Pompe Telemedicine Developmental Study
| Status: | Recruiting |
|---|---|
| Conditions: | Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | 6 - 18 |
| Updated: | 11/23/2018 |
| Start Date: | December 2015 |
| End Date: | July 2019 |
| Contact: | Stephanie Austin |
| Email: | stephanie.austin@duke.edu |
| Phone: | 919-668-1347 |
Developmental Outcomes of School-aged Children With Infantile-onset Pompe Disease: A Telemedicine Approach to Assessment and Cognitive Training
The primary purpose of this study is to:
- Document the developmental outcomes of individuals with Pompe disease treated with
long-term enzyme-replacement therapy (ERT) through school-age (ages 6-18) using measures
of cognitive functioning, academic skills, and speech and language abilities.
- Investigate possible cognitive processing speed weaknesses using BrainBaseline
neurocognitive assessment software.
- Investigate the relationship between behavior and other developmental factors including
speech and language ability and cognitive ability.
- Explore if the use of selected iPad applications may help strengthen cognitive
processing speed in children with Pompe disease
- Document the developmental outcomes of individuals with Pompe disease treated with
long-term enzyme-replacement therapy (ERT) through school-age (ages 6-18) using measures
of cognitive functioning, academic skills, and speech and language abilities.
- Investigate possible cognitive processing speed weaknesses using BrainBaseline
neurocognitive assessment software.
- Investigate the relationship between behavior and other developmental factors including
speech and language ability and cognitive ability.
- Explore if the use of selected iPad applications may help strengthen cognitive
processing speed in children with Pompe disease
The study will last approximately 2-3 years. It includes 2-4 visits to Duke for developmental
assessments. The baseline/year 1 visit may take place at Duke (may take 1-2 days) or remotely
via iPad. The follow up 1/year 2 visit may take place at Duke (1-2 days) or remotely via an
iPad, depending upon each child's particular situation.
assessments. The baseline/year 1 visit may take place at Duke (may take 1-2 days) or remotely
via iPad. The follow up 1/year 2 visit may take place at Duke (1-2 days) or remotely via an
iPad, depending upon each child's particular situation.
Inclusion Criteria:
- Age range 6-18 years
- Diagnosis of classic Pompe disease by enzyme or molecular methods
- Patient, parent, or legal guardian is willing and able to give written informed
consent
- English speaking child and care giver.
Exclusion Criteria:
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