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CPAP for Infantile Pompe Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02405624
Recruitment Status : Unknown
Verified March 2015 by National Taiwan University Hospital.
Recruitment status was:  Recruiting
First Posted : April 1, 2015
Last Update Posted : April 1, 2015
Information provided by (Responsible Party):
National Taiwan University Hospital

Brief Summary:
Patients with infantile onset Pompe disease will be trained with continuous positive airway pressure to see if hypernasality can be improved.

Condition or disease Intervention/treatment Phase
Pompe Disease Procedure: continuous positive airway pressure Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy of Continuous Positive Airway Pressure of Treatment of Hypernasality of Children With Infantile Pompe Disease
Study Start Date : June 2014
Estimated Primary Completion Date : December 2015
Estimated Study Completion Date : June 2016

Arm Intervention/treatment
Experimental: CPAP training
Patients will be instructed and follow the continuous positive airway pressure (CPAP) training procedure
Procedure: continuous positive airway pressure
CPAP training for muscle strength

Primary Outcome Measures :
  1. decrease in hypernasality [ Time Frame: 6 months ]
    score hypernasality degree and compare

Secondary Outcome Measures :
  1. increase in speech intelligibility [ Time Frame: 6 months ]
    score speech intelligibility degree and compare

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

  1. confirm as Pompe disease
  2. Age 4 and can speak 2-words sentence by order
  3. mild to severe hypernasality
  4. have regular speech therapy for 8 weeks or longer

Exclusion Criteria:

(1)hearing loss (2)congenital pharyngeal or laryngeal structure anomalies including cleft palate (3)cerebral palsy (4)recent otitis media effusion (5)very severe hypernasality (6)use non-invasive ventilator

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 identifier (NCT number): NCT02405624

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Contact: Yin-Hsiu Chien 886223123456 ext 71937

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National Taiwan University Hospital Recruiting
Taipei, Taiwan, 100
Contact: Yin-Hsiu Chien, MD, PhD    +886223123456 ext 71937   
Sponsors and Collaborators
National Taiwan University Hospital
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Principal Investigator: Yin-Hsiu Chien, MD, PhD National Taiwan University Hospital

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Responsible Party: National Taiwan University Hospital, MD, PhD Identifier: NCT02405624     History of Changes
Other Study ID Numbers: 201401043RINA
First Posted: April 1, 2015    Key Record Dates
Last Update Posted: April 1, 2015
Last Verified: March 2015
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Glycogen Storage Disease
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors