You can narrow down the results using the filters
-
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Expand descriptionThis is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.
-
Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis
Expand descriptionThe primary objective of this study is to evaluate the safety and tolerability of tarperprumig in participants with newly diagnosed or relapsing anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
-
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Expand descriptionThe purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).
-
ABL103 in Combination With Pembrolizumab, With or Without Taxane in Advanced or Metastatic Solid Tumors
Expand descriptionThis study is to assess the safety and antitumor activity of ABL103 plus pembrolizumab, with or without taxane, in advanced or metastatic solid tumors.
-
Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN)
Expand descriptionThe primary objective of this study is to evaluate the efficacy of ALXN1920 compared with placebo in participants with PMN who are at a high risk for disease progression using 24-hour urine protein creatinine ratio (UPCR).
-
Assessing the Impact of Muvalaplin on Major Cardiovascular Events in Adults With Elevated Lipoprotein(a)
Expand descriptionThe purpose of this study is to evaluate the efficacy of muvalaplin in reducing cardiovascular risk in participants with high lipoprotein(a) who have cardiovascular disease or are at risk of a heart attack or stroke.
-
Early-stage Trial to Determine a Safe and Effective Dose for Ratutrelvir in Patients With Mild to Moderate COVID-19
Expand descriptionThis is an Early-stage Clinical Trial to Determine a Safe and Effective Dose for Ratutrelvir in Patients With Mild to Moderate COVID-19. It will also learn about the safety of drug Ratutrelvir. Participants will take a study drug as well as a standard therapy. A descriptive statistics will be used to present the study results.
-
Hearing Outcomes With the Osia 3 Sound Processor Compared to the Osia 2 Sound Processor in Adult Osia Implant Recipients
Expand descriptionThe goal of this interventional study is to evaluate the clinical performance and sound quality of the Osia 3 Sound Processor and to demonstrate its superiority compared with the Osia 2 Sound Processor in adults with mixed hearing loss, conductive hearing loss, or single-sided deafness. The main questions this study aims to answer are: * Do the findings confirm the clinical performance of the Osia 3 Sound Processor? * Is the Osia 3 Sound Processor superior compared to the Osia 2 Sound Processor? Participants will: * Undergo speech performance testing in both quiet and noisy environments * Provide ratings for a self-reported questionnaire
-
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ABS-1230 Given Orally Compared With Placebo in Healthy Participants Aged 18 to 55 Years
Expand descriptionThis first in human trial will evaluate the safety, tolerability, and pharmacokinetics of single ascending doses, multiple ascending doses, and fed and fasted doses of ABS-1230 given orally compared with placebo in adult healthy participants.
-
Study of IMC-P115C in Advanced PRAME-Positive Cancers
Expand descriptionPhase 1 First-in-human study of the safety and efficacy of IMC-P115C as a single agent and in combination with standard of care (SOC) agents in participants with advanced PRAME positive cancers. IMC-P115C is a half-life extended (HLE) ImmTAC targeting PRAME.