ACTIVE_NOT_RECRUITING

Natural History Study of Patients With Canavan Disease (CANinform Study)

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 study uses medical records that allow retrospective data extraction of critical milestone and motor function data. In addition, prospective assessments collect data relevant to the natural history of Canavan disease in children.

Official Title

A Combination Retrospective Medical History and Prospective Observational Study of Patients With Canavan Disease for Assessment of Natural History of Canavan Disease

Quick Facts

Study Start:2019-10-10
Study Completion:2025-07-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04126005

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
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Meet age criteria of a specific cohort.
  2. 2. Confirmed clinical and biochemical diagnosis of Canavan disease.
  3. 3. Available medical records since birth that permit documentation of disease characteristics and developmental milestones.
  4. 4. Parent and/or legal guardian is able to read, understand, and sign the informed consent.
  1. 1. Patient does not meet the Inclusion Criteria.

Contacts and Locations

Study Locations (Sites)

UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
United States
Massachusetts General Hospital
Boston, Massachusetts, 02114
United States

Collaborators and Investigators

Sponsor: Aspa Therapeutics

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 Date2019-10-10
Study Completion Date2025-07-31

Study Record Updates

Study Start Date2019-10-10
Study Completion Date2025-07-31

Terms related to this study

Keywords Provided by Researchers

  • Canavan Disease
  • AAV
  • AAV9
  • Gene therapy
  • Aspartoacylase
  • ASPA
  • ASPA gene
  • rAAV9
  • ACY2
  • Aminoacylase 2
  • Spongy degeneration
  • N-acetyl-L-aspartic acid (NAA)
  • N-acetylaspartate
  • Rare disease
  • Inherited Metabolic Disorders
  • Leukodystrophy
  • Leukoencephalopathies
  • Autosomal Recessive Disorder
  • Neurodevelopmental diseases
  • CANinform Study

Additional Relevant MeSH Terms

  • Canavan Disease