Genetic Disease Clinical Trials in New York, New York
3 recruitingNew York, New York
Showing 1–3 of 3 trials
Recruiting
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
Genetic Disease
National Human Genome Research Institute (NHGRI)20,000 enrolled33 locationsNCT02450851
Recruiting
Phase 3
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Beta-ThalassemiaSickle Cell DiseaseHematologic Diseases+4 more
Vertex Pharmaceuticals Incorporated26 enrolled6 locationsNCT05477563
Recruiting
STXBP1 and SYNGAP1 Related Disorders Natural History Study
Genetic DiseaseSYNGAP1-Related Intellectual DisabilitySTXBP1 Encephalopathy With Epilepsy
Children's Hospital of Philadelphia600 enrolled5 locationsNCT06555965