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A Post-Authorization Safety Study (PASS) to Assess Long-term Safety in Patients with Pompe Disease Treated with Avalglucosidase alfa in the Real-World Setting: the SAVANT (Safety assessment of AValglucosidase Alfa as a Novel Treatment) study-STUDY00005276

To collect long term safety data and evaluate the important identified risk of infusion associated reactions (IARs) including hypersensitivity and...

Refractive Outcomes in Macular Edema Prematurity-PIROSTUDY15464

Retinopathy of prematurity (ROP) is a potentially blinding eye disease that occurs in premature infants due to abnormal blood vessel development...

TITRE: Trial of Indication Based Red Cell Transfusion in ECMO-STUDY00004064

This is a randomized clinical trial to determine if indication-based blood cell (RBC) transfusion based on reduced tissue oxygen delivery,...

The Very Early Onset Inflammatory Bowel Disease (VEO-IBD) Repository -STUDY00002767

This study will create a repository of samples from patients with Very Early Onset Inflammatory Bowel Disease (VEO-IBD) to allow for study in the...

TReatment for ImmUne Mediated PathopHysiology -STUDY00003359

This is a phase 2b, double-blind, tree arm, randomized, placebo controlled trial with restricted response adaptive randomization. The trial is...

PNOC COMP: A Protocol for Children and Young Adults Diagnosed with a Central Nervous System (CNS) Tumor to Assess Cognitive, Quality of Life (QOL), and Comprehensive Effects of Therapies-STUDY00004830

The rationale for this protocol is to provide a framework to collect comprehensive outcome data on children and young adults with primary...

PLAY 2 – Preschoolers Learning and Active in PLAY-STUDY00000628

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

Phase 1b/2, randomized, double-blind, placebo-controlled, multi-center study of STMC-103H in neonates and infants at high-risk for developing allergic disease-STUDY00003212

Primary indication is to see is STMC-103H, prevents allergic disease in at-risk neonates. The primary objective of this study is to assess the...

Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children per Standard of Care / (POPS): NICHD-2019-POP02-STUDY00002420

Prospective, multicenter study of understudied drugs. Participants will receive drugs of interest administered per standard of care. Primary:...

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