- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02405624
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
Intervention / Treatment
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
- Name: Yin-Hsiu Chien
- Phone Number: 71937 886223123456
- Email: chienyh@ntu.edu.tw
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:
- 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
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
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Carbohydrate Metabolism, Inborn Errors
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Lysosomal Storage Diseases, Nervous System
- Glycogen Storage Disease
- Glycogen Storage Disease Type II
Other Study ID Numbers
- 201401043RINA
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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