X-Linked Clinical Trials in Philadelphia, Pennsylvania
7 recruitingPhiladelphia, Pennsylvania
Showing 1–7 of 7 trials
Recruiting
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
X-linked Myotubular Myopathy
Astellas Gene Therapies50 enrolled10 locationsNCT06581146
Recruiting
Phase 2
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
Alport Syndrome, X-LinkedAlport Syndrome, Autosomal Recessive
Eloxx Pharmaceuticals, Inc.24 enrolled12 locationsNCT07523581
Recruiting
Rett Syndrome Registry
Genetic DiseaseNeurologic DisorderNervous System Diseases+7 more
International Rett Syndrome Foundation3,000 enrolled19 locationsNCT05432349
Recruiting
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)
Erythropoietic Protoporphyria (EPP)X-Linked Porphyria (XLP)
Portal Therapeutics, Inc.50 enrolled19 locationsNCT07567131
Recruiting
Phase 3
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
X-linked Retinoschisis
Atsena Therapeutics Inc.97 enrolled4 locationsNCT05878860
Recruiting
Early Phase 1
Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
LeukemiaInborn Errors of MetabolismBone Marrow Failure Syndromes+2 more
Children's Hospital of Philadelphia100 enrolled1 locationNCT02356653
Recruiting
The Myelin Disorders Biorepository Project
AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more
Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369