Pump disease is a type 2 glycogen storage disorder caused by the deficiency of the alpha alpha-glucosidase enzyme. The disease is divided into two early and late categories according to the age at which symptoms begin. In this research, late-on patients are initiated, 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. In this study, patients with late pumped episodes treated with the enzyme alpha-glucosidase at 20 mg / kg every 14 days for 24 months and the effectiveness of treatment in patients with the study of skeletal, respiratory and cardiac systems and muscle and urinary enzymes Liver and psychological tests.
General information
Acronym
IRCT registration information
IRCT registration number:IRCT2017111120981N2
Registration date:2017-11-19, 1396/08/28
Registration timing:retrospective
Last update:
Update count:1
Registration date
2017-11-19, 1396/08/28
Registrant information
Name
Khadijeh Hajinaghitehrani
Name of organization / entity
Azad Medical University
Country
Iran (Islamic Republic of)
Phone
+98 21 2257 1228
Email address
khtehrani@iautmu.ac.ir
Recruitment status
Recruitment complete
Funding source
Deputy of Research of Islamic Azad University, Tehran Medical Sciences Branch
Expected recruitment start date
2014-03-05, 1392/12/14
Expected recruitment end date
2015-03-05, 1393/12/14
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Efficacy of enzyme replacement therapy with alpha-glucosidase in patient with late-onset Pompe disease
Public title
Efficacy of enzyme replacement therapy with alpha-glucosidase in patient with late-onset Pompe disease
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
1. Latent pump failure 2. No enzymatic treatment
Exclusion criteria:
1. Pregnancy 2. Unwillingness to participate in the study 3. Advanced liver and colic disease
Age
No age limit
Gender
Both
Phase
N/A
Groups that have been masked
No information
Sample size
Target sample size:
3
Randomization (investigator's opinion)
N/A
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Single
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics Committee on Research Islamic Azad University, Tehran Medical Branch
Street address
Khaghani Ave, Islamic Azad University, Tehran Medical Branch
City
Tehran
Postal code
Approval date
2016-02-24, 1394/12/05
Ethics committee reference number
IR.IAU.TMU.REC.1394.70
Health conditions studied
1
Description of health condition studied
Pompe
ICD-10 code
E74.0
ICD-10 code description
Glycogen storage disease
Primary outcomes
1
Description
Respiratory function
Timepoint
evry 6 months
Method of measurement
spirometery
2
Description
Muscular force
Timepoint
evry 6 months
Method of measurement
physical exam
Secondary outcomes
1
Description
Muscular Enzymes
Timepoint
evry 6 months
Method of measurement
lab data
Intervention groups
1
Description
20 mg of alpha-glucosidase enzyme every 14 days
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Bu-ali hospital
Full name of responsible person
Dr. Khadijeh Hajinaghi Tehrani
Street address
Damavand Ave
City
Tehran
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Research Deputy of Islamic Azad University, Tehran Medical Branch
Full name of responsible person
Elham Kohbor
Street address
Khaghani Ave, Islamic Azad University, Tehran Medical Branch
City
Tehran
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Research Deputy of Islamic Azad University, Tehran Medical Branch
Proportion provided by this source
100
Public or private sector
empty
Domestic or foreign origin
empty
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
empty
Person responsible for general inquiries
Contact
Name of organization / entity
Bu-ali Hospital
Full name of responsible person
Khadijeh Hajinaghi Tehrani
Position
Neurologist / Plan Designer
Other areas of specialty/work
Street address
Bu-ali Hospital
City
Tehran
Postal code
Phone
+98 912 149 3324
Fax
Email
Khtehrani@iautmu.ac.ir
Web page address
Person responsible for scientific inquiries
Contact
Name of organization / entity
Bu-ali Hospital
Full name of responsible person
Khadijeh Hajinaghi Tehrani
Position
Neurologist
Other areas of specialty/work
Street address
Bu-ali Hospital
City
Tehran
Postal code
Phone
+98 21 3334 8036
Fax
Email
Khtehrani@iautmu.ac.ir
Web page address
Person responsible for updating data
Contact
Name of organization / entity
Bu-ali Hospital
Full name of responsible person
Ehsan Jangholi
Position
MD
Other areas of specialty/work
Street address
Bu-ali Hospital
City
Tehran
Postal code
Phone
+98 21 3334 8036
Fax
Email
ehsanjangholi@yahoo.com
Web page address
Sharing plan
Deidentified Individual Participant Data Set (IPD)