Protocol summary
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Study aim
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The therapeutic effect and adverse event of enzyme replacement therapy
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Design
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In this research, late-onset Pumpe patients with clinical symptoms starting at adolescence, and initially as skeletal muscle weakness, and then as a result of conflicts in the heart and respiratory system, are investigated
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Settings and conduct
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20 mg of alpha-glucosidase enzyme every 14 days for 24 months
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Participants/Inclusion and exclusion criteria
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Inclusion criteria: 1. Latent pump failure 2. No enzymatic treatment Exclusion criteria: 1. Pregnancy 2. Unwillingness to participate in the study 3. Advanced liver disease and renal failure
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Intervention groups
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In this research, late-onset Pumpe patients with clinical symptoms starting at adolescence are investigated
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Main outcome variables
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Muscular Enzymes and Respiratory function
General information
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Reason for update
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Acronym
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IRCT registration information
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IRCT registration number:
IRCT2017111120981N2
Registration date:
2017-11-19, 1396/08/28
Registration timing:
retrospective
Last update:
2018-06-11, 1397/03/21
Update count:
1
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Registration date
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2017-11-19, 1396/08/28
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Registrant information
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Recruitment status
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Recruitment complete
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Funding source
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Deputy of Research of Islamic Azad University, Tehran Medical Sciences Branch
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Expected recruitment start date
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2014-03-05, 1392/12/14
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Expected recruitment end date
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2015-03-05, 1393/12/14
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Actual recruitment start date
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2014-03-11, 1392/12/20
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Actual recruitment end date
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2015-02-20, 1393/12/01
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Trial completion date
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empty
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Scientific title
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Efficacy of enzyme replacement therapy with alpha-glucosidase in patient with late-onset Pompe disease
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Public title
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Efficacy of enzyme replacement therapy with alpha-glucosidase in patient with late-onset Pompe disease
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Purpose
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Treatment
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Inclusion/Exclusion criteria
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Inclusion criteria:
Late-onset Pumpe disease
No received enzyme replacement therapy
Exclusion criteria:
Pregnancy
Unwillingness to participate in the study
Advanced liver disease and renal failure
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Age
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No age limit
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Gender
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Both
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Phase
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N/A
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Groups that have been masked
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No information
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Sample size
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Target sample size:
2
Actual sample size reached:
2
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Randomization (investigator's opinion)
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N/A
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Randomization description
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Blinding (investigator's opinion)
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Not blinded
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Blinding description
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Placebo
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Not used
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Assignment
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Single
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Other design features
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Ethics committees
1
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Ethics committee
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Approval date
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2016-02-24, 1394/12/05
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Ethics committee reference number
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IR.IAU.TMU.REC.1394.70
Health conditions studied
1
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Description of health condition studied
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Pompe
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ICD-10 code
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E74.0
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ICD-10 code description
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Glycogen storage disease
Primary outcomes
1
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Description
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Respiratory function
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Timepoint
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Every 6 months
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Method of measurement
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spirometery
2
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Description
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Muscular force
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Timepoint
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evry 6 months
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Method of measurement
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physical exam
Secondary outcomes
1
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Description
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Muscular Enzymes
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Timepoint
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evry 6 months
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Method of measurement
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lab data
Intervention groups
1
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Description
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20 mg of alpha-glucosidase enzyme every 14 days
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Category
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Treatment - Drugs
1
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Sponsor
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Grant name
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Grant code / Reference number
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Is the source of funding the same sponsor organization/entity?
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Yes
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Title of funding source
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Research Deputy of Islamic Azad University, Tehran Medical Branch
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Proportion provided by this source
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100
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Public or private sector
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Public
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Domestic or foreign origin
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Domestic
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Category of foreign source of funding
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empty
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Country of origin
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Type of organization providing the funding
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Academic
Sharing plan
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Deidentified Individual Participant Data Set (IPD)
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Yes - There is a plan to make this available
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Study Protocol
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Yes - There is a plan to make this available
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Statistical Analysis Plan
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Yes - There is a plan to make this available
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Informed Consent Form
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Yes - There is a plan to make this available
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Clinical Study Report
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Undecided - It is not yet known if there will be a plan to make this available
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Analytic Code
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Undecided - It is not yet known if there will be a plan to make this available
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Data Dictionary
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No - There is not a plan to make this available
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Title and more details about the data/document
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The only some part of data will be shared.
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When the data will become available and for how long
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5 months after publish
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To whom data/document is available
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only academic researcher
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Under which criteria data/document could be used
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By official permission
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From where data/document is obtainable
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by the corresponding author
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What processes are involved for a request to access data/document
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by the official request to the corresponding author, if she/he agrees to it.
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Comments
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