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NCT05560646
The purpose of this global Phase 2 study is to determine the efficacy, safety, and tolerability of 3 dose levels of OG-6219 in pre-menopausal women between 18 and 49 years of age (inclusive), who have moderate to severe endometriosis-related pain.
NCT04456673
Primary Objective: To evaluate the efficacy of dupilumab administered every 2 weeks in patients with moderate or severe Chronic Obstructive Pulmonary Disease (COPD) as measured by * Annualized rate of acute moderate or severe COPD exacerbation (AECOPD) Secondary Objectives: To evaluate the effect of dupilumab administered every 2 weeks on * Pre-bronchodilator forced expiratory volume in 1 second (FEV1) over 12 weeks compared to placebo * Health related quality of life, assessed by the change from baseline to Week 52 in the St. George's Respiratory Questionnaire (SGRQ) * Pre-bronchodilator FEV1 over 52 weeks compared to placebo * Lung function assessments * Moderate and severe COPD exacerbations * To evaluate safety and tolerability * To evaluate dupilumab systemic exposure and incidence of antidrug antibodies (ADA)
NCT04391569
This study evaluated the effectiveness and safety of an investigational product (IP), intravenous (IV) ganaxolone, to treat participants with status epilepticus (SE).
NCT05501366
One important difference between KE compounds is the ketone-promoting components, which determines the circulating ratio of blood ketone bodies, BHB and AcAc, and may in turn lead to important metabolic and signaling differences. Whereas some actions of the ketone bodies BHB and AcAc are shared, R-BHB has a broad range of signaling functions that are distinct from AcAc, some of which are shared by the non-circulating, non-oxidizable enantiomer, S-BHB. AcAc also has metabolic and signaling actions that are independent of BHB and is selectively oxidized in some cells that cannot oxidize BHB. Furthermore, responses to different ketone bodies vary between tissue types. A second difference between KE arises from the balance between direct delivery of ketones compared to indirectly elevating ketone concentration via metabolism of non-classical or classical ketogenic precursors. Classical ketogenesis itself may drive adaptation and some of the functional benefits associated with ketosis. BDO is included in all of the KE compounds, but it is currently unknown how consumption of BDO alone, and its metabolism via non-classical ketogenesis acutely affects metabolism. Additionally, ketogenesis is now understood to occur in certain cells outside the liver with important local biological effects, for example ketogenesis driven by medium chain fatty acids has been reported in astrocytes in vitro. Provision of systemic BHB by a KE may elicit different biological effects in some tissues such as the brain versus promoting in situ ketogenesis in that tissue. Overall, not only are functional effects of KE incompletely defined, but also it is unknown which effects are common to all KE versus which are specific to an individual KE compound (i.e., BHB Monoester vs AcAc Diester) or which may be attributable to the BDO precursor common to all of the KE. This study will be the first comparative full crossover study of all available KE and the precursor BDO at two serving sizes. Outcomes will focus on established effects of the BHB Monoester (including the effects on ketones, glucose and acid-base balance) and compare these with the effects of the AcAc Diester, C8 Ketonef Diester and BDO.
NCT05994612
Suicide is the leading cause of death among YEH and most youth do not access services that may be available to them. Therefore, this study seeks to address this gap in the research literature with the goal to identify an effective intervention that can be readily adopted by communities that serve these youth. We will test the effects of outreach-worker delivered Cognitive Therapy for Suicide Prevention (CTSP)+Services as Usual (SAU) versus SAU alone on suicidal ideation (primary outcome), substance use and depressive symptoms (secondary outcomes) at 3, 6, 9 and 12- months.
NCT02713386
This phase I/II trial studies the side effects and the best dose of ruxolitinib phosphate when given together with paclitaxel and carboplatin and to see how well they work in treating patients with stage III-IV epithelial ovarian, fallopian tube, or primary peritoneal cancer. Ruxolitinib phosphate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as paclitaxel and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ruxolitinib phosphate together with paclitaxel and carboplatin may be a better treatment for epithelial ovarian, fallopian tube, or primary peritoneal cancer compared to paclitaxel and carboplatin alone.
NCT03089398
The purpose of the study is to learn which treatment option is better for patients who have multi-vessel coronary artery disease (blockages in more than one vessel supplying blood to the heart muscle). The treatment options this study will compare are: (1) Hybrid Coronary Revascularization \[HCR\] (a combination of surgery and catheter procedures to open up clogged heart arteries) and (2) Percutaneous Coronary Intervention \[PCI\] (catheter procedures alone to open up clogged heart arteries). There are no new or "experimental" procedures being tested in this study: both HCR and PCI are well-established procedures and are regularly performed in patients who have coronary artery disease. But, the FDA has not approved the drug-eluting stents used in PCI for all types of coronary artery disease. We have received an Investigational Device Exemption from the FDA to use the drug-eluting stents in this trial in the same way that they are used in clinical practice. The study being proposed here will use rigorous scientific methods and should result in a very high level of certainty about which procedure is best for patients with coronary artery disease.
NCT05089734
The goal of this clinical study is to compare the study drug, sacituzumab govitecan-hziy (SG), versus docetaxel in participants with advanced or metastatic (cancer that has spread) non-small cell lung cancer (NSCLC).
NCT06862648
The goal of this pivotal clinical trial is to evaluate the safety and effectiveness of the SUI-100 device for the treatment of mild-to-moderate stress urinary incontinence (SUI) in females aged 22-70 years. The main questions this study aims to answer are: 1. Does treatment with the SUI-100 device reduce SUI symptoms, as measured by urine leakage. 2. Is the device safe and effective compared to the sham group? Participants will: 1. Be randomly assigned to an active or sham control group in a blinded, multicenter study. 2. Attend treatment visits during the Treatment Phase, with SUI assessments conducted at specific intervals. 3. Enter a 3-month, treatment-free follow-up phase, with SUI assessment. 4. Proceed to three monthly maintenance treatments, followed by a final SUI assessment at study exit. The total study duration will last approximately 18 months, with each individual's participation lasting approximately 6-8 months. This study aims to provide robust data on the effectiveness, safety, and durability of the SUI-100 device for treating female Stress Urinary Incontinence.
NCT03867097
This is a Phase 2, multicenter, double-blind, randomized, placebo-controlled study to evaluate the effect of iloprost on the symptomatic relief of Raynaud's Phenomenon attacks in subjects with symptomatic Raynaud's Phenomenon secondary to Systemic Sclerosis.
NCT05987592
The goal of this randomized clinical trial is to evaluate two different non-drug, virtual treatment options designed to improve the lives of patients with migraine. Both interventions involve 8 weekly sessions and an online platform with additional content and learning. Participants can stay on all their medications during this study. Information from this study may help determine how to better treat migraine.
NCT01764529
Cerebral cavernous malformations (CCMs) are clusters of abnormal blood vessels in the brain and spine. CCMs can bleed and cause strokes, seizures, and headaches. CCMs are often caused by an inherited gene mutation (alteration) in one of three CCM genes (CCM1, CCM2, or CCM3). There is a wide range of disease severity even among family members with this disease, though the natural history has not been clearly described for this particular population. This study will continue to enroll and follow participants with familial CCM to identify factors that influence CCM disease severity and progression, focusing on barriers to clinical trial preparedness. Our long-term goal is to identify measurable outcomes and robust biomarkers that will help select high-risk patients and help monitor drug response in future clinical trials. The specific goals of this study are to: * Identify factors that influence lesion progression to symptomatic hemorrhage and other outcomes, including quality of life; * Investigate the role of the gut microbiome and lesion burden in CCM disease, and * Identify blood biomarkers predictive of CCM disease severity and progression for clinical trials.
NCT05049655
An observational study to generate a long-term outcome database from medical records for infants who completed protocol SS-101-18 or infants who are breastfed, fed commercial formula, or fed a marketed ByHeart formula.
NCT01974440
The purpose of this study is to evaluate the efficacy and safety of PCI-32765 (ibrutinib) administered in combination with either bendamustine and rituximab (BR) or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) in adult participants with previously treated indolent Non-Hodgkin lymphoma.
NCT02953509
The primary objectives of this study are: * To investigate the safety and tolerability, and to define the recommended Phase 2 dose and schedule (RP2DS) for magrolimab in combination with rituximab and for magrolimab in combination with rituximab, gemcitabine, and oxaliplatin (R-GemOx). * To evaluate the efficacy of magrolimab in combination with rituximab in participants with indolent lymphoma and diffuse large B-cell lymphoma (DLBCL) and to evaluate the efficacy of magrolimab in combination with R-GemOx in autologous stem cell transplant (ASCT) ineligible DLBCL participants.
NCT05004129
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.
NCT04732221
This is a two-part (Phase 2/Phase 3) study of frespaciguat, an inhaled soluble guanylate cyclase stimulator, in participants with pulmonary arterial hypertension (PAH). The first part (Phase 2) will assess three different doses of frespaciguat compared to placebo in a base period of 12 weeks, followed by comparison of three different doses of frespaciguat during an optional 40 month extension period. The treatment dose with the best efficacy and safety profile in the phase 2 cohort base period will be selected for use in the second part (Phase 3) of the study. The primary hypothesis of Phase 2 is that at least one frespaciguat dose is superior to placebo in reducing pulmonary vascular resistance (PVR) from baseline at week 12. The purpose of the second part (Phase 3) of the study is to confirm the efficacy, safety, and tolerability of frespaciguat at the selected dose compared to placebo during a 12 week base period followed by an extension period of up to 5 years. The primary hypothesis of Phase 3 is that frespaciguat is superior to placebo in increasing 6-minute walk distance (6MWD) from baseline at week 12. Due to sponsor's decision this phase/part was not conducted.
NCT03502707
The purpose of this study for: Cohort 1 and Cohort 2: to assess the safety and reactogenicity of the intramuscular one- and two-dose regimens, with a booster at Month 12 (Cohort 1) and to select a regimen for Cohort 3. Cohort 2 and part of Cohort 1: to assess respiratory syncytial virus (RSV) neutralizing antibody levels of the regimens containing RSV pre-fusion (preF) protein compared to the one-dose adenovirus serotype 26 respiratory syncytial virus pre-fusion (Ad26.RSV.preF) regimen. Cohort 3: to assess the safety and reactogenicity of the selected regimen and a booster at Month 12 and/or Month 24.
NCT02675959
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. The Funding Source is FDA OOPD.
NCT04215991
The primary objectives of this study are to assess the safety, tolerability, and pharmacokinetics (PK) of cefiderocol after single-dose administration in hospitalized pediatric participants 3 months to \< 12 years of age with suspected or confirmed aerobic Gram-negative bacterial infections and after multiple-dose administration in hospitalized pediatric participants 3 months to \< 18 years of age with suspected or confirmed complicated urinary tract infection (cUTI), hospital-acquired bacterial pneumonia (HABP), or ventilator-associated bacterial pneumonia (VABP).