NCT02950298|Completed
Pompe Telemedicine Developmental Study
Developmental Outcomes of School-aged Children With Infantile-onset Pompe Disease: A Telemedicine Approach to Assessment and Cognitive Training
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Developmental Outcomes of School-aged Children With Infantile-onset Pompe Disease: A Telemedicine Approach to Assessment and Cognitive Training
Sponsor study identifier
observational
22
1 country
1
Study Start
December 2015
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
March 2021
Actual (reported)Study Completion
March 3, 2021
Actual (reported)The primary purpose of this study is to:
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
March 3, 2021
Actual (reported)Click on a node to explore related trials.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
December 2015
Date type not reportedThis date has not been verified as actual or estimated.
June 22, 2016
Actual (reported)November 1, 2016
Actual (reported)March 2021
Actual (reported)March 3, 2021
Actual (reported)Relationship between daily behaviors in school-aged children with Pompe disease and observed speech patterns, as assessed by speech pathology.
2 years
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.
This outcome measure will be tested using measures testing cognitive function.
2 years
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.
This outcome measure will be tested using measures testing language abilities.
2 years
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.
Relationship between daily behaviors in school-aged children with Pompe disease and development, as assessed by Conners.
2 years
Relationship between daily behaviors in school-aged children with Pompe disease and development, as assessed by BRIEF-P.
2 years
Relationship between daily behaviors in school-aged children with Pompe disease and development, as assessed by Child Behavior Checklist.
2 years
This study is open to patients with classic Pompe disease.
Duke University Medical Center
Durham, North Carolina, 27710, United States
Mackey AP, Hill SS, Stone SI, Bunge SA. Differential effects of reasoning and speed training in children. Dev Sci. 2011 May;14(3):582-90. doi: 10.1111/j.1467-7687.2010.01005.x. Epub 2010 Nov 23.
Jones HN, Muller CW, Lin M, Banugaria SG, Case LE, Li JS, O'Grady G, Heller JH, Kishnani PS. Oropharyngeal dysphagia in infants and children with infantile Pompe disease. Dysphagia. 2010 Dec;25(4):277-83. doi: 10.1007/s00455-009-9252-x. Epub 2009 Sep 10.
Priya Kishnani
Duke University
22 participants (actual enrollment reported by the registry).
First Submitted
June 22, 2016
First Posted
November 1, 2016
Study Start
December 2015
Date type not reportedThis date has not been verified as actual or estimated.
Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)
2 years
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.
2 years
Contact the study team to confirm eligibility.
Ebbink BJ, Aarsen FK, van Gelder CM, van den Hout JM, Weisglas-Kuperus N, Jaeken J, Lequin MH, Arts WF, van der Ploeg AT. Cognitive outcome of patients with classic infantile Pompe disease receiving enzyme therapy. Neurology. 2012 May 8;78(19):1512-8. doi: 10.1212/WNL.0b013e3182553c11. Epub 2012 Apr 25.
Kishnani PS, Steiner RD, Bali D, Berger K, Byrne BJ, Case LE, Crowley JF, Downs S, Howell RR, Kravitz RM, Mackey J, Marsden D, Martins AM, Millington DS, Nicolino M, O'Grady G, Patterson MC, Rapoport DM, Slonim A, Spencer CT, Tifft CJ, Watson MS. Pompe disease diagnosis and management guideline. Genet Med. 2006 May;8(5):267-88. doi: 10.1097/01.gim.0000218152.87434.f3. No abstract available.
Primary Completion
March 2021
Actual (reported)Study Completion
March 3, 2021
Actual (reported)Last Updated
October 28, 2021
Record last verified: 2021-10
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