NCT02405624

Brief Summary

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

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
3

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Jun 2014

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2014

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

March 21, 2015

Completed
11 days until next milestone

First Posted

Study publicly available on registry

April 1, 2015

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2015

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2016

Completed
Last Updated

April 1, 2015

Status Verified

March 1, 2015

Enrollment Period

1.5 years

First QC Date

March 21, 2015

Last Update Submit

March 31, 2015

Conditions

Pompe Disease

Outcome Measures

Primary Outcomes (1)

  • decrease in hypernasality

    score hypernasality degree and compare

    6 months

Secondary Outcomes (1)

  • increase in speech intelligibility

    6 months

Study Arms (1)

CPAP training

EXPERIMENTAL

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

Procedure: continuous positive airway pressure

Interventions

continuous positive airway pressurePROCEDURE

CPAP training for muscle strength

CPAP training

Eligibility Criteria

Age4 Years+
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • (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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Taiwan University Hospital

Taipei, Taiwan, 100, Taiwan

RECRUITING

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

Continuous Positive Airway Pressure

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Positive-Pressure RespirationRespiration, ArtificialAirway ManagementTherapeuticsRespiratory Therapy

Study Officials

  • Yin-Hsiu Chien, MD, PhD

    National Taiwan University Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Yin-Hsiu Chien

CONTACT

886223123456chienyh@ntu.edu.tw

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, PhD

Study Record Dates

First Submitted

March 21, 2015

First Posted

April 1, 2015

Study Start

June 1, 2014

Primary Completion

December 1, 2015

Study Completion

June 1, 2016

Last Updated

April 1, 2015

Record last verified: 2015-03

Locations

TW(1)