Madison

Researchers are looking for new ways to treat neovascular age-related macular degeneration (NVAMD). Available standard (usual) treatments for NVAMD, such as aflibercept, may not work for every person. Researchers want to learn if a trial medicine called tiespectus (also called MK-8748 or EYE201) can treat NVAMD. The goal of this trial is to learn if tiespectus works as well as aflibercept to treat NVAMD.

Researchers are evaluating intravitreal EYE103 in participants with neovascular age-related macular degeneration (NVAMD) or macular edema following branch retinal vein occlusion (BRVO). This Phase 2, randomized, dose-masked study includes four patient cohorts: treatment-naive NVAMD participants, incomplete responder (IR) NVAMD participants as monotherapy, IR NVAMD participants receiving EYE103 combined with aflibercept 2.0 mg, and treatment-naive BRVO participants. The study aims to assess safety and efficacy of different doses of EYE103 in these conditions. Participants in each cohort will be randomly assigned to receive either a low or high dose of EYE103 via intravitreal injection. All participants will receive three injections spaced four weeks apart. IR NVAMD participants in the combination therapy cohort will also receive an injection of aflibercept 2.0 mg on Day 1. The timing of enrollment into each cohort is determined by the Sponsor. Participants will undergo safety and efficacy assessments at each injection visit, with some cohorts returning two weeks after injections for further evaluations. Assessments include measuring best-corrected visual acuity using the ETDRS chart, slit-lamp biomicroscopy, fundoscopy, and spectral domain optical coherence tomography (SD-OCT) to measure central subfield thickness. The study concludes at Week 12, which is the end-of-study visit for all participants.

Researchers are evaluating the effectiveness, safety, tolerability, and pharmacodynamics of multiple doses of APL-3007 combined with Syfovre/Pegcetacoplan (APL-2) in patients aged 60 years and older diagnosed with geographic atrophy secondary to age-related macular degeneration. This Phase 2, randomized, placebo-controlled, multicenter, masked study focuses on measuring changes in retinal pigment epithelium lesions using advanced artificial intelligence-based SD-OCT imaging. Participants will receive either the combination of APL-3007 with pegcetacoplan (APL-2) or a placebo. The study includes a treatment period with multiple doses administered, aiming to assess the impact on geographic atrophy lesions over a 12-month period. Syfovre injections at 6-8 week intervals prior to enrollment are part of the inclusion criteria. During the study, participants will undergo various eye imaging assessments such as OCT and FAF to monitor lesion size and progression. Researchers will evaluate changes in lesions at month 12 compared to baseline. Safety and tolerability will be closely monitored through laboratory tests, clinical evaluations, and vaccination status requirements. The study duration includes regular visits for treatment administration and monitoring over at least one year.

The trial investigates the safety, tolerability, effectiveness, and how the body processes and responds to osivelotor in people with sickle cell disease (SCD). This Phase 2/3, multicenter study involves adults and adolescents with SCD, aiming to find the best dose and compare osivelotor to a placebo. The study also includes a long-term extension to assess ongoing safety and blood-related effects. Osivelotor is given orally as tablets. The study has three parts: Part A focuses on determining a safe and effective dose in adults; Part B compares osivelotor to placebo in adults and adolescents over 48 weeks; and the Open Label Extension (OLE) offers continued treatment to those who completed Part B to monitor long-term safety and responses. Participants will be monitored throughout the study periods with assessments of safety, tolerability, and treatment effects. These include evaluations at 12 weeks (Part A), 48 weeks (Part B), and approximately 24 months after the last participant joins (OLE). Researchers will track blood values, side effects, and disease-related events to understand how the drug works and its impact on SCD over time.

Researchers are studying the effects of two experimental drugs, pozelimab and cemdisiran, in adults aged 50 to 85 who have Geographic Atrophy (GA) caused by Age-related Macular Degeneration (AMD), a condition that affects central vision. The study aims to compare how quickly GA progresses in patients treated with cemdisiran alone, a combination of pozelimab and cemdisiran, or a placebo. Additional goals include monitoring possible side effects, measuring drug levels in the blood, and checking for antibodies that might reduce drug effectiveness or cause side effects. Participants receive subcutaneous injections of either pozelimab combined with cemdisiran, cemdisiran alone, or a placebo. The study is randomized, double-masked, and placebo-controlled, conducted at multiple centers. Treatment schedules and dosing are managed as described in the protocol, with vaccinations for meningococcal and pneumococcal infections required prior to participation. Throughout the study, participants undergo regular clinic visits where eye imaging using Fundus Autofluorescence (FAF) tracks the progression of GA lesion area over 52 weeks. Researchers also monitor safety, side effects, and immune responses, ensuring adherence to study procedures. The main outcome measured is the growth rate of the GA lesion area over one year, helping to evaluate the potential benefits and risks of the study drugs.

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 Ixoberogene Soroparvovec (Ixo-vec) for treating neovascular (wet) age-related macular degeneration (nAMD) in adults aged 50 years and older. This Phase 3, multi-center, randomized, double-masked study compares a single intravitreal injection of Ixo-vec with an active comparator, Aflibercept. The study focuses on a broad population including both treatment-naïve and treatment-experienced participants, aiming to assess if Ixo-vec is not less effective than the comparator in improving vision. Participants will receive one intravitreal injection of either Ixo-vec or Aflibercept. Ixo-vec is a gene therapy designed to reduce the treatment burden by potentially decreasing the need for frequent injections that are common in current anti-VEGF treatments for nAMD. The study includes a screening period followed by treatment administration and ongoing safety and efficacy assessments. During the study, participants will undergo regular evaluations including measurements of best corrected visual acuity (BCVA) at baseline and at Weeks 52 and 56 to monitor changes in vision. Safety, tolerability, and treatment response will be closely observed throughout the study duration. Researchers will also monitor anatomical responses to therapy and overall eye health through clinical assessments and adherence to eye drop use as required by the protocol.

Researchers are studying how certain drugs given to children and young adults by their health care providers behave in their bodies. The goal is to find the safest and most effective doses for these younger patients. This research focuses on drugs that have not been extensively studied in children but are currently prescribed as part of standard care for various conditions including infections, heart issues, respiratory problems, and more. The study collects bodily fluid samples like whole blood and other effluents from children and young adults who are prescribed these drugs by their doctors. The drugs are given as part of regular treatment, not as part of the study itself. Data collection for pharmacokinetic (PK) measures such as clearance, volume of distribution, elimination rate, half-life, absorption rate, area under the curve, maximum concentration, and time to reach maximum concentration will occur for up to 90 days after consent, with up to 210 days for participants with Down Syndrome at designated sites. Participants will be involved through sample collection and monitoring over the study period. Researchers will assess how the drugs are processed in the body using blood and fluid samples. The study includes children from birth up to 20 years old and involves safety and drug behavior evaluations. This detailed monitoring helps understand the drugs’ effects and supports safer dosing guidelines in pediatric care.

Researchers are evaluating ABBV-RGX-314, a novel one-time gene therapy, for treating neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD causes vision loss due to abnormal blood vessel growth in the retina and affects millions in the United States, Europe, and Japan. Current treatments require frequent eye injections, which can be burdensome and may lead to reduced vision over time. This Phase 3 study aims to compare the effectiveness and safety of two doses of ABBV-RGX-314 against the standard anti-VEGF drug, aflibercept, in people with wet AMD. Participants will be randomly assigned to receive one of two doses of ABBV-RGX-314 gene therapy or aflibercept injections. The gene therapy involves a one-time subretinal injection delivering a gene that produces an anti-VEGF protein to help control abnormal blood vessels. In addition, a bilateral treatment substudy will examine safety and effectiveness when both eyes are treated in participants with wet AMD in both eyes. This substudy will enroll up to 15 participants for at least 50 weeks of follow-up. During the study, participants will have their vision measured regularly to assess changes in best-corrected visual acuity (BCVA). Safety will be monitored by recording any eye-related adverse events and serious side effects. Participants will be followed for up to 54 weeks or more to evaluate how well the gene therapy maintains or improves vision compared to aflibercept and to assess overall treatment safety and tolerability.

Researchers are studying the long-term safety and effectiveness of two investigational gene therapies, giroctocogene fitelparvovec for Hemophilia A and fidanacogene elaparvovec for Hemophilia B. This phase 3 study focuses on patients who have previously received these treatments in Pfizer-sponsored clinical trials. The goal is to understand how these treatments perform over an extended period. Participants who received a single dose of either gene therapy will be followed over time, with data collection and visits based on standard medical care. The study includes testing for AAV vector integration in liver tissue when clinically indicated through biopsy or surgery. These evaluations help monitor any changes related to the gene therapies. During the study, researchers will track safety and effectiveness by monitoring occurrences of blood clots, factor inhibitor development, liver cancers, liver abnormalities, and factor activity levels. Participants will be observed from day 1 up to 10 years after treatment, with monitoring adapted to routine care. This long-term follow-up aims to provide comprehensive information about the treatments' lasting effects.