RECRUITING

Non-invasive Functional Assessment and Pathogenesis of Morquio A

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

Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all ages of patients to better understand the progress of their disease and the most serious clinical problems (cervical instability and stenosis, tracheal obstruction, hyperlaxity of joints, hip dysplasia, and small lung capacity). The outcome of this project will lead to a more precise understanding of the skeletal/pulmonary compromise and defining clinical endpoints in this disease for future clinical trials of current or developing therapies.

Official Title

Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)

Quick Facts

Study Start:2021-05-01
Study Completion:2026-04-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05284006

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. * Patients affected by MPS IVA. The diagnosis of MPS IVA is confirmed by deficient enzyme activity of \< 5% of normal activity level as measured in plasma or leukocytes.
  1. * No

Contacts and Locations

Study Contact

Shunji Tomatsu, MD PhD
CONTACT
3022987336
stomatsu@nemours.org
Greg Stets
CONTACT
302-298-6504
gstets@nemours.org

Principal Investigator

Shunji Tomatsu, MD PhD
PRINCIPAL_INVESTIGATOR
Nemours Children's Care

Study Locations (Sites)

Nemours Children's Health, Delaware Valley
Wilmington, Delaware, 19803
United States

Collaborators and Investigators

Sponsor: Nemours Children's Clinic

  • Shunji Tomatsu, MD PhD, PRINCIPAL_INVESTIGATOR, Nemours Children's Care

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 Date2021-05-01
Study Completion Date2026-04-30

Study Record Updates

Study Start Date2021-05-01
Study Completion Date2026-04-30

Terms related to this study

Keywords Provided by Researchers

  • skeletal dysplasia
  • GALNS
  • keratan sulfate
  • tracheal narrowing
  • short stature

Additional Relevant MeSH Terms

  • Mucopolysaccharidosis IV Type A
  • Morquio A Syndrome
  • MPS IVA