Waterford

Age-related macular degeneration AMD is a common condition affecting many older adults, leading to a loss of light-sensitive cells in the retina called Geographic Atrophy GA. GA starts as a small spot away from the central vision area fovea and grows over time, causing severe vision loss when it reaches the fovea. This study is investigating whether GAL-101 eye drops can slow the growth of GA and help preserve vision in affected patients. Participants in this study will use eye drops containing GAL-101 or placebo daily. The treatment involves applying two drops of the assigned solution at 5-minute intervals each day. The study eye will be chosen based on vision and retinal health criteria, and only that eye will receive the eye drops. The treatment period lasts between 12 and 24 months, with visits scheduled at screening, baseline, 2 weeks by phone, and clinic visits at 1, 3, 6, 9, and 12 months, then every 3 months thereafter until the last patient completes 12 months of treatment. Participants will undergo several assessments including imaging and vision tests to track GA lesion size and photoreceptor degeneration. The study will measure how well GAL-101 slows GA growth over 48 to 96 weeks. Safety and efficacy will be monitored throughout the treatment period. The total participation time varies but includes regular clinic visits and phone contacts for up to two years or more depending on individual treatment duration.

Researchers are evaluating treatments for newly diagnosed multiple myeloma in patients who cannot undergo autologous stem cell transplantation. This Phase 3 study compares two drug combinations belantamab mafodotin with lenalidomide and dexamethasone BRd versus daratumumab with lenalidomide and dexamethasone DRd. The goal is to see if BRd extends progression-free survival and improves minimal residual disease negative status compared to DRd. Participants receive either BRd or DRd treatment, continuing until disease progression, death, unacceptable side effects, withdrawal, or study end. Both treatment arms involve the administration of lenalidomide and dexamethasone alongside either belantamab mafodotin or daratumumab. Treatment duration may last up to approximately seven years. During the study, participants will undergo regular assessments including monitoring disease progression, response to treatment, and side effects. Measurements include progression-free survival, overall survival, and the number achieving minimal residual disease negative status. Quality of life questionnaires and blood tests will also be conducted. Safety monitoring includes eye exams and tracking adverse events throughout the study duration.

Researchers are evaluating whether combining tucatinib with trastuzumab and mFOLFOX6 works better than standard treatments for people with HER2 positive colorectal cancer that has spread or cannot be removed by surgery. This Phase 3 study also aims to learn about the side effects that may occur when taking this combination of drugs. Participants have metastatic or unresectable colorectal cancer and are randomly assigned to different treatment groups. Participants are randomly placed in one of two study groups. One group receives tucatinib taken orally twice daily along with trastuzumab given intravenously every 3 weeks and mFOLFOX6 chemotherapy every 2 weeks. The other group receives standard care, which may be mFOLFOX6 alone or combined with bevacizumab or cetuximab, both given intravenously on different schedules. Tissue samples and biopsies are collected before treatment to confirm HER2 positivity and other markers. During the study, participants will have regular evaluations including imaging scans to measure cancer progression, blood tests, and assessments of side effects and quality of life. Progression-free survival is the primary outcome measured for up to about 3 years, with other outcomes like overall survival and response rate also tracked. Safety monitoring continues for about one year after the last treatment. The study lasts several years, with ongoing follow-up to understand long-term effects and benefits.

Hidradenitis suppurativa HS is a painful inflammatory skin condition affecting areas like the underarms, groin, and genital regions. This trial evaluates the safety and effectiveness of upadacitinib, an oral drug approved for other inflammatory diseases, in adults and adolescents with moderate to severe HS who have not responded well or cannot tolerate anti-TNF therapies. The study is double-blinded and involves multiple treatment periods to assess disease activity and side effects. Participants will take oral tablets of either upadacitinib or a placebo once daily during the first two periods, each lasting 36 weeks. In Period 1, participants are randomly assigned to receive either upadacitinib or placebo. Period 2 assigns participants to one of six groups based on their response in Period 1, with treatment continuing for 20 weeks. In Period 3, eligible participants continue their assigned treatment for an additional 68 weeks, followed by a 30-day follow-up. Throughout the study, participants will attend regular outpatient visits where medical assessments will monitor treatment effects and side effects. Questionnaires and clinical evaluations will be completed to measure changes in disease activity and quality of life. The trial aims to track the percentage of participants achieving clinical response and the occurrence of adverse events over the entire study duration, which may be longer than standard care treatments.

This research aims to evaluate the effectiveness of AZD5335 compared to standard treatments in women with high-grade, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer. The study focuses on whether AZD5335 improves progression-free survival PFS among participants with either high or low levels of folate receptor alpha FR. Approximately 1100 adult women will be enrolled and divided into two groups based on their FR levels. Participants will be randomized within each group to receive either AZD5335 or the relevant standard treatment. Those with high FR levels will receive AZD5335 or mirvetuximab soravtansine, while those with low FR levels will receive AZD5335 or a chemotherapy agent chosen by the investigator, including paclitaxel, pegylated liposomal doxorubicin, or topotecan. Treatments are given intravenously, and participants will continue treatment until disease progression or other reasons for stopping. During the study, participants will undergo regular tumor assessments to monitor disease status. Researchers will follow all participants over time to evaluate overall survival. Safety and efficacy data will be reviewed periodically by an independent committee. The primary outcome is progression-free survival, measured for up to about five years. The study completion is expected by late 2029.

Researchers are investigating the proteomic and molecular features of primary and recurrent or metastatic breast tumors. The study particularly focuses on the expression of S100 protein and the estrogen receptor ER, progesterone receptor PR, and HER2 genes. The aim is to deepen understanding of the molecular pathways that drive breast cancer progression and how these pathways respond to different treatments. The research also seeks to link proteomic results with survival data and to find potential blood markers that could indicate breast cancer progression. Participants will undergo standard treatment while tissue and blood samples are collected at various times. Tissue samples, both fresh frozen and formalin-fixed paraffin-embedded, will be taken during surgery or biopsy of primary or recurrentmetastatic tumors. Blood samples will be collected before neoadjuvant treatment if applicable, and before and after surgery or biopsy. For recurrent or metastatic cases without surgery, blood will be drawn before starting treatment. Additional blood samples will be taken annually for up to five years in primary cases and up to two years in recurrent or metastatic cases. Throughout the study, clinical data will be gathered alongside the biological samples to support the research. The primary outcome measure is the investigation of proteins and their pathways in primary breast cancer over ten years. Secondary outcomes include comparing molecular profiles between primary and recurrentmetastatic tumors, identifying new molecular mechanisms of recurrence, and finding potential targets for treatment. Participants will be followed and evaluated over the long term to support these research goals.

Researchers are evaluating elacestrant compared to standard endocrine therapy in patients with estrogen receptor-positive ER and human epidermal growth factor receptor 2-negative HER2- breast cancer who have a relapse detected by circulating tumor DNA ctDNA. This international, multi-center, randomized, open-label phase III trial focuses on patients without distant metastasis who show ctDNA positivity during screening. The study aims to assess whether elacestrant can improve outcomes over the current standard endocrine treatments. The study consists of two phases. First, during the ctDNA screening phase, patients on standard adjuvant endocrine therapy will have plasma samples collected every six months for about 5.7 years to detect ctDNA. Patients who test positive will undergo imaging to confirm no distant metastasis and then be randomized 11 to either continue their current endocrine therapy or receive elacestrant 400 mg orally once daily. Treatment duration depends on prior endocrine therapy length, lasting between 2 to 6 years. Intensive follow-up with ctDNA testing and imaging occurs for up to 3 years after randomization. Participants will be monitored closely with blood tests for ctDNA at weeks 4, 16, and every 16 weeks thereafter, along with yearly mammograms, bone scans, and CT scans every 16 weeks to detect metastases or recurrences. Safety, quality of life, and overall survival are assessed throughout, with follow-up continuing until three years after the last patient enrolls. The primary outcome measured is distant metastasis-free survival at 6.25 years after the first randomization.

Researchers are evaluating the effects of negative pressure wound therapy NPWT compared to standard open wound care after surgical excision in patients with chronic pilonidal sinus disease PSD. PSD is a chronic inflammatory skin condition in the natal cleft area, often causing infection, abscess, discharge, and pain. The condition affects many, especially young adult males, and has a significant impact on quality of life and healthcare due to frequent recurrence and readmission after surgery. This multicenter randomized controlled trial aims to find the best wound care method to improve healing and patient outcomes. Participants will undergo surgical excision of chronic PSD and be randomly assigned to one of two groups. One group will receive negative pressure wound therapy dressings applied to the wound post-surgery, while the other group will have their wound dressed with standard sterile gauze after the skin is closed. The study will compare these two approaches for up to six months post-surgery, monitoring healing times and other related outcomes. During the study, participants will be followed for six months after surgery. Researchers will assess time to complete wound healing, postoperative pain, complications, quality of life using questionnaires like the EQ-5D-5L, scar appearance using the Patient and Observer Scar Assessment Scale POSAS, costs to patients, and wound recurrence rates. These assessments will occur at various points including the day of surgery, and one, three, and six months afterward, helping to understand the impact of each wound care method on recovery and patient experience.

Researchers are conducting the EXTUBE study, an international and multicenter observational research project, to understand the occurrence, risk factors, and outcomes of complications related to extubation after general anesthesia or critical illness. Extubation is a common but potentially risky procedure performed worldwide, with serious complications such as decreased oxygen delivery, cardiac arrest, brain damage, or death occurring in some cases. This study aims to fill gaps in knowledge due to limited and inconsistent past data, addressing the frequency and nature of these complications and adherence to best practices. The study observes adult patients undergoing extubation of an endotracheal tube in various settings including operating rooms, out-of-OR anesthesia locations, and intensive care units. It collects detailed data during the immediate period following extubation, focusing on complications occurring within 60 minutes and up to seven days after the procedure. No interventions or treatments are assigned instead, clinical practices and outcomes are monitored to identify risk factors and guideline adherence. Participants will be followed closely around the time of their extubation, with data collected on immediate and short-term complications such as severe hypoxemia, cardiac arrest, airway management needs, and reintubation. Researchers will evaluate complications, including airway injuries, cardiovascular events, and mortality until hospital discharge. The study involves careful documentation of clinical events and patient outcomes without altering standard care. Participants involvement duration will vary depending on their hospital stay and timing of extubation-related events.

Researchers are evaluating the efficacy and safety of Imeroprubart in adults with active Chronic Inflammatory Demyelinating Polyneuropathy CIDP, a condition affecting the peripheral nerves. This Phase 2b, multi-center, randomized, double-blind, placebo-controlled study aims to understand how well Imeroprubart works compared to placebo in treating CIDP. The study is sponsored by Immunovant Sciences GmbH and focuses specifically on adults meeting diagnostic criteria for typical or variant forms of CIDP. Participants will receive either Imeroprubart or a matching placebo by subcutaneous injection once weekly. The treatment period includes an initial 24-week phase Period 1 with Imeroprubart or placebo, followed by an extension to 52 weeks Period 2 for continued evaluation. Imeroprubart dosing is given once weekly via subcutaneous injection. Placebo is provided similarly during the first 24 weeks. During the study, participants will be monitored through clinical assessments including relapse status by Week 24, as well as measurements of disability, grip strength, muscle strength, and symptom scores. Electrodiagnostic tests support diagnosis at baseline. Safety and efficacy will be closely observed during treatment, with follow-up visits scheduled to assess outcomes. The total participation duration covers at least 24 weeks for the primary outcome assessment, with ongoing monitoring as defined by the study protocol.