CPAP for Infantile Pompe Disease

NCT ID: NCT02405624

Last Updated: 2015-04-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-06-30

Study Completion Date

2016-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Pompe Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

CPAP training

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

Group Type EXPERIMENTAL

continuous positive airway pressure

Intervention Type PROCEDURE

CPAP training for muscle strength

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

continuous positive airway pressure

CPAP training for muscle strength

Intervention Type PROCEDURE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

4 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

National Taiwan University Hospital

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

National Taiwan University Hospital

MD, PhD

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Yin-Hsiu Chien, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

National Taiwan University Hospital

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

National Taiwan University Hospital

Taipei, Taiwan, Taiwan

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Taiwan

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Yin-Hsiu Chien

Role: CONTACT

886223123456 ext. 71937

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Yin-Hsiu Chien, MD, PhD

Role: primary

+886223123456 ext. 71937

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

201401043RINA

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.