Edison

Researchers are conducting a Phase 1/2 multi-center interventional study to evaluate the safety and effectiveness of BS01 in patients with geographic atrophy (GA) caused by dry age-related macular degeneration (AMD). The study aims to assess dose-related safety and efficacy by comparing different doses of BS01, a gene therapy, against a sham injection. Up to 40 patients aged 50 to 85 years with GA secondary to dry AMD are being enrolled across two parts: an initial dose-escalation phase followed by a dose-expansion phase. The study involves intravitreal administration of BS01, a recombinant adeno-associated virus vector delivering ChronosFP, to evaluate its impact on GA. Part 1 is an open-label dose-escalation phase with up to 10 patients to identify appropriate doses based on a benefit/risk assessment. Part 2 includes approximately 30 patients receiving the selected dose(s) from Part 1 or a sham procedure without needle injection to allow controlled comparison of safety and efficacy over 12 months. Participants will undergo regular assessments including best corrected visual acuity (BCVA), imaging of the retina with fundus photography and fundus autofluorescence, and monitoring for adverse events related to the study drug. Safety evaluations focus on dose-limiting toxicities and ocular or non-ocular adverse events during 12 months of follow-up. Efficacy is measured by changes in visual acuity from baseline. The study requires clear ocular media, adequate pupil dilation, and the ability to complete all imaging procedures and follow-up visits within the study duration.

Researchers are evaluating prizloncabtagene autoleucel (prizlo-cel), a dual targeting CAR T-cell therapy that attacks CD20 and CD19, in adults with relapsed or refractory B-cell non-Hodgkin lymphoma or frontline high-risk diffuse large B-cell lymphoma. This Phase 1b/2, multicenter, open-label study aims to assess the safety, tolerability, and overall response of this treatment in these patients. The study includes adult participants with confirmed CD19 and/or CD20 positive tumors who have received prior therapies and have measurable disease. Participants will receive an infusion of prizlo-cel, an autologous CAR T-cell therapy designed to target B-cell lymphoma. The study includes two phases: Phase 1b focuses on safety and tolerability, while Phase 2 evaluates the overall response as judged by an independent review committee. Treatment involves modifying the patient's own T cells to target cancer cells expressing CD19 and CD20. This open-label design means both researchers and participants know the treatment being given. During the study, participants will be monitored for adverse events and treatment response for up to two years after the infusion. Assessments include measuring tumor response using established criteria and ongoing safety evaluations. Researchers will track side effects and effectiveness to better understand the therapy's impact over time. Participation requires regular visits and evaluations to ensure safety and document outcomes throughout the study period.

Researchers are evaluating the use of cannabidiol oral solution (CBD-OS) as an additional treatment to reduce the frequency of focal-onset seizures in people aged 12 to 75 years. This study focuses on participants with focal epilepsy, aiming to assess the safety and effectiveness of CBD-OS in both early treatment and more resistant cases. The study also explores how the drug behaves in the body and examines factors that might predict or influence treatment response, including brain imaging and cognitive testing in a substudy. Participants will receive CBD-OS starting at a dose according to the approved local product label. This is an open-label, single-arm study, meaning all participants will be treated with CBD-OS and observed over time. The treatment period and dosing schedules are designed to evaluate the drug's impact on seizure frequency and health outcomes. During the study, participants will have their seizure frequency monitored and compared to their baseline seizure rates. Additional assessments include pharmacokinetic testing, safety evaluations, and neuropsychological and functional magnetic resonance imaging tests for those in the substudy. The primary outcome is the percent change in countable focal seizure frequency compared to baseline, with overall participation including screening and treatment phases.

This research aims to understand how avacincaptad pegol, a treatment approved in the US, is used for people with geographic atrophy caused by age-related macular degeneration (AMD). Geographic atrophy is an advanced stage of AMD where cells in the retina waste away, leading to worsening central vision and possible permanent vision loss. The study focuses on observing treatment patterns and safety in routine clinical practice rather than testing new effects. Participants in this study will receive avacincaptad pegol through intravitreal injections, which are injections into the eye. The study collects information from patients who have already been prescribed this treatment by their doctors. There is no experimental intervention from the study team, and treatment decisions are made by the patients' doctors. The study follows patients for up to 3 to 5 years, depending on when they join. While in the study, participants will have regular eye exams as part of their usual care. They will also complete surveys about their eye health at the start of treatment, every 6 months for the first 2 years, then annually afterward. Researchers will track treatment details like the number and dose of injections, treatment duration, reasons for stopping treatment, and patient characteristics. Safety and treatment patterns are monitored through medical records during and after treatment.

Researchers are evaluating the safety and effectiveness of apixaban compared with aspirin in patients who recently had an intracerebral hemorrhage (ICH) and also have atrial fibrillation (AF). The study aims to find out if apixaban is better than aspirin in preventing any type of stroke or death from any cause. It also looks at whether apixaban leads to better functional recovery measured by the modified Rankin Scale. This is a phase III, randomized, double-blinded trial enrolling 700 patients over 3.5 years. Participants will be randomly assigned to receive either apixaban, an oral blood thinner that inhibits Factor Xa, or aspirin, an oral antiplatelet medication. The study lasts from 12 months up to 36 months of follow-up after enrollment. Treatments are given orally, and patients will be monitored throughout the study period. Recruitment and coordination occur through NIH/NINDS StrokeNet sites. During the study, participants will undergo assessments including brain imaging (CT or MRI) to confirm diagnosis, functional outcome measurements using the modified Rankin Scale, and monitoring for any strokes or death. Safety will be closely observed, and patients will provide informed consent before joining. The primary outcome measured is stroke or death up to 3 years, and secondary outcomes include functional status changes. Participants are followed regularly to track these outcomes and overall health status.

Researchers are evaluating the safety and effectiveness of Cardiac Contractility Modulation (CCM) therapy in people with heart failure who have a left ventricular ejection fraction (LVEF) between 40% and 70%. This clinical trial is prospective, multi-center, randomized, quadruple-blind, and sham-controlled, aiming to assess CCM therapy delivered through the OPTIMIZER Smart Mini System. The study is divided into two parts: Part I focuses on safety and effectiveness based on functional capacity and health status, while Part II focuses on clinical outcome data. Participants will have the OPTIMIZER Smart Mini System implanted and then be randomized in a 2:1 ratio to either have CCM therapy turned ON or OFF for the first 18 months, during which the trial is blinded. CCM therapy is programmed to deliver seven one-hour treatment phases evenly spaced over 24 hours. After 18 months, participants initially assigned to the CCM OFF (sham) group will have the therapy turned ON. Subjects enrolled in Part I will continue to be followed through Part II to contribute data for safety and effectiveness evaluation. Throughout the study, participants will undergo various assessments including the 6-minute walk test and the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score to measure changes in functional capacity and health status at 6 months. Safety will be monitored by tracking device- or procedure-related complications up to 12 months. The study also measures a composite of mortality, morbidity, and health status outcomes over an 18-month period. Follow-up and monitoring continue throughout the study duration to capture these outcomes and ensure participant safety.

Researchers are investigating how the healthy human brain operates near a critical point between order and chaos, which is believed to allow maximum flexibility and information processing. This study focuses on understanding brain dynamics, particularly how they relate to cognitive control and subjective effort during demanding mental tasks. The research explores how deviations from this critical brain state may be biomarkers for various brain disorders and targets for therapeutic intervention. The study uses transcranial magnetic stimulation (TMS) with two types of theta-burst stimulation (continuous TBS and intermittent TBS) to modify the balance of excitation and inhibition in the brain's right frontal eye field. Participants will receive either active stimulation or sham stimulation in separate sessions. The effects of both stimulation types on brain dynamics and cognitive control will be compared, with the stimulation expected to alter brain activity for at least 60 minutes. Participants will undergo electroencephalography, MRI scans, and behavioral tests to measure brain flexibility and cognitive task performance before and after stimulation. Researchers will assess changes in brain activity, eye movement accuracy, and subjective cognitive effort. The study includes assessments immediately after stimulation and at various time points to monitor effects, aiming to understand the relationship between brain criticality and cognitive effort in healthy adults aged 18 to 45.

This research aims to evaluate the effectiveness of a cardiac rehabilitation program for adults who have experienced a stroke. It will assess whether adding cardiac rehabilitation to the standard care treatments improves recovery of function, reduces hospital readmissions, lowers the chance of another stroke, and decreases mortality rates. The study focuses on stroke survivors who meet specific health and functional criteria and are medically cleared for cardiac rehabilitation. Participants will take part in an outpatient cardiac rehabilitation program consisting of 36 sessions of progressive exercise lasting 30 to 50 minutes each. During these sessions, participants will be closely monitored with telemetry and supervised by cardiac rehabilitation nurses and exercise physiologists. The program also includes educational sessions on cardiovascular disease risk factors such as diet, smoking cessation, physical activity, medication adherence, and behavior change. Based on initial assessments, some participants may be referred to a rehabilitation psychologist or dietician. Alongside this program, patients will continue to receive their usual care therapies prescribed by their doctors. Throughout the study, participants will undergo assessments including a 6-Minute Walk Test to measure changes in physical function from 30 days to 120 days after their stroke. Researchers will monitor participants' recovery progress, hospital readmissions, stroke recurrence, and mortality. The study involves close supervision and safety monitoring during exercise sessions, with the total participation period covering several months post-stroke to evaluate the impact of the rehabilitation program.

Researchers are evaluating whether middle meningeal artery embolization (MMAE) can be used as a safer alternative to conventional open surgery for patients with moderately symptomatic chronic subdural hematoma (CSDH). This trial aims to determine if MMAE reduces the need for rescue surgery or deaths compared to standard surgery and to assess the safety of both treatments in these patients. Participants will receive either MMAE, which involves particle embolization of the middle meningeal artery using specialized embolization devices, or conventional surgery involving surgical drainage through burr holes or craniotomy. Eligible patients undergo the assigned treatment within 72 hours after randomization. The study compares these two groups to evaluate outcomes and safety. During the study, participants share their medical history, undergo physical exams, blood tests, and head CT scans, and complete questionnaires. Researchers will monitor for adverse events and measure the need for rescue surgery or death within 180 to 210 days after randomization. The total participation duration covers the time needed to assess these primary outcomes and safety.

Researchers are evaluating the effectiveness of three medications—atogepant, topiramate, and propranolol—for preventing migraines in adults. This Phase 4 clinical trial aims to find out if one medication works better or is easier to tolerate than the others. Participants must have a history of migraine with or without aura, chronic migraine, and meet specific headache frequency and duration criteria. Participants will first track their headaches daily for four weeks. If they remain eligible, they will be randomly assigned to take one of the three study drugs—atogepant (up to 60 mg daily), propranolol (up to 80 mg twice daily), or topiramate (up to 50 mg twice daily)—for twelve weeks. The study includes several scheduled research visits at the start, at four weeks for randomization, and then at weeks four, eight, twelve, twenty-four, and forty-eight after randomization. Throughout the study, participants will use a daily headache diary and attend research visits to provide data on their migraine patterns. The main outcome measured is the proportion of participants who experience at least a 50% reduction in moderate to severe headache days during weeks 9-12 after starting the medication compared to the four weeks before medication began. Researchers will monitor treatment adherence and safety during all visits and collect data for up to 48 weeks.