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NCT02405624 Clinical Trial

CPAP for Infantile Pompe Disease

Pompe Disease Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT02405624
Other study ID # 201401043RINA
Secondary ID
Status Recruiting
Phase Phase 4
First received March 21, 2015
Last updated March 31, 2015
Start date June 2014
Est. completion date June 2016

Study information
Verified date March 2015
Source National Taiwan University Hospital
Contact Yin-Hsiu Chien
Phone 886223123456
Email chienyh@ntu.edu.tw
Is FDA regulated No
Health authority Taiwan: Bureau of Health
Study type Interventional

Clinical Trial Summary

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

Recruitment information / eligibility
Status Recruiting
Enrollment 3
Est. completion date June 2016
Est. primary completion date December 2015
Accepts healthy volunteers Accepts Healthy Volunteers
Gender Both
Age group 4 Years and older
Eligibility 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 Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Intervention

Procedure:
• continuous positive airway pressure
CPAP training for muscle strength

Locations
Country Name City State
Taiwan National Taiwan University Hospital Taipei

Sponsors (1)
Lead Sponsor Collaborator
National Taiwan University Hospital

Country where clinical trial is conducted

Taiwan, 

Outcome
Type Measure Description Time frame Safety issue
Primary decrease in hypernasality score hypernasality degree and compare 6 months No
Secondary increase in speech intelligibility score speech intelligibility degree and compare 6 months No
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