Pompe DiseasePhase 4NCT02405598
Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
A Phase 4 study testing Salbutamol in people with Pompe Disease, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
evaluate if beta 2-adrenergic agonist can have adjuvant effect to patients with infantile-onset Pompe disease under enzyme replacement therapy Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Pompe Disease
- What is being tested?
- Salbutamol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteTaiwan
- Study dates
- Oct 2013to 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirm diagnosis as Pompe disease
- age 2 years or above
- under at least 1 year regular recombinant human acid alpha glucosidase, and no dosage/frequency change in recent 3 months
- Provide inform consent
Who cannot join
- chronic heart disease such as arrhythmia, cardiomyopathy, cardiac infarction
- history of seizure
- history of diabetes mellitus
- history of hyperthyroidism
- hypokalemia
- pregnant
- allergy to β-agonists
- under medications including diuretics, digoxin, beta-blockers, etc
Trial locations and contacts
1 site in 1 country.
About this study
evaluate if beta 2-adrenergic agonist can have adjuvant effect to patients with infantile-onset Pompe disease under enzyme replacement therapy
- Conditions
- Pompe Disease
- Being tested
- Salbutamol
- 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.
SalbutamolDrug
- Albuterol Syrup (1mL= Salbutamol 0.4mg, 60ml/bot)
- VENTOLIN TABLETS 2MG (SALBUTAMOL SULPHATE)
| Study group | Role | What they receive |
|---|---|---|
| Salbutamol | Experimental | 1. age 2-6 year: 0.1mg/kg tid x 2 weeks, then gradually increase to 0.2mg/kg tid (daily total maximal12mg)
2. age 6-12 year: 2mg tid x 2 weeks, then gradually increase to 4mg tid (daily total maximal 24mg)
3. age 12 year and above: 4mg tidx 2 weeks, then gradually increase to 8mg tid (daily total maximal 32mg) Receives: Drug: Salbutamol |
What is being measured
The primary outcome is the main question the study is designed to answer.
10% decrement of creatinine kinase
measure and compare serum creatinine kinase levels
Measured: 6 months
Improvement in 6-minutes walk test
measure and compare 6-minutes walk test
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 startOct 2013
- Primary completionJul 2015
- Study completionJun 2016
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
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hsiao Sang Chu, MD, National Taiwan University Hospital
- Investigators
- Yin-Hsiu Chien, MD, PhD, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02405598
- Sponsor's protocol ID
- 201308031MINC
- Status last verified
- Sep 2016
- First submitted
- 22 Mar 2015
- Last updated
- 20 Sep 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
- ClinicalTrials.gov ID
- NCT02405598
- Acronym / sponsor ID
- 201308031MINC
- Conditions
- Pompe Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- Oct 2013
- Primary completion
- Jul 2015 (actual)
- Study completion
- Jun 2016 (actual)
- 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.
evaluate if beta 2-adrenergic agonist can have adjuvant effect to patients with infantile-onset Pompe disease under enzyme replacement therapy
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Salbutamol | Experimental | Drug: Salbutamol1. age 2-6 year: 0.1mg/kg tid x 2 weeks, then gradually increase to 0.2mg/kg tid (daily total maximal12mg) 2. age 6-12 year: 2mg tid x 2 weeks, then gradually increase to 4mg tid (daily total maximal 24mg) 3. age 12 year and above: 4mg tidx 2 weeks, then gradually increase to 8mg tid (daily total maximal 32mg) |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Confirm diagnosis as Pompe disease
- age 2 years or above
- under at least 1 year regular recombinant human acid alpha glucosidase, and no dosage/frequency change in recent 3 months
- Provide inform consent
Key exclusion criteria (8 listed)
- chronic heart disease such as arrhythmia, cardiomyopathy, cardiac infarction
- history of seizure
- history of diabetes mellitus
- history of hyperthyroidism
- hypokalemia
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 |
|---|---|---|
| Salbutamol1. Albuterol Syrup (1mL= Salbutamol 0.4mg, 60ml/bot) 2. VENTOLIN TABLETS 2MG (SALBUTAMOL SULPHATE) | Drug | Salbutamol |
Primary outcome
10% decrement of creatinine kinase
Time frame: 6 months
measure and compare serum creatinine kinase levels
Improvement in 6-minutes walk test
Time frame: 6 months
measure and compare 6-minutes walk test
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Sep 2016.
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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 Salbutamol 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 8 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: finished, as of the sponsor's last update on 20 Sep 2016. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
10% decrement of creatinine kinase (time frame: 6 months). The study lists 2 primary outcomes in all. 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 20 Sep 2016.