CPAP for Infantile Pompe Disease | NCT02405624

Studies
CPAP for Infantile Pompe Disease

NCT02405624

Sponsor

National Taiwan University Hospital

Status

Unknown status

Last Update Posted

Apr 1, 2015Estimated

Enrollment

3Estimated

Phase

Phase 4

Conditions

Pompe Disease

Interventions

continuous positive airway pressure

Countries

Taiwan

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2015

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 2014

Primary Completion Date

Dec 2015Estimated

Completion Date

Jun 2016Estimated

First Submitted Date

Mar 21, 2015

First Submission Date that Met QC Criteria

Mar 31, 2015

First Posted Date

Apr 1, 2015Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 31, 2015

Last Update Posted Date

Apr 1, 2015Estimated

Sponsor/Collaborators Module

Responsible Party, by Official Title

National Taiwan University Hospital, MD, PhD, National Taiwan University HospitalPrincipal Investigator

Lead Sponsor

National Taiwan University HospitalOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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 provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 4

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

3Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
CPAP training	Experimental	Patients will be instructed and follow the continuous positive airway pressure (CPAP) training procedure	Procedure: continuous positive airway pressure

Interventions

Name	Type	Description	Arm Group Labels	Other Names
continuous positive airway pressure	Procedure	CPAP training for muscle strength	CPAP training

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
decrease in hypernasality	score hypernasality degree and compare	6 months

Secondary Outcomes

Measure	Description	Time Frame
increase in speech intelligibility	score speech intelligibility degree and compare	6 months

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

4 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Yin-Hsiu Chien	Contact	886223123456	71937	chienyh@ntu.edu.tw

Overall Officials

Name	Affiliation	Role
Yin-Hsiu Chien, MD, PhD	National Taiwan University Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
National Taiwan University Hospital	Recruiting	Taipei	Taiwan	100	Taiwan

Condition Browse Module

MeSH Terms

ID	Term
D006009	Glycogen Storage Disease Type II

Ancestor Terms

ID	Term
D020140	Lysosomal Storage Diseases, Nervous System
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D045422	Continuous Positive Airway Pressure

Ancestor Terms

ID	Term
D011175	Positive-Pressure Respiration
D012121	Respiration, Artificial
D058109	Airway Management
D013812	Therapeutics

Browse Leaves

Not provided

Browse Branches

Not provided