target lobe and subsequent assessment of CV status using Chartis Pulmonary Assessment System.
- 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)
📍 University of Alabama at Birmingham · Birmingham, AL · +12 more US sites · Get directions →
This is an observational study of 1000 participants to further define the nature of early chronic obstructive pulmonary disease (COPD) in younger, at-risk individuals. The study has three main goals: * To use CT scan imaging to identify which smokers will develop COPD.
- COPD (chronic lung disease)
📍 Research Site · Birmingham, AL · +39 more US sites · Get directions →
This Phase IIb dose-ranging study will evaluate the efficacy and safety of 2 different doses of AZD6793 compared with placebo tablets in participants with moderate to very severe chronic obstructive pulmonary disease.
- Heart / cardiovascular diseaseKidney disease
Sotagliflozin to Slow Kidney Function Decline in Persons With Type 1 Diabetes and Diabetic Kidney Disease
💵 May compensate📍 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.
- COPD (chronic lung disease)
📍 Dignity Health St. Joseph's Hospital and Medical Center · Phoenix, AZ · +13 more US sites · Get directions →
This is a single-arm, prospective, multi-center, Registry study to evaluate the long-term safety and effectiveness of the Spiration Valve System (SVS) for the treatment of severe emphysema in a post-market setting.
- Sickle cell disease
📍 University of South Alabama Medical Center · Mobile, AL · +13 more US sites · Get directions →
The study, known as the Peer suppoRt for adolescents and Emerging adults with Sickle cell pain: promoting ENgagement in Cognitive behavioral thErapy (PRESENCE), aims to determine the effectiveness of digital CBT in reducing pain, opioid use, and healthcare utilization among AYAs with SCD. It also seeks to understand the role of personalized peer support in enhancing engagement and outcomes of digital CBT interventions.
- Sickle cell disease
📍 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.
- Multiple sclerosis (MS)
📍 Arizona Neuroscience Research, LLC · Pheonix, AZ · +12 more US sites · Get directions →
Orelabrutinib is a CNS-penetrable BTK inhibitor. This is a phase 3, randomized, double-blind, parallel-group, multicenter study to evaluate the efficacy and safety of orelabrutinib compared with placebo in patients with PPMS.
- Sickle cell disease
📍 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.
- Heart / cardiovascular diseaseHigh blood pressureKidney disease
📍 Stanford Hospital and Clinics · Stanford, CA · +11 more US sites · Get directions →
The purpose of the SPYRAL GEMINI Pilot Study is to evaluate that multi-organ denervation with the Gemini System is safe and provide evidence of blood pressure reduction when studied in an uncontrolled hypertensive population with and without high cardiovascular risk.
- Multiple sclerosis (MS)
📍 Yale School of Medicine · New Haven, CT · +12 more US sites · Get directions →
This study is a prospective, multi-center, randomized, double blinded, placebo-controlled study of OCR treatment-discontinuation in patients with early RMS. All eligible participants will be initiated on OCR using the standard approved administration schedule of two 300 mg infusions separated by 14 days (i.e., Days 0 and 14) for a total of 600 mg, followed by 600 mg infusions at Month 6,12, 18, and 24.
- COPD (chronic lung disease)
📍 DM Clinical Research - Phoenix · Phoenix, AZ · +11 more US sites · Get directions →
This is a Phase 2 study of the efficacy, safety, tolerability and pharmacokinetics of two doses of EXPD-101 in participants with COPD. Study participants will be randomized to receive either study drug or placebo administered once daily for 52 weeks.
- COPD (chronic lung disease)
📍 University of Alabama at Birmingham Hospital · Birmingham, AL · +39 more US sites · Get directions →
A Randomized, Double-blind, Placebo-controlled, Multiple Dose-Ranging Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Solrikitug in Participants with Chronic Obstructive Pulmonary Disease (COPD).
- COPD (chronic lung disease)
📍 University of Alabama at Birmingham - School of Medicine- Site Number : 8400003 · Birmingham, AL · +9 more US sites · Get directions →
LPS18583 is a multinational, randomized, double-blind, placebo-controlled, parallel-group, Phase 4 study with 2 treatment groups. The purpose of this study is to assess the effect of dupilumab compared with placebo on airway inflammation, resistance, and remodeling including mucus plugging and its association with improvement on lung function, exacerbations, and quality of life improvement in participants aged 40 years of age up to 85 years of age (inclusive).
- Multiple sclerosis (MS)
📍 Local Institution - 0114 · Loma Linda, CA · +11 more US sites · Get directions →
The purpose of this study is to evaluate the effectiveness, safety, tolerability, drug levels and drug effects of ozanimod compared to fingolimod in children and adolescents with relapsing remitting multiple sclerosis (RRMS).
- Multiple sclerosis (MS)
📍 Arizona Neuroscience Research, LLC · Phoenix, AZ · +12 more US sites · Get directions →
Orelabrutinib is a CNS-penetrable BTK inhibitor. This is a phase 3, randomized, double-blind, parallel-group, multicenter study to evaluate the efficacy and safety of orelabrutinib compared with placebo in patients with non-active Secondary Progress MS.
- Multiple sclerosis (MS)Crohn's / ulcerative colitis (IBD)
📍 Knowledge Research Center · Orange, CA · +11 more US sites · Get directions →
The most clinically meaningful way to discover new targets of T cells in autoimmune diseases is to study the tissues of patients with active autoimmune disease mediated organ inflammation. These tissues contain both cytotoxic and helper T cells that are driving their disease, and these T cells are being guided by TCRs that recognize tissue-specific targets.
- COPD (chronic lung disease)
📍 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
📍 University of Alabama, Birmingham (UAB) · Birmingham, AL · +8 more US sites · Get directions →
The goal of this observational study is to help us understand more about the best ways to help individuals living with Sickle Cell Disease (SCD) get the best care. The main question it aims to answer is: How to find individuals unaffiliated from SCD specialist care use three distinct pathways?
- Multiple sclerosis (MS)
📍 Profound Research, LLC · Carlsbad, CA · +8 more US sites · Get directions →
The main purpose of this study is to assess the bioequivalence of ocrelizumab SC test formulation to the marketed ocrelizumab SC reference formulation in participants with either relapsing multiple sclerosis (RMS) or primary progressive multiple sclerosis (PPMS). The study consists of 2 phases: a controlled phase, where participants in each group will receive one dose of test or reference formulation and a continuation phase, where all participants in both groups will receive ocrelizumab SC test formulation.
- Multiple sclerosis (MS)
📍 Research Site · Tucson, AZ · +8 more US sites · Get directions →
This trial is a Phase 1b, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with Multiple Sclerosis.
- COPD (chronic lung disease)
📍 University of Alabama-Birmingham Hospital - UAB Lung Health Center · Birmingham, AL · +9 more US sites · Get directions →
The dNerva Lung Denervation System is one-time treatment intended to improve breathing in Chronic Obstructive Pulmonary Disease (COPD) patients on standard medical care. The purpose of this study is to confirm the safety and efficacy of the Nuvaira Lung Denervation System in the treatment of COPD.
- Sickle cell disease
📍 University of Alabama · Birmingham, AL · +9 more US sites · Get directions →
The main purpose of this study is to create a longitudinal cohort of those with Sickle Cell Trait (SCT) to better understand the hematologic phenotype for those that carry HbS, assess for differences in those with varying quantities of HbS and assess for potential clinical complications of SCT.
- Obesity / overweightHeart / cardiovascular disease
📍 Site 108 · Chandler, AZ · +11 more US sites · Get directions →
The purpose of this study is to learn about the effects of BGE-102 on blood biomarkers including hsCRP, which is a lab value used to measure inflammation. BGE-102 decreases inflammation which may improve obesity and cardiovascular health.
- Obesity / overweightHeart / cardiovascular disease
Maridebart Cafraglutide in Heart Failure With Preserved or Mildly Reduced Ejection Fraction and Obesity
💵 May compensate📍 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).
- Heart / cardiovascular diseaseHigh blood pressure
📍 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.
- Sickle cell disease
📍 University Of Alabama · Birmingham, AL · +6 more US sites · Get directions →
This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator.
- COPD (chronic lung disease)
📍 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.
- 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.
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