Rennaz, Switzerland

National Collaborative to Improve Care of Children With Complex Congenital Heart Disease

The purpose of this initiative is to improve care and outcomes for infants with HLHS by: 1) expanding the established NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, 2) improving implementation of consensus standards, tested by teams, into everyday practice across pediatric cardiology centers, and 3) engaging parents as partners in improving care and outcomes.

Neuroblastoma Maintenance Therapy Trial

Pediatric Precision Laboratory Advanced Neuroblastoma Therapy

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

Principal Investigators: The principal investigators (PIs) will be transplant physicians at all participating U.S. transplant centers. Study Design: This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. Primary Objective: The primary objective of this study is to examine the incidence of neutrophil recovery of ≥500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licensed. Secondary Objectives: In patients receiving a non-licensed CBU: - Assess incidence of transmission of infection - Assess incidence of serious infusion reaction - Determine 1 year overall survival after cord blood transplantation - Assess cumulative incidence of acute graft vs. host disease (GVHD) grades II to IV and grades III to IV - Assess cumulative incidence of chronic GVHD - Determine platelet engraftment of >20,000 mcL and >50,000 mcL

The Pediatric Anesthesia Quality Improvement Project

Mucopolysaccharidosis I (MPS I) Registry

The MPS I Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact: - In Asia-Pacific - Vivian Liu, +65-6431-2548, Vivian.liu@genzyme.com - In Europe - +31-35-699-1232, europe@mpsiregistry.com - In Latin America - +617-591-5500, help@mpsiregistry.com - In North America - +617-591-5500, help@mpsiregistry.com

Fabry Disease Registry & Pregnancy Sub-registry

DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma

In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 2500 mg/m2 BID on each day of study. Subjects will be evaluated in 3 Cohorts: Cohort 1: Molecular High Risk Medulloblastoma Cohort 2: Molecular Very High Risk Medulloblastoma Cohort 3: Relapsed/Refractory Medulloblastoma A total of 118 subjects across all cohorts will be enrolled to ensure that there will be 107 evaluable subjects (32-39 per cohort)

Pompe Disease Registry Protocol

Study Design Time Perspective: Retrospective and Prospective