Edison

Researchers are studying BS01, a gene therapy delivered by intravitreal injection, in people aged 50 to 85 with geographic atrophy GA caused by dry age-related macular degeneration AMD. This Phase 12 multi-center trial aims to assess the safety and effectiveness of BS01. The study includes an initial dose-escalation phase followed by a dose-expansion phase to compare the selected dose against a sham injection control group. The trial has two parts Part 1 is an open-label dose-escalation study involving up to 10 patients to determine the best dose based on benefit and risk. Part 2 will involve up to 30 patients randomized to receive either the selected doses of BS01 or a sham injection without needle, allowing controlled comparison of safety and efficacy. BS01 is a recombinant adeno-associated virus vector expressing ChronosFP. Participants will undergo eye exams and imaging to monitor geographic atrophy area and visual acuity. The study will evaluate adverse events for safety over 12 months in Phase 1 and measure changes in best corrected visual acuity BCVA over 12 months in Phase 2. Assessments include fundus photography, fundus autofluorescence, and optical coherence tomography. Participants will be monitored for treatment side effects and disease progression throughout the study period.

Researchers are investigating the effects of APL-3007 combined with SyfovrePegcetacoplan APL-2 in patients with geographic atrophy caused by age-related macular degeneration AMD. This Phase 2 randomized, placebo-controlled study aims to assess the efficacy, safety, tolerability, and pharmacodynamics of these treatments in this eye condition. The study involves multiple centers and uses a masked design to ensure unbiased results. Participants will be assigned to one of three groups two receiving different doses or frequencies of APL-3007 in combination with pegcetacoplan APL-2, and one receiving a placebo along with pegcetacoplan APL-2. The study will evaluate the treatments given as multidose regimens. The treatments focus on complement C3 inhibition to potentially impact disease progression. Throughout the study, participants will undergo assessments including artificial intelligence-based imaging to measure retinal pigment epithelium lesion area and photoreceptor degeneration, safety evaluations through adverse event reporting and visual acuity tests, and blood tests to assess serum markers. These evaluations occur over 12 months to monitor changes from baseline. Participants involvement includes regular visits for these assessments, with the study tracking treatment effects and safety over the duration.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and early antitumor effects of IDP-001 in adults with advanced or metastatic squamous and non-squamous non-small cell lung cancer and other squamous cell solid tumors such as head and neck, esophageal, cervical, and cutaneous cancers. This is a Phase 12 open-label study focused on these advanced cancers where standard treatments are limited or no longer effective. Participants will receive IDP-001 as an intravenous infusion. The study includes two parts Phase 1 Part 1 involves dose escalation to determine safety and tolerability over 3-week cycles, while Phase 1 Part 2 focuses on indication selection, evaluating response rates and duration over approximately 6 months. The study is non-randomized and open-label, with all participants receiving the investigational drug. During the study, participants will undergo tumor biopsies and regular assessments including imaging to measure tumor response using RECIST criteria. Safety monitoring includes tracking adverse events and dose modifications. Pharmacokinetic measurements and antibody levels against the drug will be collected. The study duration varies but includes up to 6 months of treatment evaluation and longer-term follow-up for survival outcomes up to 18 months.

Researchers are evaluating JNJ-95804306 for people with relapsed or refractory hematological malignancies, which are cancers starting in blood-forming tissues or immune system cells. The study includes two parts Part 1 focuses on finding a safe and tolerable dose of JNJ-95804306, while Part 2 further assesses its safety and anti-tumor activity alone or combined with standard care treatments. This phase 1 trial aims to identify suitable dose regimens and understand how the drug works in different blood cancers like AML, MDS, CLL, SLL, and NHL. Participants receive JNJ-95804306 orally. In Part 1, dose escalation is used to determine the recommended phase 2 dose RP2D. Part 2 involves dose expansion, where participants receive JNJ-95804306 alone or with standard of care SoC therapies at the identified doses, depending on their specific cancer type. For U.S. sites, only JNJ-95804306 monotherapy is given without SoC. The study includes different arms based on cancer type and treatment combination, monitoring various regimens and their effects. During the trial, participants undergo assessments for safety, including monitoring for dose-limiting toxicities up to 28 days after the first full dose and tracking adverse events for over six years. Researchers evaluate anti-tumor responses using specific criteria for each cancer type, including complete and overall responses. Blood samples are taken to measure drug concentrations and pharmacokinetics. Participants health status is regularly checked, and safety is closely monitored throughout the study, which may last several years.

Non-small cell lung cancer NSCLC is the most common lung cancer type, and this study focuses on people with NSCLC whose tumors have a specific faulty KRAS G12D gene mutation. Researchers are comparing a new drug called setidegrasib to the standard treatment docetaxel in adults with advanced or metastatic NSCLC who have already received chemotherapy and immunotherapy. The goal is to see if setidegrasib can help people live longer without their cancer worsening and to evaluate other effects on symptoms, tumor response, and safety. Participants in the study will be randomly assigned to receive either setidegrasib or docetaxel, both given through intravenous infusions. Setidegrasib is given on days 1, 8, and 15 of each 21-day cycle, while docetaxel is given on day 1 of each 21-day cycle. Treatment continues until the cancer progresses, side effects prevent continuation, or other stopping reasons occur. Some participants initially on docetaxel may switch to setidegrasib if their cancer worsens. Safety checks and monitoring occur regularly throughout the study. During the study, participants will have tumor scans every 6 weeks for the first year, then every 9 weeks until their cancer progresses. After progression, clinical staff will follow up by phone every 12 weeks. Researchers will assess how long participants live without cancer progression and overall survival, along with symptom changes, tumor response, quality of life, and side effects. The study may last up to several years, with ongoing evaluation of participant health and treatment effects.

Researchers are evaluating cannabidiol oral solution CBD-OS in participants aged 12 to 75 years who have focal-onset seizures FOS. This study aims to assess the effectiveness and safety of CBD-OS as an additional treatment to reduce seizure frequency compared to baseline. It also explores health outcomes in early line and refractory participants, along with pharmacokinetics, safety, and potential predictors of treatment response using functional magnetic resonance imaging fMRI and neuropsychological testing in a substudy. Participants will receive open-label CBD-OS starting with doses based on the approved local product label. The study is a single-group, open-label trial where all participants are treated with CBD-OS. It involves continuous administration of the drug with monitoring of effects and safety across the study period. During the study, participants will undergo evaluations including seizure frequency monitoring, health outcome assessments, pharmacokinetic sampling, fMRI scans, and neuropsychological testing for those in the substudy. Researchers will measure the percent change in countable focal seizure frequency compared to baseline, tracked for up to 16 weeks. Safety and treatment response predictors will also be monitored throughout the trial, which lasts until November 2027.

Researchers are observing how avacincaptad pegol is used in routine clinical practice for people with geographic atrophy caused by age-related macular degeneration AMD. Geographic atrophy is an advanced form of AMD where retinal cells waste away, leading to worsening central vision and potential permanent vision loss. This observational study collects information on treatment and medical events related to avacincaptad pegol without influencing the doctors treatment decisions. Participants in this study have chosen to begin treatment with avacincaptad pegol, delivered as intravitreal injections into the eye. The study records how often and how long the treatment is given, along with reasons for stopping treatment. It monitors participants receiving this treatment in one or both eyes as part of their regular medical care, without additional interventions from the study. During the study, participants will have eye exams as part of their usual care and will complete surveys about their eye health at the start of treatment, every six months for two years, and then yearly. Researchers will gather data from medical records to track treatment usage, vision changes, and any adverse events for up to about five years. This long-term observation helps understand real-world treatment patterns and safety.

Researchers are evaluating the use of apixaban compared to aspirin to prevent stroke or death in patients who have had a recent intracerebral hemorrhage ICH and also have atrial fibrillation AF. This phase III randomized, double-blinded trial aims to determine if apixaban is superior in preventing any type of stroke or death, as well as if it leads to better functional recovery measured by the modified Rankin Scale. The study will enroll 700 patients and follow them for 12 to 36 months to assess these outcomes. Participants will be randomly assigned to receive either apixaban or aspirin. Apixaban dosing is typically 5 mg twice daily, with a reduced dose of 2.5 mg twice daily for those meeting specific criteria such as older age, lower body weight, or certain medication use. Aspirin is given once daily at a dose of 81 mg. The study includes a treatment period after recent ICH, with careful monitoring for safety and efficacy. During the study, participants will undergo regular assessments including evaluation of stroke occurrence, death, and changes in functional status using the modified Rankin Scale. Safety and adherence will be monitored throughout the follow-up period, which ranges from 12 months up to 3 years. The research team will collect data to understand the benefits and risks of apixaban versus aspirin in this patient population.

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 designed as a multi-center, randomized, quadruple-blind, sham-controlled study to compare CCM therapy delivered through the OPTIMIZER Smart Mini System against a sham control. The trial includes two parts the first focuses on functional capacity and health status, while the second examines clinical outcomes. Participants will have the OPTIMIZER Smart Mini System implanted and be randomly assigned in a 21 ratio to either receive active CCM therapy CCM ON or sham therapy CCM OFF for the first 18 months. CCM therapy involves delivering seven one-hour treatment phases spread evenly over each 24-hour period. After 18 months, those initially in the sham group will have CCM therapy activated. Part I of the trial enrolls 450 participants, who will be followed through Part II, which includes up to an additional 1,050 participants. During the study, participants will undergo screening and baseline evaluations before device implantation. Researchers will assess changes in walking distance using a 6-minute walk test and health status via the Kansas City Cardiomyopathy Questionnaire at 6 months. Safety will be monitored by tracking device- or procedure-related complications over 12 months. The study will also evaluate a composite of mortality, morbidity, and health status outcomes up to 18 months. The total duration of participant involvement spans up to 18 months with ongoing follow-up and monitoring.

Researchers are studying how the brain operates near a critical point between order and chaos, which may help with flexibility, memory, and cognitive control. This study focuses on healthy young adults performing challenging cognitive tasks and how brain activity changes in response to transcranial magnetic stimulation. The goal is to understand how different types of theta-burst stimulation affect brain dynamics and cognitive effort. Participants will receive three types of stimulation in separate sessions continuous theta-burst stimulation cTBS, intermittent theta-burst stimulation iTBS, and sham placebo stimulation. Each session targets the right frontal eye field using a transcranial magnetic stimulation device. The order of these sessions is randomized and blinded to both participants and researchers until after data collection. During the study, brain activity will be recorded using EEG and MRI before and after stimulation, along with tests of eye movement accuracy and cognitive effort ratings. Researchers will measure changes in brain criticality, excitationinhibition balance, and task performance immediately and up to 72 minutes after stimulation. The study includes a screening visit and multiple sessions, with participant involvement lasting several hours across visits.