Pompe DiseasePhase 4NCT02405624
CPAP for Infantile Pompe Disease
A Phase 4 study testing continuous positive airway pressure in people with Pompe Disease, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
Patients with infantile onset Pompe disease will be trained with continuous positive airway pressure to see if hypernasality can be improved. Read more about this study
- Who is it for?
- Ages 4 years and olderHealthy volunteers can take part
- Conditions the study is for
- Pompe Disease
- What is being tested?
- continuous positive airway pressure
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteTaiwan
- Study dates
- Jun 2014to Jun 2016
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- (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 locations and contacts
1 site in 1 country.
Taiwan1 site, 1 recruiting
- Recruiting
National Taiwan University Hospital
Taipei, Taiwan, 100
Yin-Hsiu Chien, MD, PhD, +886223123456 ext. 71937, chienyh@ntu.edu.tw
About this study
Official title: Efficacy of Continuous Positive Airway Pressure of Treatment of Hypernasality of Children With Infantile Pompe Disease
Patients with infantile onset Pompe disease will be trained with continuous positive airway pressure to see if hypernasality can be improved.
- Conditions
- Pompe Disease
- Being tested
- continuous positive airway pressure
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
continuous positive airway pressureProcedure or surgery
CPAP training for muscle strength
| Study group | Role | What they receive |
|---|---|---|
| CPAP training | Experimental | Patients will be instructed and follow the continuous positive airway pressure (CPAP) training procedure Receives: Procedure: continuous positive airway pressure |
What is being measured
The primary outcome is the main question the study is designed to answer.
decrease in hypernasality
score hypernasality degree and compare
Measured: 6 months
Secondary outcomes (1)
increase in speech intelligibility
score speech intelligibility degree and compare
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2015 (estimated)
- Study startJun 2014
- Primary completionDec 2015 (estimated)
- Study completionJun 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe DiseaseStudies of the same condition, open and under way.
- More trials run by National Taiwan University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, National Taiwan University Hospital, National Taiwan University Hospital
- Investigators
- Yin-Hsiu Chien, MD, PhD, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02405624
- Sponsor's protocol ID
- 201401043RINA
- Status last verified
- Mar 2015
- First submitted
- 21 Mar 2015
- Last updated
- 1 Apr 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Continuous Positive Airway Pressure of Treatment of Hypernasality of Children With Infantile Pompe Disease
- ClinicalTrials.gov ID
- NCT02405624
- Acronym / sponsor ID
- 201401043RINA
- Conditions
- Pompe Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- Jun 2014
- Primary completion
- Dec 2015 (estimated)
- Study completion
- Jun 2016 (estimated)
- First posted
- 1 Apr 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients with infantile onset Pompe disease will be trained with continuous positive airway pressure to see if hypernasality can be improved.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CPAP training | Experimental | Procedure: continuous positive airway pressurePatients will be instructed and follow the continuous positive airway pressure (CPAP) training procedure |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- (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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| continuous positive airway pressureCPAP training for muscle strength | Procedure or surgery | CPAP training |
Primary outcome
decrease in hypernasality
Time frame: 6 months
score hypernasality degree and compare
1 secondary outcome
increase in speech intelligibility
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yin-Hsiu Chien, MD, PhD principal investigator
National Taiwan University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing continuous positive airway pressure in people with Pompe Disease, run by National Taiwan University Hospital at 1 site in Taiwan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 1 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yin-Hsiu Chien (886223123456 ext. 71937, chienyh@ntu.edu.tw), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
decrease in hypernasality (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Apr 2015.