O'Fallon

Researchers are studying participants with Congenital Myasthenic Syndromes (CMS) caused by mutations in the DOK7, MUSK, AGRN, or LRP4 genes. The goal is to understand symptoms, quality of life, and disease activity in these patients over time. This natural history study gathers important information about how CMS affects participants and tracks changes in their health. Participants will attend up to four study visits during which clinical assessments are performed. These assessments focus on evaluating symptoms and overall quality of life related to CMS. No specific treatments or interventions are administered, as the study is observational and aims to collect detailed data about the condition's progression. Throughout the study, researchers collect data retrospectively and prospectively over a period of up to 12 months. This includes information on diagnosis, healthcare use, medications, and changes in health status related to CMS. The study carefully monitors participants to better characterize the natural course of the disease and its impact on daily living.

Researchers are monitoring the long-term safety of efgartigimod, a biological treatment, in patients with generalized myasthenia gravis (gMG). This study is a non-interventional, prospective, post-authorization safety study designed to observe patients who are either starting or already receiving efgartigimod, as well as those with gMG who have not been treated with it. The study aims to compare the occurrence of serious infections and other safety outcomes over a period of up to 10 years. Patients with gMG who are expected to begin efgartigimod treatment at enrollment or who are currently in their first treatment cycle will be included in the efgartigimod group. Those with gMG who are not starting efgartigimod and have never received it will be enrolled in a non-efgartigimod group. There are no assigned treatments or interventions from the researchers; the study simply observes patients as they receive routine care. Participants will be followed for up to 10 years to track safety outcomes, including serious infections. Data collection will involve routine clinical assessments and monitoring as per standard medical practice. The study observes the real-world use of efgartigimod and its safety profile in managing gMG, providing valuable long-term information without altering patients' treatment plans.

Researchers are evaluating the effectiveness and safety of combining inavolisib with a cyclin-dependent kinase 4 and 6 inhibitor (CDK4/6i) and letrozole compared to placebo plus CDK4/6i and letrozole. This study focuses on participants with endocrine-sensitive PIK3CA-mutated hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer. It aims to assess treatment outcomes in the first-line setting for this specific breast cancer type. Participants will be assigned to receive either oral inavolisib once daily or a matching oral placebo once daily. All participants will also receive a CDK4/6 inhibitor on either Days 1-21 or Days 1-28 of each 28-day cycle, along with daily oral letrozole. This randomized, double-blind study will compare these two treatment combinations to monitor differences in disease progression and safety. Throughout the study, researchers will evaluate progression-free survival from the time of randomization until disease progression or death, up to 7 years. Participants will undergo assessments including tumor measurements by RECIST criteria, performance status evaluations, and monitoring of blood and organ function before treatment begins. Safety and efficacy will be closely observed during treatment, aiming to provide detailed long-term data on the study therapies.

Researchers are evaluating the anti-tumor activity of amivantamab combined with pembrolizumab and carboplatin compared to pembrolizumab, 5-fluorouracil (5-FU), and platinum therapy (carboplatin or cisplatin) in participants with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). This trial focuses on participants who have not received prior systemic treatment in the recurrent/metastatic setting. HNSCC is a type of cancer affecting the outer tissue layer of the mouth and throat and other head and neck regions. Participants will receive either amivantamab added to pembrolizumab and carboplatin or the standard care regimen of pembrolizumab, 5-FU, and a platinum agent (carboplatin or cisplatin). 5-FU will be given as an infusion over a 4-day period. The study is a phase 3, randomized, open-label, multicenter trial comparing these treatment combinations. During the study, researchers will monitor overall survival and the objective response rate using standard tumor evaluation criteria for up to about 3 years and 7 months. Participants will undergo assessments to measure disease response, including imaging and other evaluations, to track how well the treatments work. Safety and side effects will also be monitored throughout the trial period.

Researchers are studying adults with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) to understand why they choose certain treatments, why some switch to HyQvia from other therapies, and how satisfied they are with these treatments. The study also aims to assess the impact of CIDP on work productivity and daily activities, the signs and symptoms of CIDP, and collect information about any medical problems or side effects during HyQvia treatment. Additionally, it evaluates how effective HyQvia is in routine clinical care and monitors healthcare needs like emergency or hospital visits. This is a non-interventional study where participants receive treatment as determined by their doctors or clinics. Participants include both new users planning to switch to HyQvia within 6 weeks and current users who have recently started HyQvia treatment. Data collection involves medical record reviews, interviews at the start and end of the study, and questionnaires completed by participants. The study does not assign any specific treatment but observes the use of HyQvia in real-world settings. During the study, participants complete questionnaires measuring their treatment preferences, satisfaction, quality of life, disability, and work productivity at multiple times up to 12 months. Researchers also gather clinical information and monitor any side effects or healthcare interventions. Participants must be able to communicate in their local language if they take part in optional interviews. The study tracks outcomes and experiences related to HyQvia use for up to one year or until participants stop treatment or the study.

This research aims to evaluate the safety and effectiveness of iza-bren, a bi-specific antibody-drug conjugate targeting EGFR and HER3 with a topoisomerase inhibitor, compared to the treatment of physician's choice (paclitaxel, nab-paclitaxel, carboplatin plus gemcitabine, or capecitabine). The study focuses on patients with previously untreated, locally advanced, recurrent inoperable, or metastatic triple-negative breast cancer (TNBC) or estrogen receptor (ER)-low, HER2-negative breast cancer who are not eligible for anti-PD(L)1 or endocrine therapies. The trial is conducted in two phases, phase 2 and phase 3, to thoroughly assess these treatments.

Researchers are evaluating the effectiveness of JNJ-79635322 in adults with relapsed or refractory multiple myeloma (RRMM), a type of cancer affecting plasma cells. Participants must have received at least three prior treatments including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody. This Phase 2 study focuses on patients whose disease has either returned after treatment or did not respond to previous therapies. JNJ-79635322 will be given as an injection under the skin. The study is open-label, meaning both researchers and participants know the treatment being given. The trial includes monitoring participants for response to treatment over a period of up to two years and nine months. During the study, participants will undergo regular assessments to measure disease progression and treatment response, including laboratory tests to confirm measurable disease. Researchers will track the overall response rate to the drug and monitor safety. Participants must have good performance status and will be followed closely throughout the treatment phase to evaluate how well the drug works and to watch for any side effects or complications.

Researchers are evaluating whether adding immunotherapy drugs brentuximab vedotin and nivolumab to standard chemotherapy, with or without radiation, can improve survival for patients aged 5 to 60 years with newly diagnosed stage I or II classical Hodgkin lymphoma. This phase III trial compares outcomes in groups based on their early response to initial chemotherapy, aiming to understand if immunotherapy can lead to better progression-free survival and overall survival compared to standard treatment alone. The study also looks at side effects, quality of life, and long-term health impacts across different patient groups. Participants first receive two cycles of standard ABVD chemotherapy every 28 days, followed by imaging to classify their response as rapid or slow early responders and their risk status as favorable or unfavorable. Based on these factors, patients are assigned to one of eight treatment arms that include either continued standard chemotherapy regimens or immunotherapy with brentuximab vedotin and nivolumab, sometimes combined with involved-site radiation therapy. Treatments are given intravenously or orally depending on the drugs, and cycles typically last 28 days. Imaging and blood samples are collected regularly throughout the study. Throughout the trial, participants undergo frequent scans such as FDG-PET, CT, MRI, and PET-CT to monitor their disease status. Blood samples and questionnaires assess treatment effects and quality of life. After completing treatment, patients have scheduled follow-up visits every 3 months for the first year, then every 6 months for two years, and annually up to 12 years to track long-term outcomes, side effects, and survival. The main measurements focus on progression-free survival, overall survival, treatment-related adverse events, and patient-reported experiences.

Researchers are evaluating two treatment combinations for patients with melanoma that has spread to the brain and has a specific BRAF-V600 mutation. This phase II trial compares encorafenib, binimetinib, and nivolumab against ipilimumab and nivolumab to determine which approach better controls and shrinks brain metastases from melanoma. The study also aims to assess overall survival, response rates, treatment duration, and side effects of each regimen. Participants are randomly assigned to one of two groups. One group receives encorafenib orally once daily, binimetinib orally twice daily, and nivolumab intravenously every 28 days. The other group receives nivolumab intravenously and ipilimumab intravenously during the first four cycles, with cycles every 21 days initially, then every 28 days thereafter. Treatment continues unless the disease worsens or side effects become unacceptable. After treatment ends, participants have follow-up visits every six months for two years, then yearly until three years after starting the study. During the trial, participants undergo brain MRIs to monitor tumor response using standardized criteria. Imaging, tumor tissue, spinal fluid, stool, and blood samples are collected for research. Safety and effectiveness are carefully assessed through scans, physical exams, lab tests, and side effect monitoring. Progression-free survival up to three years after randomization is the main outcome. Participants remain in the study for about three years with periodic evaluations to track their health and disease status.

Researchers are evaluating the effectiveness of remibrutinib compared to dupilumab in adults with moderate to severe chronic spontaneous urticaria (CSU) that is not adequately controlled by second generation H1-antihistamines (sgH1-AHs). This Phase 3b, multi-center, randomized, double-blind, double-dummy study is conducted in the US and focuses on early treatment effects at 4 weeks and earlier. The study includes a screening period of up to 4 weeks, followed by a 12-week core treatment period where about 400 participants are randomly assigned to receive either remibrutinib (25 mg twice daily by mouth) with a placebo injection or dupilumab (a 600 mg loading dose followed by 300 mg every 2 weeks by injection) with a placebo tablet. All participants continue their stable dose of sgH1-AH during this period, with the option to add rescue doses if needed, not exceeding four times the standard dose per day. After the core period, participants may join an optional open-label extension to receive remibrutinib for an additional 12 weeks if the drug is not commercially available. Participants will complete daily diaries and regular assessments to track urticaria symptoms and treatment effects. Researchers will measure changes in the Weekly Urticaria Activity Score (UAS7) from the start to Week 4. Safety follow-up will occur for 12 weeks after treatment ends, with phone calls and site visits as needed, continuing longer if participants join the extension. The total study duration includes screening, treatment, optional extension, and safety follow-up phases.