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Trial record 68 of 103 for:    Pompe Disease

Pompe Telemedicine Developmental Study

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ClinicalTrials.gov Identifier: NCT02950298
Recruitment Status : Recruiting
First Posted : November 1, 2016
Last Update Posted : November 21, 2018
Sponsor:
Information provided by (Responsible Party):
Duke University

Brief Summary:

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

Condition or disease
Pompe Disease Glycogen Storage Disease II

Detailed Description:
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.

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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: Developmental Outcomes of School-aged Children With Infantile-onset Pompe Disease: A Telemedicine Approach to Assessment and Cognitive Training
Study Start Date : December 2015
Estimated Primary Completion Date : July 2019
Estimated Study Completion Date : July 2019





Primary Outcome Measures :
  1. Relationship between daily behaviors in school-aged children with Pompe disease and observed speech patterns, as assessed by speech pathology. [ Time Frame: 2 years ]
  2. Document the developmental outcomes and cognitive function of individuals with Pompe disease treated with long-term ERT through school-age (ages 6-18), as assessed by PPVT-4. [ Time Frame: 2 years ]
    This outcome measure will be tested using measures testing cognitive function.

  3. Document the developmental outcomes and language abilities of individuals with Pompe disease treated with long-term ERT through school-age (ages 6-18), as assessed by CELF-5. [ Time Frame: 2 years ]
    This outcome measure will be tested using measures testing language abilities.

  4. Document the developmental outcomes and cognitive function of individuals with Pompe disease treated with long-term ERT through school-age (ages 6-18), as assessed by Woodcock-Johnson Test of Achievement. [ Time Frame: 2 years ]
  5. Document the developmental outcomes and cognitive function of individuals with Pompe disease treated with long-term ERT through school-age (ages 6-18), as assessed by Leiter. [ Time Frame: 2 years ]

Secondary Outcome Measures :
  1. Relationship between daily behaviors in school-aged children with Pompe disease and development, as assessed by Conners. [ Time Frame: 2 years ]
  2. Relationship between daily behaviors in school-aged children with Pompe disease and development, as assessed by BRIEF-P. [ Time Frame: 2 years ]
  3. Relationship between daily behaviors in school-aged children with Pompe disease and development, as assessed by Child Behavior Checklist. [ Time Frame: 2 years ]


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Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
This study is open to patients with classic Pompe disease.
Criteria

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:


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02950298


Contacts
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Contact: Stephanie Austin 919-668-1347 stephanie.austin@duke.edu
Contact: Ela Stefanescu 919-681-4026 mihaela.stefanescu@duke.edu

Locations
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United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Duke University
Investigators
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Principal Investigator: Priya Kishnani Duke University

Publications:
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Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT02950298     History of Changes
Other Study ID Numbers: Pro00059526
First Posted: November 1, 2016    Key Record Dates
Last Update Posted: November 21, 2018
Last Verified: November 2018

Keywords provided by Duke University:
Pompe
Glycogen Storage Disease II
Telemedicine
Duke University Medical Center

Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors