CPAP for Infantile Pompe Disease

National Taiwan University

Status and phase: Unknown; Phase 4

Conditions: Pompe Disease

Treatments: Procedure: continuous positive airway pressure

Study type: Interventional

Funder types: Other

Identifiers

NCT02405624

201401043RINA

Details and patient eligibility

About

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

Enrollment: 3 estimated patients

Sex: All

Ages: 4+ years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

CPAP training

Experimental group

Description:

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

Treatment:

Procedure: continuous positive airway pressure