Skeletal Muscle Atrophy and Dysfunction in Human Cancer
Nonsmall Cell Lung CancerCancer and its treatment can have profound effects on skeletal muscle, the most well-recognized being atrophy, weakness and diminished oxidative capacity. These adaptations negatively impact quality of life, treatment decisions and survival. Despite these consequences, the factors promoting these adaptations remain poorly defined and understudied in human patients. To address this gap in knowledge, our goal in this study is to examine the role of muscle disuse as a regulator of muscle size and function in human cancer patients
Fractional Laser Drug Delivery of a Local Anesthetic
AgingFace; AtrophyThe purpose of this study is to assess the clinical efficacy of fractional CO2 laser-assisted delivery of a topically applied anesthetic to the perceived pain during filler injection.
Oral Postbiotics in Patients With Macular Atrophy
Geographic AtrophyAge-Related Macular DegenerationA pilot study to evaluate the safety and efficacy of oral therapy with Postbiotics in patients with geographic atrophy secondary to age-related macular degeneration, myopia, or angioid streaks.
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1...
Muscular AtrophySpinalOpen-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate Risdiplam (RO7034067) for 24-months at the dose selected in Part 1.
PRIMA US-Feasibility Study in Atrophic Dry AMD
Dry Age-related Macular DegenerationIn this early feasibility trial the safety and performance of the new retinal prothesis PRIMA is tested in five subjects suffering from atrophic dry age related macular degeneration.
An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects...
Geographic Atrophy Secondary to Age-related Macular DegenerationThis is a phase 3, open-label, multicenter, extension study to evaluate the long-term safety and efficacy of pegcetacoplan (APL-2) in subjects with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) who participated in Study APL2-103 (NCT03777332) or completed the treatment at Month 24 of either Study APL2-303 (Derby, NCT03525613) or Study APL2-304 (Oaks, NCT03525600).
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated...
Spinal Muscular AtrophySpinal Muscular Atrophy Type 39 moreThis Phase 3 trial (Study SRK-015-003) is being conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and are receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam), to confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Spinal Muscular Atrophy Type ISpinal Muscular Atrophy Type II2 moreThis is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy Associated With Age-related Macular...
Geographic AtrophyThe purpose of this study is to evaluate the safety and tolerability of intravitreal injection of ONL1204 Ophthalmic Solution in patients with geographic atrophy associated with AMD. GA associated with AMD is one of the world's leading causes of visual disability. It is a progressive disease with no approved therapy to slow or arrest the process of continual photoreceptor and retinal epithelial (RPE) cell loss. A safe and effective therapy for GA will have vast societal benefits. ONL1204 is being developed for this purpose. ONL1204 is a first-in-class inhibitor of fragment apoptosis stimulator receptor-mediated cell death in development for to reduce rates of vision in patients with GA associated with AMD. ONL1204 has demonstrated protection of multiple retinal cell types in several preclinical models of acute ocular injury and the protection of RPE in AMD models. ONL1204 Ophthalmic Solution is currently in a Phase 1 clinical study in patients with macula-off retinal detachment to evaluate safety and tolerability of a single-dose of ONL1204 Ophthalmic Solution. The study is ongoing and uses the same doses and route of administration as this Phase 1b study in patients with GA.
Immediate Implants With Customized Abutments
Edentulous Alveolar Ridge AtrophyTooth extraction is followed by marked alterations of the tissue volume. The resorption process of at least the surrounding mucosa can be altered by using immediate implant placement and additional features such as immediate provisional crown, platform switching and grafting the gap between implant and buccal bone plate. The support of the gingival margin with a provisional crown led to an increase of buccal gingival tissue. Teeth scheduled for extraction will be used to study the effect of a customized healing abutment. The following groups will be randomly assigned: Control: A commercially available healing abutment will be screwed on top of the implant and a collagen sponge will be used to protect the graft. Test: A customized PEEK abutment mimicking the subgingival emergence of the extracted tooth will be screw retained on top of the implant to seal the extraction site.