447 | Term | Boston Children's Hospital

Vedolizumab IV in Pediatric Participants With Ulcerative Colitis (UC) or Crohn's Disease (CD)

The purpose of this study is to evaluate vedolizumab pharmacokinetics (PK), safety and tolerability in pediatric participants with moderately to severely active UC or CD.

A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for F508del.

Fontan Imaging Biomarkers (FIB) Study

The goal of the Fontan Imaging Biomarkers (FIB) study is to identify the associations of blood and urine biomarkers to imaging parameters of ventricular mechanics.

A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

This study will evaluate the efficacy and safety of OC5 in patients with PH.

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency

This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with monogenic, interleukin-18 (IL 18) driven autoinflammation due to Nucleotide-binding oligomerization domain, ...

Biokinetics Study for Tc-99m MAG3 in Pediatric Molecular Imaging

The radiation exposure resulting from medical imaging is a topic of some concern. Nuclear medicine provides potentially life-saving information regarding physiological processes, and is of particular ...

Biokinetics Study for Tc-99m MDP in Pediatric Molecular Imaging

Biokinetics Study of Tc-99m DMSA in Pediatric Molecular Imaging

Rare Diseases Clinical Research Network: Neurophysiological Correlates

The overall purpose of this project is to advance understanding of the neurophysiological features of Rett syndrome (RTT), MECP2 Duplication (MECP2 Dup) and RTT-related disorders (CDKL5, FOXG1) to ...

The Boston Circulatory Arrest Study: Antecedents and Correlates of Well-Being in Adults With Congenital Heart Disease

In the landmark Boston Circulatory Arrest Study, neurologic and developmental status was measured following infant heart surgery and then prospectively at ages 1, 2.5, 4, 8, and 16 years, with ...

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