CPAP for Infantile Pompe Disease

March 31, 2015 updated by: National Taiwan University Hospital

Efficacy of Continuous Positive Airway Pressure of Treatment of Hypernasality of Children With Infantile Pompe Disease

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

Study Overview

Status

Unknown

Conditions

Study Type

Interventional

Enrollment (Anticipated)

3

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

      • Taipei, Taiwan, 100
        • Recruiting
        • National Taiwan University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
decrease in hypernasality
Time Frame: 6 months
score hypernasality degree and compare
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
increase in speech intelligibility
Time Frame: 6 months
score speech intelligibility degree and compare
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Yin-Hsiu Chien, MD, PhD, National Taiwan University Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2014

Primary Completion (Anticipated)

December 1, 2015

Study Completion (Anticipated)

June 1, 2016

Study Registration Dates

First Submitted

March 21, 2015

First Submitted That Met QC Criteria

March 31, 2015

First Posted (Estimate)

April 1, 2015

Study Record Updates

Last Update Posted (Estimate)

April 1, 2015

Last Update Submitted That Met QC Criteria

March 31, 2015

Last Verified

March 1, 2015

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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