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- Parkinson's disease
📍 Banner Sun Health Research Institute · Sun City, AZ · +39 more US sites · Get directions →
The goal of this Phase 2 clinical trial is to investigate the efficacy and safety of NEU-411 in men and women aged 40-80 years with early Parkinson's Disease (PD) who have predicted elevations in the activity of the "leucine-rich repeat kinase 2" ("LRRK2" for short) pathway based on their genetic profile. A DNA test will be used to identify the "LRRK2-driven" population with predicted elevation in the LRRK2 pathway.
- Parkinson's disease
📍 University of Alabama at Birmingham · Birmingham, AL · +26 more US sites · Get directions →
This study will examine the long-term safety of buntanetap in participants with PD. This will be a 36-month open-label safety study.
- Parkinson's disease
📍 Mayo Clinic · Phoenix, AZ · +33 more US sites · Get directions →
The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.
- 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.
- Multiple sclerosis (MS)
📍 INECO Neurociencias Orono · Rosario, Santa Fe Province · Get directions →
This open label, single arm study will evaluate the PK and PD effects of fenebrutinib in children and adolescents with RMS aged between 10 and \< 18 years. This study consists of a Dose Exploration Period and an Optional Extension Period.
- Lupus (SLE)
📍 Clnica SER da Bahia · Salvador, Estado de Bahia · Get directions →
This study will assess how mosunetuzumab works in people who have systemic lupus erythematosus (SLE) who may or may not also have active lupus nephritis (LN).
- Lupus (SLE)
📍 Local Institution - 0106 · Phoenix, AZ · +39 more US sites · Get directions →
The purpose of this study is to evaluate the efficacy, safety and drug levels of CC-97540 in participants with active systemic lupus erythematosus (SLE) including lupus nephritis with inadequate response to glucocorticoids and at least 2 immunosuppressants.
- Parkinson's disease
📍 University of Arkansas for Medical Sciences · Little Rock, AR · +24 more US sites · Get directions →
This is a Phase 2 study in people with Parkinson's disease who experience motor fluctuations while taking levodopa. The study will evaluate how effective two different doses of the study drug AP-472 are when added to levodopa treatment, compared with a placebo.
- Lupus (SLE)
📍 AKDHC Medical Research ServicesLLC · Phoenix, AZ · +25 more US sites · Get directions →
The overall purpose of this two-part study is to evaluate the efficacy, safety and tolerability of iptacopan (LNP023) in addition to standard of care treatment.
- 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.
- Multiple sclerosis (MS)
📍 The Hospital for Sick Children · Toronto, Ontario · Get directions →
A randomized multiple baseline feasibility trial where participants will start taking metformin at one of 3 randomly determined points (3-months, 6-months or 9 months) during the 12-month trial. All subjects will be on a daily dose of metformin for a minimum of 3 months and a maximum of 9 months.
- 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.
- Lupus (SLE)
📍 University of California, San Francisco · San Francisco, CA · +18 more US sites · Get directions →
The study is a 1-year 2-part double-blinded placebo controlled 2-arm clinical trial. Treatment arms are (1) MMF dosed as per body-surface area (MMFBSA; 600mg/m2 body surface area per dose about every 12 hours) and (2) pharmacokinetically-guided precision-dosing of MMF (MMFPK; MMF dosed twice daily to achieve an area under the concentration-time curve (AUC0-12h) of MPA \>60-70 mg\*h/L.
- Lupus (SLE)
📍 University of California Irvine · Orange, CA · +20 more US sites · Get directions →
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
- Lupus (SLE)
📍 Wallace & Lee Center · Beverly Hills, CA · +29 more US sites · Get directions →
This study is open to adults with systemic lupus erythematosus (SLE). The purpose of this study is to find out whether a medicine called BI 3000202 helps people with SLE.
- 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
- Parkinson's disease
📍 Site-049 · Birmingham, AL · +14 more US sites · Get directions →
A study to determine if BHV-8000 is efficacious, safe and tolerable in adults diagnosed with early Parkinson's disease.
- Lupus (SLE)
📍 University Of Alabama · Birmingham, AL · +18 more US sites · Get directions →
The purpose of this study is to evaluate the efficacy and safety of rapcabtagene autoleucel (administered once following lymphodepletion) in patients with active, refractory systemic lupus erythematosus (SLE) or active, refractory lupus nephritis (LN).
- Lupus (SLE)
📍 Nkarta Investigational Site · Little Rock, AR · +16 more US sites · Get directions →
This is a Phase 1/2, open-label, multi-center, multi-cohort, non-randomized dose escalation and dose expansion basket study to determine the safety and tolerability of NKX019 (allogeneic CAR NK cells targeting CD19) in participants with autoimmune diseases.
- Lupus (SLE)
📍 Research Site · San Francisco, CA · +14 more US sites · Get directions →
This is a Phase 1b/2, single-arm, open-label, multi-center, clinical study of AZD0120, a CD19/BCMA dual CAR T cell therapy, to evaluate the safety, tolerability, and efficacy in adult participants with refractory Systemic Lupus Erythematosus.
- Lupus (SLE)
📍 University of California San Diego School of Medicine: Division of Rheumatology, Allergy and Immunology · La Jolla, CA · +16 more US sites · Get directions →
This is a multi-center double-blind placebo controlled clinical trial evaluating the efficacy of VIB4920 combined with mycophenolate mofetil (MMF) and prednisone in achieving a renal response in participants with active lupus nephritis (LN).
- 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.
- Lupus (SLE)Rheumatoid arthritis
📍 HonorHealth Research and Innovation Institute · Scottsdale, AZ · +16 more US sites · Get directions →
The main objective is to assess the safety and tolerability of inebilizumab in adult participants with active and refractory systemic lupus erythematosus (SLE) with nephritis (Subprotocol A) and to assess the safety and tolerability of subcutaneous (SC) blinatumomab in adult participants with active and refractory SLE with and without nephritis (Subprotocol B Part A) and in adult participants with active refractory rheumatoid arthritis (RA) (Subprotocol C Part A). The trial will also assess the efficacy of SC blinatumomab in adult participants with active and refractory SLE with and without nephritis (Subprotocol B Part B and Subprotocol C Part B).
- Lupus (SLE)
📍 Loma Linda University health · Loma Linda, CA · +10 more US sites · Get directions →
This phase II, randomized, double-blind, placebo-controlled study is designed to evaluate the safety, efficacy and pharmacokinetics (PK) of obinutuzumab in adolescent participants (AP) aged 12 to less than 18 with biopsy-confirmed proliferative lupus nephritis (LN). It will also evaluate open label safety and PK of obinutuzumab in pediatric participants (PP), aged 5 to \<12 with LN.
- 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.
- Lupus (SLE)
📍 Sierra Pacific Arthritis and Rheumatology Centers · Fresno, CA · +15 more US sites · Get directions →
This study aims to examine the efficacy and safety of obexelimab in participants with systemic lupus erythematosus (SLE).
- 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)
📍 Byers Hall · San Francisco, CA · Get directions →
The main purpose of this study is to assess whether hyperpolarized carbon imaging in relapsing remitting multiple sclerosis (MS) patients can be used to predict response to anti-CD20 disease modifying therapy. Study procedures will include magnetic resonance imaging (MRI) assessments with a hyperpolarized pyruvate sequence, clinical assessment as well as blood markers of disease progression.
- Lupus (SLE)
📍 Investigative Site · Beverly Hills, CA · +9 more US sites · Get directions →
The goal of this clinical trial is to learn if VENT-03 works to treat patients with cutaneous lupus erythematosus (CLE) who may or may not have systemic lupus erythematosus (SLE). Another goal is to learn about the safety of VENT-03 and how it is processed by the body.
- 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)
📍 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.
- 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.
- Lupus (SLE)
📍 US06-0 · La Palma, CA · +5 more US sites · Get directions →
A Phase 1/2a Study of DB-2304 in Healthy Participants and Participants with Systemic Lupus Erythematosus or Cutaneous Lupus Erythematosus
- 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
📍 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
📍 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.
- Sickle cell disease
📍 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.
- Parkinson's disease
📍 Cornell University Weill Medical College · New York, NY · Get directions →
This is a phase 1/2 clinical study to evaluate the safety, tolerability, feasibility, and preliminary efficacy of NouvNeu001 in patients with advanced Parkinson's Disease.
- Lupus (SLE)
📍 State University of New York Upstate Medical Center (SUNY) · Syracuse, NY · +1 more US site · Get directions →
The purpose of this trial is to evaluate the efficacy and safety of obecabtagene autoleucel (obe-cel) administered once following lymphodepletion in participants with severe, refractory systemic lupus erythematosus (SLE) and active lupus nephritis (LN).
- Parkinson's disease
📍 Institute for Neurodegenerative Disorders · New Haven, CT · +1 more US site · Get directions →
The study is a longitudinal, multi-center study to assess progression of \[18F\] AV-133 imaging in Prodromal PD participants. Participants will be followed for up to 24 months.
- 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.
- Dementia / Alzheimer'sParkinson's diseaseMultiple sclerosis (MS)
Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers
📍 UCSF · San Francisco, CA · +2 more US sites · Get directions →
This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs. 18F-OP-801 is intended as a biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation.
- High cholesterolLupus (SLE)
📍 Hospital for Special Surgery · New York, NY · +1 more US site · Get directions →
This treatment trial evaluates the addition of an anti-tumor necrosis factor-alpha drug, certolizumab, to usual treatment (a heparin agent and low-dose aspirin) in pregnant women with antiphospholipid syndrome (APS) and repeatedly positive tests for lupus anticoagulant (LAC) to determine if this regimen will improve pregnancy outcomes. All enrolled patients will receive certolizumab, and pregnancy outcomes will be compared to those of women with APS and repeatedly positive tests for LAC enrolled in a previous study by the investigators.
- Parkinson's disease
📍 University of Kentucky Medical Center · Lexington, KY · +1 more US site · Get directions →
The Goal of this study is to evaluate the safety, tolerability, and clinical responses following implantation of DSP-1083. Study enrolls both male and female patients in 2 cohorts.
- Parkinson's disease
📍 Purdue University · West Lafayette, IN · +1 more US site · Get directions →
Airway protective disorders are a prevalent and progressive consequence of Parkinson's Disease (PD), and often result in aspiration pneumonia which is the leading cause of death in PD. Despite this, a large number of patients with PD do not access specialized services to address these critical deficits.
- Lupus (SLE)
📍 H01- Children's National Hospital · Washington D.C., DC · +1 more US site · Get directions →
Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis
- Multiple sclerosis (MS)
📍 Johns Hopkins University · Baltimore, MD · +1 more US site · Get directions →
The goal of this clinical trial is to evaluate if the study drug will reduce brain and retinal atrophy by reducing inflammation and subsequently slowing neurodegeneration in people with Multiple Sclerosis. The main outcome for the trial is change in normalized brain parenchymal volume (nBPV), measured by magnetic resonance imaging (MRI).
- Multiple sclerosis (MS)
📍 University of Kansas Medical Center · Kansas City, KS · +1 more US site · Get directions →
The goal of this clinical trial is to treat male and female participants with two types of Multiple Sclerosis (MS) called primary progressive or secondary progressive MS. The main questions the trial aims to answer are the following: * Is TRX319 safe when administered to patients with progressive forms of MS?
- Parkinson's disease
📍 Institute for Neuroimmune Medicine · Davie, FL · +2 more US sites · Get directions →
This study will be evaluating the safety and efficacy of insulin and glutathione in subjects with Parkinson's Disease compared to placebo.
- Lupus (SLE)
📍 MD Anderson Cancer Center · Houston, TX · +1 more US site · Get directions →
The goal of Safety Lead-In is to confirm the safety of tafasitamab when given to patients with SSc, SLE, and LN. The goal of Phase 1 is to find the recommended dose of AD-PluReceptor-NK cells in combination with tafasitamab and lymphodepleting chemotherapy that can be given to patients with the disease.
- Sickle cell disease
📍 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
📍 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).
- 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.
- Parkinson's disease
📍 Jesse Brown VA Medical Center, Chicago, IL · Chicago, IL · +1 more US site · Get directions →
The purpose of this study is to examine safety, feasibility, and the behavioral and brain effects of a non-invasive treatment, repetitive transcranial magnetic stimulation (rTMS), for Veterans with Parkinson's disease or atypical parkinsonism and mild impairments in their thinking. The hypothesis is that rTMS can improve thinking for people with Parkinson's disease or atypical parkinsonism who are experiencing mild problems with their thinking ability.
- Sickle cell disease
📍 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.
- Sickle cell disease
📍 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
- DepressionParkinson's disease
📍 University of California, San Francisco · San Francisco, CA · Get directions →
The purpose of this study is to understand whether people with Parkinson's Disease and depression have improvement in their symptoms after psilocybin therapy.
- Parkinson's disease
📍 San Francisco VA Medical Center, San Francisco, CA · San Francisco, CA · Get directions →
Parkinson's disease (PD) is a devastating illness that has a growing impact on Veterans. One of the most disabling symptoms is depression, which is common in PD and linked to poor quality of life and higher risk of suicide.
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