Non-invasive Functional Assessment and Pathogenesis of Morquio A

RecruitingOBSERVATIONAL
Enrollment

60

Participants

Timeline

Start Date

May 1, 2021

Primary Completion Date

April 30, 2026

Study Completion Date

April 30, 2026

Conditions
Mucopolysaccharidosis IV Type AMorquio A SyndromeMPS IVA
Interventions
DIAGNOSTIC_TEST

Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.

This study includes 15 major assessments: clinical assessment procedures; anthropometric measurements; activity of daily living and quality-of-life questionnaires; gait kinematics and kinetics analysis; pulmonary function tests (PFT); skeletal radiographs and dual-energy x-ray absorptiometry (DXA); MRI in cervical spine, temporal bones, and hip; computed tomography angiography (CTA) for tracheal obstruction; CT for temporal bones; anesthetic encounters; joint mobility; hearing function; biochemical analyses; and pathological analyses.

Trial Locations (1)

19803

RECRUITING

Nemours Children's Health, Delaware Valley, Wilmington

All Listed Sponsors
collaborator

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

lead

Nemours Children's Clinic

OTHER