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Therapies for Autoimmune Disease

Our investigators are world leaders in the creation of innovative genetic therapies with the potential to transform the treatment of autoimmune...

SC-N176: Chimeric Antigen Receptor T-cell Pediatrics Living with Uncertainty (CAR T-PLUS) Intervention Proof of Concept Study-STUDY00005072

The objective of this study will be to apply the ORBIT model to complete intervention development, refinement, and pilot testing of novel...

A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCRαβ+/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PID-STUDY00002451

Busulfan exposure-finding for SCID with TCRαβ+/CD19+ depleted transplantation

A Randomized Trial of Dichoptic Treatment for Amblyopia in Children 4 to 7 Years of Age -STUDY00005027

To determine if dichoptic therapy using Luminopia is confirmed to be non- inferior to stardard part-time patching for best- corrected visual...

POEM: A PrOspective Registry of Pediatric Cellular Therapy Patients at risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS)-STUDY00004501

This is a prospective international multi-center registry and biorepository trial of children and adolescents/young adults (AYA) undergoing...

MSTEP R01-STUDY00002697

To learn more about this study, please email the study team member listed below.

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (TSC-STEPS)-STUDY00004338

This trial will use a phase IIb, randomized, double-blind, placebo-controlled, multicenter clinical trial design to evaluate the safety and...

A Randomized Trial to Evaluate Sequential vs Simultaneous Spectacles plus Patching for Amblyopia in Children 3 to <13 Years Old (ATS22) protocol-STUDY00002734

This study is enrolling participants between 3 to under 13 years to evaluate sequential vs simultaneous spectacles plus patching for Amblyopia.

Pediatric Options for Migraine Relief: A Randomized, Double-Blind, Placebo-Controlled Study of Lasmiditan for Acute Treatment of Migraine: PIONEER-PEDS1-STUDY00002623

This study is being done to see how safe lasmiditan is and how well it will work to help people with migraines. This study will see how lasmiditan...

Pediatric Immune Monitoring to Optimize Superior Allograft Outcomes in Transplantation Outcomes of KidneyCare in Renal Allografts-STUDY00003047

This is a prospective, multicenter observational study, with inbuilt real-time longitudinal sampling, and batched testing of novel measures....