RECRUITING

Registry of Patients Diagnosed With Lysosomal Storage Diseases

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Conditions

Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV AMucopolysaccharidosis VIMucopolysaccharidosis VIIPompe Disease Infantile-OnsetNeuronopathic Gaucher DiseaseWolman DiseaseLysosomal Storage DiseaseLSDsInborn Error of MetabolismHurler SyndromeSly SyndromeHunter SyndromeMucopolysaccharidosis IVaMPSMucopolysaccharidosis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.

Official Title

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Quick Facts

Study Start:2022-05-31
Study Completion:2050-05-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05619900

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 64 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Patients aged 0-64 with a diagnosis of a lysosomal storage disease
  2. * Pregnant patients whose fetus has a diagnosis of a lysosomal storage disease
  1. * There are no current exclusion criteria

Contacts and Locations

Study Contact

Billie Lianoglou, MS
CONTACT
415-476-2461
billie.lianoglou@ucsf.edu
Emma Canepa, MS, CCRP
CONTACT
415-476-7255
Emma.Canepa@ucsf.edu

Principal Investigator

Tippi C MacKenzie, MD
PRINCIPAL_INVESTIGATOR
University of California, San Francisco

Study Locations (Sites)

University of California San Francisco
San Francisco, California, 94143
United States

Collaborators and Investigators

Sponsor: University of California, San Francisco

  • Tippi C MacKenzie, MD, PRINCIPAL_INVESTIGATOR, University of California, San Francisco

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-05-31
Study Completion Date2050-05-31

Study Record Updates

Study Start Date2022-05-31
Study Completion Date2050-05-31

Terms related to this study

Keywords Provided by Researchers

  • Lysosomal Storage Disease
  • LSDs
  • Inborn Error of Metabolism
  • Hurler Syndrome
  • Sly Syndrome
  • Hunter Syndrome
  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis II
  • Mucopolysaccharidosis IVa
  • Mucopolysaccharidosis VI
  • Mucopolysaccharidosis VII
  • Pompe Disease Infantile-Onset
  • Neuronopathic Gaucher Disease
  • Wolman Disease
  • MPS
  • Mucopolysaccharidosis

Additional Relevant MeSH Terms

  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis II
  • Mucopolysaccharidosis IV A
  • Mucopolysaccharidosis VI
  • Mucopolysaccharidosis VII
  • Pompe Disease Infantile-Onset
  • Neuronopathic Gaucher Disease
  • Wolman Disease