RECRUITING

Cognitive and Neurological Pathologies in Pompe Disease

Conditions

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

The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves. Additionally, the investigators will study the relationship between the abnormal changes in brain, nervous system findings, and developmental outcomes. The investigators will collect clinical information from clinic visits as well as assessments such as neuroimaging (magnetic resonance imaging (MRI), magnetic resonance spectroscopy (MRS), and diffusion tensor imaging (DTI)), cognition, academic skills, speech and language function, physical therapy and quantitative muscle ultrasound. Subjects will be in this study for at least 3 years and up to 6 years.

Official Title

Cognitive and Neurological Pathologies in Pompe Disease

Quick Facts

Study Start:2016-08-03
Study Completion:2025-03
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04639336

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:0 Years to 18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Subject has a confirmed and documented diagnosis of infantile Pompe Disease (IPD) or Late onset Pompe Disease (LOPD)
  1. * Refusal to give informed consent

Contacts and Locations

Study Contact

Gretchen Nichting
CONTACT
+1 919 660 0757
gretchen.nichting@duke.edu

Principal Investigator

Priya Kishnani, MD
PRINCIPAL_INVESTIGATOR
Duke University

Study Locations (Sites)

Duke University Medical Center
Durham, North Carolina, 27710
United States

Collaborators and Investigators

Sponsor: Duke University

  • Priya Kishnani, MD, PRINCIPAL_INVESTIGATOR, Duke University

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 Date2016-08-03
Study Completion Date2025-03

Study Record Updates

Study Start Date2016-08-03
Study Completion Date2025-03

Terms related to this study

Additional Relevant MeSH Terms

  • Pompe Disease