Glycogen Storage Disease Type IINCT02761421
Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT
An observational study of Glycogen Storage Disease Type II, run by Taipei Veterans General Hospital, Taiwan at 1 site in Taiwan.
Trial summary
To investigate the motor development, motor function and electrodiagnostics presentation in IOPD under ERT. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Glycogen Storage Disease Type II
- What is being tested?
- observation study
- How many people
- 15 participants
- Where
- 1 siteTaiwan
- Study dates
- Jan 2016to Dec 2016
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The investigators included all patients with infantile onset Pompe disease (IOPD) who were diagnosed and follow-up at Taipei Veteran General Hospital (TVGH) and excluded patients whose guardian did not provide the inform consent.
Who may be able to join
- All patients with infantile onset Pompe disease (IOPD) who were diagnosed and follow-up at Taipei Veteran General Hospital (TVGH).
Who cannot join
- Patients whose guardian did not provide the inform consent.
Trial locations and contacts
1 site in 1 country.
Taiwan1 site, 1 recruiting
- Recruiting
Taipei Veteran General Hospital : Taipei City, Taiwan 11217, R.O.C.
Taipei, 11217
chihjou lai, Master, 886-2-28712121 ext. 7384, irbopinion@vghtpe.gov.tw
About this study
Official title: Effect of Motor Development, Motor Function and Electrodiagnostic Characteristic of IOPD Under ERT
To investigate the motor development, motor function and electrodiagnostics presentation in IOPD under ERT.
Read the detailed description
Background
Pompe disease is an autosomal recessive disease, enzyme replacement therapy (ERT) and new born screen (NBS) had been initiated in Taiwan since 2008. However, residual muscle weakness were noted. Decrease in amplitude of nerve compound muscle action potential (CMAP) and increase spontaneous activity were reported before the imitation of ERT. However, the motor development, motor function and electrodiagnostic presentation which were important in further direction of treatment and rehabilitation program arrangement in infantile Pompe disease (IOPD) under ERT is lacking.
Aim
To investigate the motor development, motor function and electrodiagnostic presentation in IOPD under ERT.
Method
This is an observational, prospective, longitudinal, follow-up study. Motor development was assessed by Alberta Infant Motor Scale (AIMS) and Peabody Developmental Motor Scales, Second Edition (PDMS II); motor function was assessed by Pediatric Evaluation of Disability Inventory (PEDI) and Gross Motor Function Measure (GMFM). Electrodiagnosis studies include nerve conduction study (NCS) and electromyography (EMG).
Expect Effects
Obtain the characteristics of motor development, motor function and electrodiagnosis presentation of IOPD under ERT, and the relation between motor development, motor function and electrodiagnosis presentation.
- Conditions
- Glycogen Storage Disease Type II
- Being tested
- observation study
- Keywords
- IOPD
- 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.
observation studyOther
observation study
| Study group | Role | What they receive |
|---|---|---|
| patients with IOPD | observation all patients with IOPD Receives: Other: observation study |
What is being measured
The primary outcome is the main question the study is designed to answer.
Raw Score of Alberta Infant Motor Scale Development quotient of Peabody Developmental Motor Scales, Second Edition
Measured: 1 year
Development Quotient of PDMS-II
Measured: 1 year
Secondary outcomes (2)
Raw Score of Pediatric Evaluation of Disability Inventory Total score of GMFM
Measured: 1 year
Total Score of GMFM
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2016 (estimated)
- Study startJan 2016
- Primary completionDec 2016 (estimated)
- Study completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage Disease Type IIStudies of the same condition, open and under way.
- More trials run by Taipei Veterans General Hospital, TaiwanThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Taipei Veterans General Hospital, Taiwan
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02761421
- Sponsor's protocol ID
- 2015-12-014C
- Status last verified
- Dec 2015
- First submitted
- 26 Apr 2016
- Last updated
- 4 May 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: publication in journal
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Motor Development, Motor Function and Electrodiagnostic Characteristic of IOPD Under ERT
- ClinicalTrials.gov ID
- NCT02761421
- Acronym / sponsor ID
- 2015-12-014C
- Conditions
- Glycogen Storage Disease Type II
- Keywords
- IOPD
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Taipei Veterans General Hospital, TaiwanOther government
- Study start
- Jan 2016
- Primary completion
- Dec 2016 (estimated)
- Study completion
- Dec 2016 (estimated)
- First posted
- 4 May 2016 (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.
To investigate the motor development, motor function and electrodiagnostics presentation in IOPD under ERT.
Detailed description
Background
Pompe disease is an autosomal recessive disease, enzyme replacement therapy (ERT) and new born screen (NBS) had been initiated in Taiwan since 2008. However, residual muscle weakness were noted. Decrease in amplitude of nerve compound muscle action potential (CMAP) and increase spontaneous activity were reported before the imitation of ERT. However, the motor development, motor function and electrodiagnostic presentation which were important in further direction of treatment and rehabilitation program arrangement in infantile Pompe disease (IOPD) under ERT is lacking.
Aim
To investigate the motor development, motor function and electrodiagnostic presentation in IOPD under ERT.
Method
This is an observational, prospective, longitudinal, follow-up study. Motor development was assessed by Alberta Infant Motor Scale (AIMS) and Peabody Developmental Motor Scales, Second Edition (PDMS II); motor function was assessed by Pediatric Evaluation of Disability Inventory (PEDI) and Gross Motor Function Measure (GMFM). Electrodiagnosis studies include nerve conduction study (NCS) and electromyography (EMG).
Expect Effects
Obtain the characteristics of motor development, motor function and electrodiagnosis presentation of IOPD under ERT, and the relation between motor development, motor function and electrodiagnosis presentation.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients with IOPD | – | Other: observation studyobservation all patients with IOPD |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The investigators included all patients with infantile onset Pompe disease (IOPD) who were diagnosed and follow-up at Taipei Veteran General Hospital (TVGH) and excluded patients whose guardian did not provide the inform consent.
Key inclusion criteria (1 listed)
- All patients with infantile onset Pompe disease (IOPD) who were diagnosed and follow-up at Taipei Veteran General Hospital (TVGH).
Key exclusion criteria (1 listed)
- Patients whose guardian did not provide the inform consent.
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 |
|---|---|---|
| observation studyobservation study | Other | patients with IOPD |
Primary outcome
Raw Score of Alberta Infant Motor Scale Development quotient of Peabody Developmental Motor Scales, Second Edition
Time frame: 1 year
Development Quotient of PDMS-II
Time frame: 1 year
2 secondary outcomes
Raw Score of Pediatric Evaluation of Disability Inventory Total score of GMFM
Time frame: 1 year
Total Score of GMFM
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2016.
- Individual participant dataSharing planned: Yes. publication in journal
Related trials for Glycogen Storage Disease Type II
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Recruiting
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingObservational study
Pompe Disease Registry Protocol
Glycogen Storage Disease Type II, Pompe Disease
Not yet recruitingObservational study
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Glycogen Storage Disease Type II, run by Taipei Veterans General Hospital, Taiwan 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 1 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 4 May 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.
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 chihjou lai (886-28712121 ext. 7384, irbopinion@vghtpe.gov.tw), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Raw Score of Alberta Infant Motor Scale Development quotient of Peabody Developmental Motor Scales, Second Edition (time frame: 1 year). 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 4 May 2016; AzTrials last refreshed its copy on 6 Nov 2026.