Back to resultsCPAP for Infantile Pompe Disease
Primary Purpose
Pompe Disease
Status
Unknown status
Phase
Phase 4
Locations
Taiwan
Study Type
Interventional
Intervention
continuous positive airway pressure
Sponsored by
About this trial
This is an interventional treatment trial for Pompe Disease
Eligibility Criteria
Inclusion Criteria:
- confirm as Pompe disease
- Age 4 and can speak 2-words sentence by order
- mild to severe hypernasality
- 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
Sites / Locations
- National Taiwan University HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CPAP training
Arm Description
Patients will be instructed and follow the continuous positive airway pressure (CPAP) training procedure
Outcomes
Primary Outcome Measures
decrease in hypernasality
score hypernasality degree and compare
Secondary Outcome Measures
increase in speech intelligibility
score speech intelligibility degree and compare
Full Information
NCT ID
NCT02405624
First Posted
March 21, 2015
Last Updated
March 31, 2015
Sponsor
National Taiwan University Hospital
1. Study Identification
Unique Protocol Identification Number
NCT02405624
Brief Title
CPAP for Infantile Pompe Disease
Official Title
Efficacy of Continuous Positive Airway Pressure of Treatment of Hypernasality of Children With Infantile Pompe Disease
Study Type
Interventional
2. Study Status
Record Verification Date
March 2015
Overall Recruitment Status
Unknown status
Study Start Date
June 2014 (undefined)
Primary Completion Date
December 2015 (Anticipated)
Study Completion Date
June 2016 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
National Taiwan University Hospital
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
Patients with infantile onset Pompe disease will be trained with continuous positive airway pressure to see if hypernasality can be improved.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
CPAP training
Arm Type
Experimental
Arm Description
Patients will be instructed and follow the continuous positive airway pressure (CPAP) training procedure
Intervention Type
Procedure
Intervention Name(s)
continuous positive airway pressure
Intervention Description
CPAP training for muscle strength
Primary Outcome Measure Information:
Title
decrease in hypernasality
Description
score hypernasality degree and compare
Time Frame
6 months
Secondary Outcome Measure Information:
Title
increase in speech intelligibility
Description
score speech intelligibility degree and compare
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria:
confirm as Pompe disease
Age 4 and can speak 2-words sentence by order
mild to severe hypernasality
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Yin-Hsiu Chien
Phone
886223123456
Ext
71937
Email
chienyh@ntu.edu.tw
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yin-Hsiu Chien, MD, PhD
Organizational Affiliation
National Taiwan University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yin-Hsiu Chien, MD, PhD
Phone
+886223123456
Ext
71937
Email
chienyh@ntu.edu.tw
12. IPD Sharing Statement
Learn more about this trial
CPAP for Infantile Pompe Disease
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