Atrophy Clinical Trials in Philadelphia, Pennsylvania
13 recruitingPhiladelphia, Pennsylvania
Showing 1–13 of 13 trials
Recruiting
Phase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
Muscular Atrophy, Spinal
Hoffmann-La Roche28 enrolled19 locationsNCT05861999
Recruiting
Phase 2
A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular Degeneratio
Geographic Atrophy secondary to Age Related Macular Degeneration
Apellis Pharmaceuticals, Inc.240 enrolled68 locationsNCT07215390
Recruiting
Phase 3
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
Age related macular degeneration (AMD)Geographic Atrophy (GA)
Regeneron Pharmaceuticals975 enrolled216 locationsNCT06541704
Recruiting
Phase 2
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA)
argenx60 enrolled17 locationsNCT07287982
Recruiting
Phase 2
A Study to Optimize Subretinal Surgical Delivery and to Evaluate Safety and Activity of Opregen in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (GAlette); Adaptive Optics (AO) Retinal Imaging Substudy in Association With Study GR44251
Geographic Atrophy
Genentech, Inc.60 enrolled17 locationsNCT05626114
Recruiting
ALS/MND Natural History Study Data Repository
ALSKennedy DiseasePLS+3 more
Massachusetts General Hospital5,000 enrolled18 locationsNCT05966038
Recruiting
Phase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
Hoffmann-La Roche28 enrolled16 locationsNCT05861986
Recruiting
Phase 2
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Spinal Muscular AtrophySMASpinal Muscular Atrophy Type 3+3 more
Scholar Rock, Inc.52 enrolled25 locationsNCT07047144
Recruiting
High Resolution Retinal Imaging
Geographic AtrophyAge-Related Macular DegenerationRetinitis Pigmentosa+2 more
University of Pennsylvania600 enrolled1 locationNCT01866371
Recruiting
Not Applicable
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Spinal Muscular AtrophyScoliosisSpine Deformity
Alcyone Therapeutics, Inc90 enrolled21 locationsNCT05866419
Recruiting
Phase 1
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
Muscular Atrophy, Spinal
Biogen58 enrolled19 locationsNCT06555419
Recruiting
UPenn Observational Research Repository on Neurodegenerative Disease
Corticobasal Syndrome(CBS)Progressive Supranuclear Palsy (PSP)Primary Progressive Aphasia(PPA)+6 more
University of Pennsylvania1,000 enrolled1 locationNCT04715399
Recruiting
The Myelin Disorders Biorepository Project
AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more
Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369