Royal Oak

Researchers are studying people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have previously completed treatment with the drug nerandomilast. The study aims to understand how well participants tolerate long-term treatment with nerandomilast and whether it helps improve lung function or delays worsening symptoms, hospitalizations, or death. This is a phase 3 open-label extension trial sponsored by Boehringer Ingelheim. Participants will take nerandomilast tablets daily for up to 1 year and 10 months. They may continue their usual treatments for pulmonary fibrosis alongside the study treatment. There is no placebo group, and all participants receive nerandomilast during this extension period. Throughout the study, participants will visit their doctors regularly for health check-ups and lung function tests. Researchers will closely monitor any side effects and changes in lung function, particularly forced vital capacity (FVC), over nearly two years. The study will track time until symptom worsening, hospital visits, or death to assess the treatment's long-term safety and effects.

Researchers are evaluating the safety and effectiveness of pulsed field ablation (PFA) therapy for treating persistent atrial fibrillation (PersAF) that does not respond to medication. This study compares two approaches: PFA targeting the pulmonary veins plus electrographic flow (EGF) identified extra-pulmonary vein sources, and PFA targeting the pulmonary veins plus the left atrial posterior wall. The goal is to see if the new method with EGF mapping is at least as effective as the standard approach in managing symptoms of PersAF. Participants will be randomly assigned to one of two groups. The control group will receive pulmonary vein isolation (PVI) plus posterior wall ablation (PWA) using PFA, with EGF mapping performed but not used for treatment decisions. The treatment group will receive PVI plus ablation of active sources identified by EGF mapping. The FARAPOINT catheter may be used for narrow posterior wall channels if found. All subjects will undergo mapping and ablation with devices including the FARAPULSE PFA system and Opal HDx mapping system. Ablation procedures and mapping will be performed according to protocol. During the study, participants will be monitored for safety up to 60 days and effectiveness up to 365 days after treatment. Follow-up will include assessments to track heart rhythm and symptoms, with the use of a LUX-Dx insertable cardiac monitor for continuous monitoring. Participants must be able to comply with follow-up visits and device usage instructions. The study aims to capture data on safety outcomes and measure how well the ablation controls atrial fibrillation over one year.

Researchers are evaluating the effects of pelacarsen (TQJ230), given as a monthly subcutaneous injection, on slowing the progression of calcific aortic valve stenosis in adults aged 50 to under 80 years with elevated Lipoprotein(a) levels. This phase 2, randomized, double-blind, placebo-controlled multicenter trial aims to assess the efficacy, safety, and tolerability of pelacarsen compared to a matching placebo. The study is sponsored by Novartis Pharmaceuticals and focuses on patients with mild to moderate calcific aortic valve stenosis who are optimally treated for cardiovascular risk factors. Participants receive either pelacarsen 80 mg or a placebo via a prefilled syringe injected under the skin once every month. The study includes two groups: one receiving the active drug and the other receiving a placebo, both administered in the same way. The trial will last up to 36 months, during which patients will be monitored for changes in aortic valve function and calcium levels. Throughout the study, participants will undergo evaluations including measurement of peak aortic jet velocity and aortic valve calcium scores at 36 months. Additional assessments include changes in Lipoprotein(a) levels at 12 months and monitoring of fibrocalcific thickening of the valve and clinical events over the course of the trial. Safety will be closely monitored to ensure participants' well-being during the entire study period.

Researchers are evaluating the safety and effectiveness of belzupacap sarotalocan (bel-sar) compared to a sham control in adults with primary indeterminate lesions or small choroidal melanoma. This phase 3 trial aims to establish whether bel-sar, administered via a specialized eye injection, can better manage these eye tumors. The study is carefully designed to keep participants and assessors unaware of the treatment given to ensure unbiased results. Participants receive either a high or low dose of bel-sar through a suprachoroidal microinjector followed by activation with an infrared laser, or they receive sham injections and sham laser treatments as a control. The treatment uses a special device to deliver the drug in the space around the eye, and the laser helps activate the drug. Three study groups are compared: high dose bel-sar with laser, low dose bel-sar with laser, and sham procedures. During the trial, participants will be monitored for up to 65 weeks to assess tumor progression and other outcomes. Researchers will evaluate the time it takes for tumors to progress and other combined measures related to the disease. Safety and treatment effects will be closely observed throughout the study period. The study started in December 2023 and is expected to end in August 2028, with participants involved in scheduled assessments and monitoring visits.

Researchers are evaluating corneal endothelial cells in people with neovascular age-related macular degeneration (nAMD) who are treated with the Port Delivery System (PDS) refilled every 24 weeks. This Phase IV study is open-label and multicenter, aiming to assess changes in corneal endothelial cell density and other eye health measures in participants receiving this treatment. Participants will have the PDS implant surgically placed in the study eye at the beginning of the study. The implant is filled with a ranibizumab solution before insertion and then refilled every 24 weeks. Supplemental ranibizumab injections may also be given if needed. The study monitors the participants during the treatment period, which lasts up to approximately one year. During the study, participants will undergo exams including specular microscopy to measure corneal endothelial cell density, visual acuity tests, and other eye health assessments at baseline, 24 weeks, and 48 weeks. Researchers will track any serious or special ocular adverse events, device effects, and other safety outcomes. Participants are followed closely to understand the effects of the PDS implant and treatment on eye structures over time.

Researchers are investigating the best way to combine chemotherapy and radiation therapy for patients aged 3 to 29 years with localized non-germinomatous germ cell tumors (NGGCT) in the brain. This phase II trial aims to optimize treatment based on how well the tumor responds to initial chemotherapy, with the goal of reducing spinal cord relapses and adjusting therapy for better disease control. The study also compares different radiation types and examines cognitive and physical effects in children and young adults with NGGCT. Participants first receive induction chemotherapy consisting of carboplatin, etoposide, and ifosfamide over six cycles every 21 days. Based on tumor response, patients are assigned to one of two plans: Plan A involves whole ventricular plus spinal canal irradiation (WVSCI), delivered daily for 6 weeks, while Plan B includes high-dose chemotherapy with stem cell transplant followed by radiation therapy to the whole brain and spine. Some patients may undergo second-look surgery depending on tumor response before continuing treatment. Throughout the study, participants undergo MRI scans, collection of cerebrospinal fluid and blood samples, and questionnaires assessing cognitive, social, and behavioral functioning. Researchers monitor tumor response, progression-free survival, overall survival, and patterns of disease recurrence for up to 10 years. Safety and side effects are also evaluated to better understand long-term outcomes of these treatment approaches.

Researchers are evaluating the safety and effectiveness of the BWI OMNYPULSE™ pulsed field ablation (PFA) system for treating people with symptomatic paroxysmal atrial fibrillation (PAF), a condition causing irregular heart rates and abnormal blood flow. This study aims to assess the safety and 12-month effectiveness of this treatment approach in managing PAF symptoms. Participants will receive a pulsed field ablation procedure using the OMNYPULSE™ catheter combined with the TRUPULSE™ generator to isolate pulmonary veins and deliver pulsed field energy to treat atrial fibrillation. After the procedure, participants will be followed for 12 months to monitor outcomes related to the treatment. During the study, participants will undergo assessments to measure adverse events within 7 days after the procedure and track freedom from atrial tachyarrhythmia episodes between day 91 and day 365. Researchers will also evaluate changes in quality of life related to atrial fibrillation from the start of the study through 12 months. Participants will be monitored throughout the follow-up period to gather safety and effectiveness data.

Researchers are evaluating camizestrant, an oral selective estrogen receptor degrader, compared to standard endocrine therapy in patients with early-stage ER-positive, HER2-negative breast cancer. This Phase III open-label study focuses on individuals at intermediate or high risk for disease recurrence who have completed locoregional therapy and at least 2 years, up to 5 years, of standard adjuvant endocrine therapy. The goal is to assess if camizestrant improves invasive breast cancer-free survival and other related outcomes. Participants are randomly assigned to receive either camizestrant or continue with standard endocrine therapy chosen by their investigator, which may include aromatase inhibitors such as exemestane, letrozole, anastrozole, or tamoxifen. Treatment duration for both groups is planned for 60 months (5 years). The study allows prior use of CDK4/6 inhibitors and excludes patients with specific medical conditions or prior use of similar investigational agents. During the study, patients will be regularly monitored for invasive breast cancer-free survival, invasive disease-free survival, distant relapse-free survival, overall survival, and safety measures, including adverse events and changes in laboratory and vital signs. Quality of life assessments related to symptoms like arthralgia, hot flushes, and vaginal dryness will also be conducted. Follow-up for participants will continue for up to 10 years from the last patient's randomization.

Researchers are evaluating how well combination chemotherapy works in treating patients with newly diagnosed stages 2 to 4 diffuse anaplastic Wilms tumor (DAWT) and patients with relapsed favorable histology Wilms tumor (FHWT). This phase II trial compares the effects of two chemotherapy regimens, UH-3 and ICE/Cyclo/Topo, on event-free survival and overall survival, aiming to improve outcomes based on different relapse risk groups and prior treatments. The study also explores kidney toxicity, genetic markers, surgery impacts, and radiation therapy techniques to reduce side effects and better understand tumor behavior. Participants are assigned to one of two treatment groups. In Arm I (Regimen UH-3), patients receive cycles of vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan intravenously over various days in a 21-day cycle, with radiation therapy at week 7 of cycle 3 if needed. In Arm II (Regimen ICE/Cyclo/Topo), patients receive cycles of carboplatin, etoposide, ifosfamide, cyclophosphamide, and topotecan intravenously over 10 cycles every 21 days, with surgery and/or radiation therapy during certain cycles as clinically indicated. Throughout the trial, patients undergo multiple imaging tests including CT scans, PET scans, chest x-rays, MRIs, abdominal ultrasounds, and bone scans, along with blood sample collections and biopsies. After completing treatment, follow-up visits occur every 3 months for the first 2 years, then every 6 months for years 3 and 4, and once at year 5. The main outcomes measured are event-free survival and overall survival up to 5 years from study entry, with ongoing monitoring for treatment effects and safety.

Researchers are evaluating the effects of selumetinib compared to the standard treatment with carboplatin and vincristine for patients with newly diagnosed or previously untreated low-grade glioma (LGG) that lacks the BRAFV600E mutation and is not linked to systemic neurofibromatosis type 1. This phase III trial aims to determine if selumetinib is as effective as the standard chemotherapy regimen and to assess which treatment may be better, including their impact on patients' quality of life. Participants are randomly assigned to one of two groups. In the first group, patients receive vincristine sulfate intravenously on specific days during an induction phase, combined with carboplatin given intravenously over 60 minutes on certain days, followed by a maintenance phase with repeated cycles of these drugs. In the second group, patients take selumetinib sulfate orally twice daily in 28-day cycles for up to 27 cycles, with echocardiography performed at the start of treatment. Blood samples and MRI scans are collected throughout the study for monitoring. During the study, patients undergo regular assessments including MRI scans to monitor tumor status, blood tests, and quality of life evaluations. Follow-up visits occur every three months during the first year, every six months for years two and three, and annually for up to ten years after treatment completion. The main outcome measured is event-free survival up to ten years following randomization, along with secondary outcomes like tumor response, visual acuity, motor function, cognitive and behavioral functioning, and overall survival. Safety and treatment effects are closely monitored throughout the trial.