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Efficacy of enzyme replacement therapy with alpha-glucosidase in patient with late-onset Pompe disease

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History
# Registration date Revision Id
2 2018-05-11, 1397/02/21 48855
1 2017-11-19, 1396/08/28 18508
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  • Protocol summary

    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.
    empty
    بیماری پمپه ,اختلال ذخیره گلیکوژن تیپ 2 می باشد که در اثر کمبود آنزیم آگلوکوزیدازآلفا ایجاد میگردد. این بیماری بر اساس سن شروع علائم به 2 دسته زودرس و دیررس تقسیم می گردد. در این تحقیق بیماران نوع دیررس تحت بررسی قرار می گیرند که علائم بالینی از سنین نوجوانی شروع شده و در ابتدا به صورت ضعف عضلات اسکلتی می باشد و سپس به صورت درگیری سیستم قلبی و تنفسی بروز می کند. در این تحقیق به بیماران مبتلا به نوع دیررس پمپه درمان جایگزین با آنزیم آگلوکوزیدازآلفا به میزان mg/kg20 هر 14 روز یکبار به مدت 24 ماه داده می شود و میزان اثربخشی درمان در بیماران با مطالعه ی سیستم اسکلتی,تنفسی و قلبی وآنزیم های عضلانی و کبدی و تست های روانشناختی بررسی می گردند.
    empty
    empty
    The therapeutic effect and adverse event of enzyme replacement therapy
    empty
    برررسی اثرات درمانی و عوارض جایگزینی آنزیمی
    empty
    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
    empty
    در این تحقیق بیماران نوع دیررس تحت بررسی قرار می گیرند که علائم بالینی از سنین نوجوانی شروع شده و در ابتدا به صورت ضعف عضلات اسکلتی می باشد و سپس به صورت درگیری سیستم قلبی و تنفسی بروز می کند.
    empty
    20 mg of alpha-glucosidase enzyme every 14 days for 24 months
    empty
    20 میلی گرم آنزیم آلفا گلوکزیداز هر 14 روز به مدت 24 ماه
    empty
    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
    empty
    شرایط ورود: 1. ابتلا به پمپه دیررس 2. عدم دریافت درمان های آنزیمی شرایط خروج از مطالعه: 1. بارداری 2. عدم تمایل به شرکت در مطالعه 3. ابتلا به بیماریهای کبدی و کلیوی پیشرفته
    empty
    In this research, late-onset Pumpe patients with clinical symptoms starting at adolescence are investigated
    empty
    در این تحقیق بیماران نوع دیررس تحت بررسی قرار می گیرند که علائم بالینی از سنین نوجوانی شروع شده
    empty
    Muscular Enzymes and Respiratory function
    empty
    سطح آنزیم های عضلانی و عملکرد تنفسی

  • General information

    3
    2
    empty
    2
    empty
    2014-03-11, 1392/12/20
    empty
    2015-02-20, 1393/12/01
    Late-onset Pumpe disease
    No received enzyme replacement therapy
    ابتلا به پمپه دیررس
    عدم دریافت درمان های آنزیمی
    Pregnancy
    Unwillingness to participate in the study
    Advanced liver disease and renal failure
    بارداری
    عدم تمایل به شرکت در مطالعه
    ابتلا به بیماریهای کبدی و کلیوی پیشرفته

  • Primary outcomes

    #1
    evry 6 months
    Every 6 months

  • Recruitment centers

    #1
    Name of recruitment center - English: Bu-ali hospital
    Name of recruitment center - Persian: بیمارستان بوعلی
    Full name of responsible person - English: Dr. Khadijeh Hajinaghi Tehrani
    Full name of responsible person - Persian: دکتر خدیجه حاجی نقی تهرانی
    Street address - English: Damavand Ave
    Street address - Persian: خیابان دماوند
    City - English: Tehran
    City - Persian: تهران
    Province:
    Country: Iran (Islamic Republic of)
    Postal code:
    Phone:
    Fax:
    Email:
    Web page address:
    Name of recruitment center - English: Bu-ali hospital
    Name of recruitment center - Persian: بیمارستان بوعلی
    Full name of responsible person - English: Dr. Khadijeh Hajinaghi Tehrani
    Full name of responsible person - Persian: دکتر خدیجه حاجی نقی تهرانی
    Street address - English: Damavand Ave
    Street address - Persian: خیابان دماوند
    City - English: Tehran
    City - Persian: تهران
    Province: Tehran
    Country: Iran (Islamic Republic of)
    Postal code: 19395-1495
    Phone: +98 21 2200 6660
    Fax:
    Email: dr_tehrani10@yahoo.com
    Web page address:

  • Sponsors / Funding sources

    #1
    contact.organization_id:
    Name of organization / entity - English: Research Deputy of Islamic Azad University, Tehran Medical Branch
    Name of organization / entity - Persian: معاونت پژوهشی دانشگاه آزاد اسلامی واحد تهران پزشکی
    Full name of responsible person - English: Elham Kohbor
    Full name of responsible person - Persian: خانم الهام كوه بر
    Street address - English: Khaghani Ave, Islamic Azad University, Tehran Medical Branch
    Street address - Persian: خیابان خاقانی- دانشگاه آزاد اسلامی واحد تهران پزشکی
    City - English: Tehran
    City - Persian: تهران
    Province:
    Country: Iran (Islamic Republic of)
    Postal code:
    Phone:
    Fax:
    Email:
    Web page address:
    contact.organization_id:
    Name of organization / entity - English: Research Deputy of Islamic Azad University, Tehran Medical Branch
    Name of organization / entity - Persian: معاونت پژوهشی دانشگاه آزاد اسلامی واحد تهران پزشکی
    Full name of responsible person - English: Elham Kohbor
    Full name of responsible person - Persian: خانم الهام كوه بر
    Street address - English: Khaghani Ave, Islamic Azad University, Tehran Medical Branch
    Street address - Persian: خیابان خاقانی- دانشگاه آزاد اسلامی واحد تهران پزشکی
    City - English: Tehran
    City - Persian: تهران
    Province: Tehran
    Country: Iran (Islamic Republic of)
    Postal code: 19395-1495
    Phone: +98 21 2200 6660
    Fax:
    Email: dr_tehrani10@yahoo.com
    Web page address:
    empty
    Public
    empty
    Domestic
    empty
    Academic

  • Person responsible for general inquiries

    contact.organization_id:
    Name of organization / entity - English: Bu-ali Hospital
    Name of organization / entity - Persian: بیمارستان بوعلی
    Full name of responsible person - English: Khadijeh Hajinaghi Tehrani
    Full name of responsible person - Persian: خدیجه حاجی نقی تهرانی
    Position - English: Neurologist / Plan Designer
    Position - Persian: متخصص نورولوژی/ مجری طرح
    Latest degree:
    Area of specialty/work: 0
    Area of specialty/work title - English:
    Area of specialty/work title - Persian:
    Street address - English: Bu-ali Hospital
    Street address - Persian: بیمارستان بوعلی
    City - English: Tehran
    City - Persian: تهران
    Province:
    Province - English:
    Province - Persian:
    contact.provinces_available: 1
    Country: Iran (Islamic Republic of)
    Postal code:
    Phone: +98 912 149 3324
    Mobile: 00
    Fax:
    Email: Khtehrani@iautmu.ac.ir
    Web page address:
    contact.organization_id:
    Name of organization / entity - English: Bu-ali Hospital
    Name of organization / entity - Persian: بیمارستان بوعلی
    Full name of responsible person - English: Khadijeh Hajinaghi Tehrani
    Full name of responsible person - Persian: خدیجه حاجی نقی تهرانی
    Position - English: Neurologist / Plan Designer
    Position - Persian: متخصص نورولوژی/ مجری طرح
    Latest degree: specialist
    Area of specialty/work: 86
    Area of specialty/work title - English:
    Area of specialty/work title - Persian:
    Street address - English: Bu-ali Hospital
    Street address - Persian: بیمارستان بوعلی
    City - English: Tehran
    City - Persian: تهران
    Province: Tehran
    Province - English:
    Province - Persian:
    contact.provinces_available: 1
    Country: Iran (Islamic Republic of)
    Postal code: 19395-1495
    Phone: +98 912 149 3324
    Mobile: +98 912 149 3324
    Fax:
    Email: Khtehrani@iautmu.ac.ir
    Web page address:

  • Person responsible for scientific inquiries

    contact.organization_id:
    Name of organization / entity - English: Bu-ali Hospital
    Name of organization / entity - Persian: بیمارستان بوعلی
    Full name of responsible person - English: Khadijeh Hajinaghi Tehrani
    Full name of responsible person - Persian: خدیجه حاجی نقی تهرانی
    Position - English: Neurologist
    Position - Persian: متخصص نورولوژی
    Latest degree:
    Area of specialty/work: 0
    Area of specialty/work title - English:
    Area of specialty/work title - Persian:
    Street address - English: Bu-ali Hospital
    Street address - Persian: بیمارستان بوعلی
    City - English: Tehran
    City - Persian: تهران
    Province:
    Province - English:
    Province - Persian:
    contact.provinces_available: 1
    Country: Iran (Islamic Republic of)
    Postal code:
    Phone: +98 21 3334 8036
    Mobile: +98 912 149 3324
    Fax:
    Email: Khtehrani@iautmu.ac.ir
    Web page address:
    contact.organization_id:
    Name of organization / entity - English: Bu-ali Hospital
    Name of organization / entity - Persian: بیمارستان بوعلی
    Full name of responsible person - English: Khadijeh Hajinaghi Tehrani
    Full name of responsible person - Persian: خدیجه حاجی نقی تهرانی
    Position - English: Neurologist
    Position - Persian: متخصص نورولوژی
    Latest degree: specialist
    Area of specialty/work: 86
    Area of specialty/work title - English:
    Area of specialty/work title - Persian:
    Street address - English: Bu-ali Hospital
    Street address - Persian: بیمارستان بوعلی
    City - English: Tehran
    City - Persian: تهران
    Province: Tehran
    Province - English:
    Province - Persian:
    contact.provinces_available: 1
    Country: Iran (Islamic Republic of)
    Postal code: 19395-1495
    Phone: +98 21 3334 8036
    Mobile: +98 912 149 3324
    Fax:
    Email: Khtehrani@iautmu.ac.ir
    Web page address:

  • Person responsible for updating data

    contact.organization_id:
    Name of organization / entity - English: Bu-ali Hospital
    Name of organization / entity - Persian: بیمارستان بوعلی
    Full name of responsible person - English: Ehsan Jangholi
    Full name of responsible person - Persian: احسان جانقلی
    Position - English: MD
    Position - Persian: دکتری حرفه ای
    Latest degree:
    Area of specialty/work: 0
    Area of specialty/work title - English:
    Area of specialty/work title - Persian:
    Street address - English: Bu-ali Hospital
    Street address - Persian: بیمارستان بوعلی
    City - English: Tehran
    City - Persian: تهران
    Province:
    Province - English:
    Province - Persian:
    contact.provinces_available: 1
    Country: Iran (Islamic Republic of)
    Postal code:
    Phone: +98 21 3334 8036
    Mobile: +98 912 248 2350
    Fax:
    Email: ehsanjangholi@yahoo.com
    Web page address:
    contact.organization_id:
    Name of organization / entity - English: Bu-ali Hospital
    Name of organization / entity - Persian: بیمارستان بوعلی
    Full name of responsible person - English: Ehsan Jangholi
    Full name of responsible person - Persian: احسان جانقلی
    Position - English: MD
    Position - Persian: دکتری حرفه ای
    Latest degree: med_doctor
    Area of specialty/work: 30
    Area of specialty/work title - English:
    Area of specialty/work title - Persian:
    Street address - English: Bu-ali Hospital
    Street address - Persian: بیمارستان بوعلی
    City - English: Tehran
    City - Persian: تهران
    Province: Tehran
    Province - English:
    Province - Persian:
    contact.provinces_available: 1
    Country: Iran (Islamic Republic of)
    Postal code: 19395-1495
    Phone: +98 21 3334 8036
    Mobile: +98 912 248 2350
    Fax:
    Email: ehsanjangholi@yahoo.com
    Web page address:

  • Sharing plan

    yes
    yes
    yes
    yes
    undecided
    undecided
    no
    empty
    The only some part of data will be shared.
    empty
    فقط بخشی از دیتا های افراد مورد مطالعه به اشتراک گذاشته خواهد شد.
    empty
    5 months after publish
    empty
    5 ماه پس از چاپ
    empty
    only academic researcher
    empty
    فقط محققین دانشگاهی
    empty
    By official permission
    empty
    با اجازه رسمی محقق
    empty
    by the corresponding author
    empty
    از طریق نویسنده مسول طرح
    empty
    by the official request to the corresponding author, if she/he agrees to it.
    empty
    از طریق نامه رسمی خطاب به نویسنده مسوول و در صورت موافقت

Protocol summary

Study aim
The therapeutic effect and adverse event of enzyme replacement therapy
Design
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
Settings and conduct
20 mg of alpha-glucosidase enzyme every 14 days for 24 months
Participants/Inclusion and 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 disease and renal failure
Intervention groups
In this research, late-onset Pumpe patients with clinical symptoms starting at adolescence are investigated
Main outcome variables
Muscular Enzymes and Respiratory function

General information

Reason for update
Acronym
IRCT registration information
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
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
2014-03-11, 1392/12/20
Actual recruitment end date
2015-02-20, 1393/12/01
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:
Late-onset Pumpe disease No received enzyme replacement therapy
Exclusion criteria:
Pregnancy Unwillingness to participate in the study Advanced liver disease and renal failure
Age
No age limit
Gender
Both
Phase
N/A
Groups that have been masked
No information
Sample size
Target sample size: 2
Actual sample size reached: 2
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
Province
Tehran
Postal code
19395-1495
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
Every 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
Province
Tehran
Postal code
19395-1495
Phone
+98 21 2200 6660
Email
dr_tehrani10@yahoo.com

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
Province
Tehran
Postal code
19395-1495
Phone
+98 21 2200 6660
Email
dr_tehrani10@yahoo.com
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
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic

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
Latest degree
Specialist
Other areas of specialty/work
Neurology
Street address
Bu-ali Hospital
City
Tehran
Province
Tehran
Postal code
19395-1495
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
Latest degree
Specialist
Other areas of specialty/work
Neurology
Street address
Bu-ali Hospital
City
Tehran
Province
Tehran
Postal code
19395-1495
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
Latest degree
Medical doctor
Other areas of specialty/work
General Practitioner
Street address
Bu-ali Hospital
City
Tehran
Province
Tehran
Postal code
19395-1495
Phone
+98 21 3334 8036
Fax
Email
ehsanjangholi@yahoo.com
Web page address

Sharing plan

Deidentified Individual Participant Data Set (IPD)
Yes - There is a plan to make this available
Study Protocol
Yes - There is a plan to make this available
Statistical Analysis Plan
Yes - There is a plan to make this available
Informed Consent Form
Yes - There is a plan to make this available
Clinical Study Report
Undecided - It is not yet known if there will be a plan to make this available
Analytic Code
Undecided - It is not yet known if there will be a plan to make this available
Data Dictionary
No - There is not a plan to make this available
Title and more details about the data/document
The only some part of data will be shared.
When the data will become available and for how long
5 months after publish
To whom data/document is available
only academic researcher
Under which criteria data/document could be used
By official permission
From where data/document is obtainable
by the corresponding author
What processes are involved for a request to access data/document
by the official request to the corresponding author, if she/he agrees to it.
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