CPAP for Infantile Pompe Disease

Phase 4

• Conditions: Pompe Disease
• Interventions: Procedure: continuous positive airway pressure

• Registration Number: NCT02405624
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-06
• Status Verified: 2015-03
• Study First Submitted: 2015-03-21
• Study First Submitted QC: 2015-03-31
• Last Update Submitted: 2015-03-31
• Study First Posted: 2015-04-01
• Last Update Posted: 2015-04-01
• Primary Completion: 2015-12
• Study Completion: 2016-06

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 3

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

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
CPAP training	continuous positive airway pressure	Patients will be instructed and follow the continuous positive airway pressure (CPAP) training procedure

Primary Outcome Measures

Name	Time	Method
decrease in hypernasality	6 months	score hypernasality degree and compare

Secondary Outcome Measures

Name	Time	Method
increase in speech intelligibility	6 months	score speech intelligibility degree and compare

Trial Locations

Locations (1)

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan