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A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subje…
STOP (Shared Decision Making to Treat Or Prevent) HIV in Criminal Legal Involved Populations
This study seeks to compare the effectiveness of two Patient Navigation models of care to evaluate the proportion who initiate PrEP/ART and substance use/subst…
A Phase 1b Multicenter, Non-randomized, Open-label Study to Investigate the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Pharmacodynamics of Single Ascending Doses of a New Subcutaneous Formulation of Ocrelizumab in Patients With Multiple Sclerosis
The main purpose of this study is to evaluate the safety and tolerability of the ocrelizumab subcutaneous (SC) test formulation in participants with multiple s…
Profiling of Radiological Factors in Treatment and Outcomes in Prostate Cancer
Background: Prostate cancer is one of the most common cancers for men in the U.S. There are some new ways to take pictures of the cancer. There are also new w…
A Study to Evaluate the Biological Effect of Tofersen in Adults With Amyotrophic Lateral Sclerosis Without Mutations in SOD1
The goal of this clinical trial is to evaluate whether tofersen is safe and effective in adults with non-SOD1 ALS. Tofersen is currently approved by the U.S. F…
An Open-Label Pilot Study of Esomeprazole in Children With Autism
Autism is a pervasive developmental disorder characterized by core deficits in social behavior and communication and the presence of repetitive/stereotyped beh…
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12…
Project ASTHMA - Aligning With Schools To Help Manage Asthma and Decrease Health Inequities
Children with asthma from communities that experience health inequities frequently do not receive guideline-based asthma care, and as a result, account for a d…
Assessment of Traumatic Brain Injury Using Transcranial Magnetic Stimulated Evoked Potentials (TMS-EEG)
Patients evaluated at Penn Presbyterian Medical Center for traumatic brain injury (TBI), who sign the informed consent, will undergo assessment of electrophysi…
The CATSINDO Trial - Clinical and Translational Study in Newly Diagnosed Osteosarcoma
The goal of this study is to learn whether children, adolescents, and young adults with newly diagnosed high-grade osteosarcoma can be safely discharged from t…
A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter, Clinical Trial With Extension Period to Evaluate the Efficacy and Safety of Xeomin® Injections for the Prevention of Chronic Migraine
In this clinical trial, participants with chronic migraine will receive injections with Xeomin or Placebo into muscles of the head and neck. The purpose is to …
The Phase III 'High Five Trial' Five Fraction Radiation For High-Risk Prostate Cancer
This phase III trial compares stereotactic body radiation therapy (SBRT), (five treatments over two weeks using a higher dose per treatment) to usual radiation…