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- 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.
- 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)
- 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.
- 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
- Fatty liver disease (NASH/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
- 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.
- 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.
- Fatty liver disease (NASH/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.
- 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).
- 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
- 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.
- Fatty liver disease (NASH/MASH)
📍 Arizona Liver Health · Chandler, AZ · +39 more US sites · Get directions →
This study is researching an investigational drug, ALN-HSD called "study drug". This study is focused on participants who are known to have Metabolic dysfunction-Associated SteatoHepatitis (MASH).
- Fatty liver disease (NASH/MASH)
📍 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.
- 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.
- Fatty liver disease (NASH/MASH)
A Study of Efimosfermin Alfa in Participants With Biopsy-confirmed Cirrhosis (Compensated) Due to MASH
💵 May compensate📍 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.
- 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.
- 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.
- Fatty liver disease (NASH/MASH)
📍 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.
- Fatty liver disease (NASH/MASH)
A Pivotal Clinical Study to Investigate Efimosfermin Alfa in Participants With Biopsy-confirmed F2- or F3-stage MASH
💵 May compensate📍 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.
- 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.
- 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?
- Fatty liver disease (NASH/MASH)
A Study Evaluating Efruxifermin in Subjects With Compensated Cirrhosis Due to NASH/MASH
💵 May compensate📍 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.
- 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.
- 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.
- Fatty liver disease (NASH/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.
- Fatty liver disease (NASH/MASH)
LIVERAGE™: A Study to Test Whether Survodutide Helps People With a Liver Disease Called NASH/MASH Who Have Moderate or Advanced Liver Fibrosis
💵 May compensate📍 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)
A Study to Evaluate the Efficacy and Safety of Pegozafermin in Participants With Compensated Cirrhosis Due to MASH
💵 May compensate📍 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
📍 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.
- Fatty liver disease (NASH/MASH)
Non-Invasive Quantification of Liver Health in NASH (N-QUAN)
💵 May compensate📍 Arizona Liver Health · Chandler, AZ · +6 more US sites · Get directions →
To evaluate, in patients with suspected NASH referred for liver biopsy, the diagnostic performance of CT1 at discriminating those with NAS≥4 \& F≥2 from those without.
- 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.
- Sickle cell disease
📍 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).
- Multiple sclerosis (MS)
📍 Shepherd Center · Atlanta, GA · +5 more US sites · Get directions →
The MS pilot study will assess the safety and investigate the remyelinating effects of the SetPoint System (study device) in adult patients with patients diagnosed with relapsing-remitting multiple sclerosis (RRMS). The SetPoint System is intended for adjunctive use with standard of care therapy for RRMS.
- Multiple sclerosis (MS)
📍 University of Alabama at Birmingham · Birmingham, AL · +5 more US sites · Get directions →
This study is structured around three main aims. In Aim 1, investigators will conduct community-based participatory research (CBPR) to develop culturally tailored methods to assess childhood adversity in multiple sclerosis (MS).
- Multiple sclerosis (MS)
📍 Washington VA Medical Center · Washington D.C., DC · +5 more US sites · Get directions →
CAFE-MS will assess the effectiveness of two online programs for fatigue in multiple sclerosis (MS). Although they differ, both of these online programs contain information about MS and fatigue intended to help people with MS understand and manage their fatigue.
- Fatty liver disease (NASH/MASH)
LIVERAGE™ - Cirrhosis: A Study to Test Whether Survodutide Helps People With a Liver Disease Called NASH/MASH Who Have Cirrhosis
💵 May compensate📍 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)
📍 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.
- Multiple sclerosis (MS)
📍 Stanford University Medical Center · Stanford, CA · +4 more US sites · Get directions →
The primary purpose of the study is to evaluate the safety and tolerability of a single-ascending intravenous (IV) dose (Part 1), a single-ascending subcutaneous (SC) dose (Part 2), and multiple ascending SC doses (Part 3) of RO7121932 in participants with multiple sclerosis (MS).
- Multiple sclerosis (MS)
📍 Boston Clinical Trials Inc - Internal Medicine · Boston, MA · +7 more US sites · Get directions →
The primary objective of this trial is to evaluate the safety and reactogenicity of mRNA-1195 in participants with multiple sclerosis.
- Multiple sclerosis (MS)
📍 Profound Research, LLC · Carlsbad, CA · +5 more US sites · Get directions →
The main purpose of this study is to evaluate the safety and tolerability of the ocrelizumab subcutaneous (SC) test formulation in participants with multiple sclerosis (MS). The study consists of two treatment phases: a dose-escalation and dose-continuation phase.
- Sickle cell disease
📍 Children's National Health System · Washington D.C., DC · +4 more US sites · Get directions →
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
- Sickle cell disease
📍 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
📍 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.
- Multiple sclerosis (MS)
📍 PROVIDE Virtual Research Coordination Center · San Francisco, CA · +4 more US sites · Get directions →
The primary objective of the lactation study is to characterize the presence and concentration of BRIUMVI™ in breast milk among breastfeeding participants who receive BRIUMVI™ therapeutically for the treatment of relapsing forms of multiple sclerosis (RMS).
- Obesity / overweightFatty liver disease (NASH/MASH)
Fibrosis Lessens After Metabolic Surgery
💵 May compensate📍 Banner Health Center · Phoenix, AZ · +3 more US sites · Get directions →
Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), a major global public health concern, is commonly associated with obesity, diabetes, and dyslipidemia. MASLD is currently the most common cause of chronic liver disease affecting about 80% of people with obesity, ranging from simple fat deposits in the liver to Metabolic Dysfunction-Associated Steatohepatitis (MASH), cellular injury, advanced fibrosis, cirrhosis, or hepatocellular carcinoma.
- 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.
- Sickle cell disease
📍 Children's Hospital of Illinois at OSF-Saint Francis Medical Center · Peoria, IL · +5 more US sites · Get directions →
Despite the important work of previous sickle cell disease (SCD) cohort studies, there remain many understudied areas that require investigation. An important knowledge deficit is the slow but progressive process of chronic end-organ dysfunction.
✓ Study care freeLearn more → - 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.
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