History
# Registration date Revision Id
2 2024-01-17, 1402/10/27 293614
1 2023-10-27, 1402/08/05 283215
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  • Protocol summary

    Investigation of the effects of trehalose on quality of life status and paraclinical findings of patients with lysosomal storage disorders (Niemann-Pick type A & B and mucopolysaccharidosis type III (Sanfilippo))
    Investigation of the effects of trehalose on quality of life status and paraclinical findings of patients with lysosomal storage disorders (Niemann-Pick and mucopolysaccharidosis type III (Sanfilippo))
    بررسی اثر ترهالوز بر کیفیت زندگی و یافته های آزمایشگاهی بیماران مبتلا به بیماریهای ذخیره ای لیزوزومی (نیمن پیک تیپ A و B و موکوپلی ساکاریدوز تیپ 3 (Sanfilippo))
    بررسی اثر ترهالوز بر کیفیت زندگی و یافته های آزمایشگاهی بیماران مبتلا به بیماریهای ذخیره ای لیزوزومی (نیمن پیک و موکوپلی ساکاریدوز تیپ 3 (Sanfilippo))
    Single-arm clinical trial (without control group), phase 2, on 50 patients
    Single-arm clinical trial (without control group), phase 2, on 40 patients
    کارازمایی بالینی یک گروهه بدون گروه کنترل، فاز2 روی 50 بیمار
    کارازمایی بالینی یک گروهه بدون گروه کنترل، فاز2 روی 40 بیمار
    Patients with Niemann-Pick type A & B or mucopolysaccharidosis type III (Sanfilippo) with mentioned inclusion criteria, will be enrolled to this study. The Trehalose (15 gr per week) will be administered intravenous (IV) for 12 weeks in Akbar pediatric Hospital. The patients will be assessed before and after the intervention.
    Patients with Niemann-Pick or mucopolysaccharidosis type III (Sanfilippo) with mentioned inclusion criteria, will be enrolled to this study. The Trehalose (15 gr per week) will be administered intravenous (IV) for 12 weeks. The patients will be assessed before and after the intervention.
    بیماران با تشخیص قطعی نیمن پیک تیپ A و B ویا موکوپلی ساکاریدوز تیپ3(سندروم سن فیلیپو) که واجدکرایتریای ورود باشند، وارد مطالعه میشوند و به مدت 12هفته (3ماه) ترهالوز را بصورت وریدی با دوز 15 گرم در هفته در بیمارستان کودکان اکبر دریافت خواهند کرد. در ابتدا و انتهای مطالعه بیماران از نظر پیامدهایی که درادامه توضیح داده میشود تحت ارزیابی قرار میکیرند.
    بیماران با تشخیص قطعی نیمن پیک ویا موکوپلی ساکاریدوز تیپ3(سندروم سن فیلیپو) که واجدکرایتریای ورود باشند، وارد مطالعه میشوند و به مدت 12هفته (3ماه) ترهالوز را بصورت وریدی با دوز 15 گرم در هفته دریافت خواهند کرد. در ابتدا و انتهای مطالعه بیماران از نظر پیامدهایی که درادامه توضیح داده میشود تحت ارزیابی قرار میکیرند.
    Inclusion Criteria: Patients with Niemann-Pick type A & B or mucopolysaccharidosis type III (Sanfilippo) that sign informed consent (For children, their parents or legal guardians should sign informed consent).
    Inclusion Criteria: Patients with Niemann-Pick or mucopolysaccharidosis type III (Sanfilippo) that sign informed consent (For children, their parents or legal guardians should sign informed consent).
    شرایط ورود: بیماران نیمن پیک تیپ A و B ویا موکوپلی ساکاریدوز تیپ3(سندروم سن فیلیپو) که خودشان یا والدین یا قیم قانونی انها (درمورد کودکان) رضایت جهت شرکت در مطالعه داشته باشد.
    شرایط ورود: بیماران نیمن پیک ویا موکوپلی ساکاریدوز تیپ3(سندروم سن فیلیپو) که خودشان یا والدین یا قیم قانونی انها (درمورد کودکان) رضایت جهت شرکت در مطالعه داشته باشد.
  • General information

    50
    40
    2024-01-21, 1402/11/01
    2024-04-08, 1403/01/20
    2026-01-21, 1404/11/01
    2026-04-09, 1405/01/20
    empty
    Due to the rarity of the disease and death of some of previously identified patients (suffering from San Filippo or Niemann-Pick syndrome), the sample size is reduced. All types of Niemann-Pick and mucopolysaccharidosis type III (Sanfilippo) will be entered to this trial.
    empty
    با توجه به نادر بودن بیماری و فوت تعدادی از بیماران شناسایی شده (مبتلا به سندرم سن فیلیپو و یا نیمن پیک) حجم نمونه کاهش یافت. تمام تیپهای نیمن پیک و موکوپلی ساکاریدوز تیپ 3 وارد مطالعه خواهند شد.
    Investigation of the effects of trehalose on quality of life status and paraclinical findings of patients with lysosomal storage disorders (Niemann-Pick type A & B and mucopolysaccharidosis type III (Sanfilippo))
    Investigation of the effects of trehalose on quality of life status and paraclinical findings of patients with lysosomal storage disorders (Niemann-Pick and mucopolysaccharidosis type III (Sanfilippo))
    بررسی اثر ترهالوز بر کیفیت زندگی و یافته های آزمایشگاهی بیماران مبتلا به بیماریهای ذخیره ای لیزوزومی (نیمن پیک تیپ A و B و موکوپلی ساکاریدوز تیپ 3 (Sanfilippo))
    بررسی اثر ترهالوز بر کیفیت زندگی و یافته های آزمایشگاهی بیماران مبتلا به بیماریهای ذخیره ای لیزوزومی (نیمن پیک و موکوپلی ساکاریدوز تیپ 3 (Sanfilippo))
  • Recruitment centers

    #1
    Name of recruitment center - English: Akbar Hospital
    Name of recruitment center - Persian: بیمارستان اکبر
    Full name of responsible person - English: Amirhossein Sahebkar
    Full name of responsible person - Persian: امیرحسین صاحبکار
    Street address - English: Shahid Fakouri Blvd, Akbar Hospital
    Street address - Persian: بلوار شهید فکوری، بیمارستان اکبر
    City - English: Mashhad
    City - Persian: مشهد
    Province: Razavi Khorasan
    Country: Iran (Islamic Republic of)
    Postal code: ٩١٧٧٨٩٧١۵٧
    Phone: +98 51 3871 3801
    Fax:
    Email: sahebkara@mums.ac.ir
    Web page address:
    Name of recruitment center - English: Emam Hossein Children`s Hospital
    Name of recruitment center - Persian: بیمارستان کودکان امام حسین (ع)
    Full name of responsible person - English: Amirhossein Sahebkar
    Full name of responsible person - Persian: امیرحسین صاحبکار
    Street address - English: Emam Hossein Children`s Hospital, Km 10 Imam Khomeini Street
    Street address - Persian: خیابان امام خمینی، کیلومتر 10، بیمارستان کودکان حضرت امام حسین (ع)
    City - English: Isfahan
    City - Persian: اصفهان
    Province: Isfehan
    Country: Iran (Islamic Republic of)
    Postal code: 8195163381
    Phone: +98 31 3386 6266
    Fax:
    Email: sahebkara@mums.ac.ir
    Web page address:
  • Sponsors / Funding sources

    #1

    Name of organization / entity - English:
    Name of organization / entity - Persian:
    Full name of responsible person - English: Mohsen Tafaghodi
    Full name of responsible person - Persian: محسن تفقدی
    Street address - English: Ghoreshi Building, Daneshgah Street
    Street address - Persian: خیابان دانشگاه، ساختمان قرشی
    City - English: Mashhad
    City - Persian: مشهد
    Province: Razavi Khorasan
    Country: Iran (Islamic Republic of)
    Postal code: 9138813944
    Phone: +98 51 3841 2081
    Fax:
    Email: vcresearch@mums.ac.ir
    Web page address:
    contact.organization_id:
    Name of organization / entity - English: Vice-chancellor of research and technology of the Ministry of health and medical education
    Name of organization / entity - Persian: معاونت تحقیقات و فناوری وزارت بهداشت
    Full name of responsible person - English: Yunes Panahi
    Full name of responsible person - Persian: یونس پناهی
    Street address - English: Ministry of Health and Medical Education, Eivanak Blvd., Farahzadi Blvd., Qods Town
    Street address - Persian: شهرک قدس، بلوار فرحزادی، بلوار ایوانک، وزارت بهداشت، درمان و آموزش پزشکی
    City - English: Tehran
    City - Persian: تهران
    Province: Tehran
    Country: Iran (Islamic Republic of)
    Postal code: 1467664961
    Phone: +98 21 8145 5211
    Fax:
    Email: info@hbi.ir
    Web page address: https://research.behdasht.gov.ir/
    Mashhad University of Medical Sciences
    Vice-chancellor of research and technology of the Ministry of health and medical education
    دانشگاه علوم پزشکی مشهد
    معاونت تحقیقات و فناوری وزارت بهداشت
  • Person responsible for general inquiries


    Name of organization / entity - English:
    Name of organization / entity - Persian:
    Full name of responsible person - English: Amirhossein Sahebkar
    Full name of responsible person - Persian: امیرحسین صاحبکار
    Position - English: Professor
    Position - Persian: استاد
    Latest degree: phd
    Area of specialty/work:
    Area of specialty/work title - English: Pharmaceutical biotechnology
    Area of specialty/work title - Persian: بیوتکنولوژی دارویی
    Street address - English: School of Pharmacy, University Campus, Azadi Square
    Street address - Persian: میدان آزادی، پردیس دانشگاه، دانشکده داروسازی
    City - English: Mashhad
    City - Persian: مشهد
    Province: Razavi Khorasan
    Province - English:
    Province - Persian:
    contact.provinces_available: 1
    Country: Iran (Islamic Republic of)
    Postal code: 9177948954
    Phone: +98 51 3180 1100
    Mobile: +98 915 122 1496
    Fax:
    Email: sahebkara@mums.ac.ir
    Web page address:

    Name of organization / entity - English:
    Name of organization / entity - Persian:
    Full name of responsible person - English: Mohammad Baghernia
    Full name of responsible person - Persian: محمد باقرنیا
    Position - English: Assistant Professor
    Position - Persian: استادیار
    Latest degree: phd
    Area of specialty/work: 34
    Area of specialty/work title - English:
    Area of specialty/work title - Persian:
    Street address - English: Department of Community Nutrition, School of Nutrition and Food Sciences, Isfahan University of Medical Sciences, Hezar Jerib Avenue
    Street address - Persian: خیابان هزارجریب، دانشگاه علوم پزشکی اصفهان، دانشکده تغذیه و علوم غذایی، گروه تغذیه جامعه
    City - English: Isfahan
    City - Persian: اصفهان
    Province: Isfehan
    Province - English:
    Province - Persian:
    contact.provinces_available: 1
    Country: Iran (Islamic Republic of)
    Postal code: 8174673461
    Phone: +98 31 3792 3154
    Mobile: +98 913 210 1627
    Fax:
    Email: Bagherniya@yahoo.com
    Web page address:

Protocol summary

Study aim
Investigation of the effects of trehalose on quality of life status and paraclinical findings of patients with lysosomal storage disorders (Niemann-Pick and mucopolysaccharidosis type III (Sanfilippo))
Design
Single-arm clinical trial (without control group), phase 2, on 40 patients
Settings and conduct
Patients with Niemann-Pick or mucopolysaccharidosis type III (Sanfilippo) with mentioned inclusion criteria, will be enrolled to this study. The Trehalose (15 gr per week) will be administered intravenous (IV) for 12 weeks. The patients will be assessed before and after the intervention.
Participants/Inclusion and exclusion criteria
Inclusion Criteria: Patients with Niemann-Pick or mucopolysaccharidosis type III (Sanfilippo) that sign informed consent (For children, their parents or legal guardians should sign informed consent).
Intervention groups
Intervention group: IV Trehalose 15 gr per week for 12 weeks made by Dr. Rajabi Pharmaceutical Co.
Main outcome variables
Primary Outcome: Quality of life (by Peds.QL in <18 and SF-36 for those over the age of 18). Secondary outcomes: The size of liver and spleen, Plasma Sphingomyelin Level, Serum heparan sulfate level, Heparan sulfate and glycosaminoglycans levels in the urine, Lung HRCT, CBC, Liver enzymes (AST, ALT), LDH, Creatinine.

General information

Reason for update
Due to the rarity of the disease and death of some of previously identified patients (suffering from San Filippo or Niemann-Pick syndrome), the sample size is reduced. All types of Niemann-Pick and mucopolysaccharidosis type III (Sanfilippo) will be entered to this trial.
Acronym
IRCT registration information
IRCT registration number: IRCT20130829014521N23
Registration date: 2023-10-27, 1402/08/05
Registration timing: prospective

Last update: 2024-02-04, 1402/11/15
Update count: 1
Registration date
2023-10-27, 1402/08/05
Registrant information
Name
Amirhossein Sahebkar
Name of organization / entity
Mashhad University of Medical Sciences
Country
Iran (Islamic Republic of)
Phone
+98 51 1882 9260
Email address
sahebkara@mums.ac.ir
Recruitment status
recruiting
Funding source
Expected recruitment start date
2024-04-08, 1403/01/20
Expected recruitment end date
2026-04-09, 1405/01/20
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Investigation of the effects of trehalose on quality of life status and paraclinical findings of patients with lysosomal storage disorders (Niemann-Pick and mucopolysaccharidosis type III (Sanfilippo))
Public title
Investigation of the effects of trehalose on quality of life of patients with lysosomal storage disorders
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Patients with Niemann-Pick and mucopolysaccharidosis type III Informed consents of the patients or their parents(for children)
Exclusion criteria:
Special medical conditions
Age
No age limit
Gender
Both
Phase
2
Groups that have been masked
No information
Sample size
Target sample size: 40
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
Research Ethics Committees of Mashhad University of Medical Sciences
Street address
Ghoreshi Building, Daneshgah Street
City
Mashhad
Province
Razavi Khorasan
Postal code
9138813944
Approval date
2023-09-23, 1402/07/01
Ethics committee reference number
IR.MUMS.REC.1402.213

Health conditions studied

1

Description of health condition studied
Niemann-Pick
ICD-10 code
E75.2
ICD-10 code description
Other sphingolipidosis

2

Description of health condition studied
Mucopolysaccharidosis type III (Sanfilippo)
ICD-10 code
E76.2
ICD-10 code description
Other mucopolysaccharidoses

Primary outcomes

1

Description
Quality of life
Timepoint
Baseline and after 12 weeks
Method of measurement
Peds-ql questionnaire and SF-36 will be fulfilled for those under and over 18, respectively.

Secondary outcomes

1

Description
The size of liver and spleen
Timepoint
Baseline and after 12 weeks
Method of measurement
Sonography

2

Description
Plasma Sphingomyelin Level
Timepoint
Baseline and after 12 weeks
Method of measurement
Blood test

3

Description
Serum heparan sulfate level
Timepoint
Baseline and after 12 weeks
Method of measurement
Blood test

4

Description
Heparan sulfate and glycosaminoglycans levels in the urine
Timepoint
Baseline and after 12 weeks
Method of measurement
Urine Analysis

5

Description
Lung HRCT (High-resolution computed tomography)
Timepoint
Baseline and after 12 weeks
Method of measurement
HRCT

6

Description
Complete Blood Count (CBC)
Timepoint
Baseline and after 12 weeks
Method of measurement
Blood test

7

Description
Liver enzymes including glutamic-oxaloacetic transaminase (SGOT) and Serum glutamic pyruvic transaminase (SGPT)
Timepoint
Baseline and after 12 weeks
Method of measurement
Blood test

8

Description
Creatinine
Timepoint
Baseline and after 12 weeks
Method of measurement
Blood test

9

Description
Lactate dehydrogenase (LDH)
Timepoint
Baseline and after 12 weeks
Method of measurement
Blood test

Intervention groups

1

Description
Intervention group: IV Trehalose 15 gr per week for 12 weeks
Category
Treatment - Drugs

Recruitment centers

1

Recruitment center
Name of recruitment center
Emam Hossein Children`s Hospital
Full name of responsible person
Amirhossein Sahebkar
Street address
Emam Hossein Children`s Hospital, Km 10 Imam Khomeini Street
City
Isfahan
Province
Isfehan
Postal code
8195163381
Phone
+98 31 3386 6266
Email
sahebkara@mums.ac.ir

Sponsors / Funding sources

1

Sponsor
Name of organization / entity
Vice-chancellor of research and technology of the Ministry of health and medical education
Full name of responsible person
Yunes Panahi
Street address
Ministry of Health and Medical Education, Eivanak Blvd., Farahzadi Blvd., Qods Town
City
Tehran
Province
Tehran
Postal code
1467664961
Phone
+98 21 8145 5211
Email
info@hbi.ir
Web page address
https://research.behdasht.gov.ir/
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Vice-chancellor of research and technology of the Ministry of health and medical education
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
Esfahan University of Medical Sciences
Full name of responsible person
Mohammad Baghernia
Position
Assistant Professor
Latest degree
Ph.D.
Other areas of specialty/work
Nutrition
Street address
Department of Community Nutrition, School of Nutrition and Food Sciences, Isfahan University of Medical Sciences, Hezar Jerib Avenue
City
Isfahan
Province
Isfehan
Postal code
8174673461
Phone
+98 31 3792 3154
Email
Bagherniya@yahoo.com

Person responsible for scientific inquiries

Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Amirhossein Sahebkar
Position
Professor
Latest degree
Ph.D.
Other areas of specialty/work
Pharmaceutical biotechnology
Street address
School of Pharmacy, University Campus, Azadi Square
City
Mashhad
Province
Razavi Khorasan
Postal code
9177948954
Phone
0098513180110
Email
sahebkara@mums.ac.ir

Person responsible for updating data

Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Amirhossein Sahebkar
Position
Professor
Latest degree
Ph.D.
Other areas of specialty/work
Pharmaceutical biotechnology
Street address
School of Pharmacy, University Campus, Azadi Square
City
Mashhad
Province
Razavi Khorasan
Postal code
9177948954
Phone
+98 51 3180 1100
Email
sahebkara@mums.ac.ir

Sharing plan

Deidentified Individual Participant Data Set (IPD)
Undecided - It is not yet known if there will be a plan to make this available
Study Protocol
Undecided - It is not yet known if there will be a plan to make this available
Statistical Analysis Plan
Undecided - It is not yet known if there will be a plan to make this available
Informed Consent Form
Undecided - It is not yet known if there will be 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
Undecided - It is not yet known if there will be a plan to make this available
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