Investigation of the therapeutic effects of Trehalose in lysosomal storage diseases (Niemann-Pick types A & B, and Mucopolysaccharidosis type III/Sanfilippo syndrome)
Reduction of Symptoms in LSD patients by Trehalose therapeutic effect
Design
This study has been designed as a non randomized controlled trial without control group and placebo.
Settings and conduct
Endocrinology and metabolism research institute, Imam Reza hospital
Participants/Inclusion and exclusion criteria
Patients with definitive diagnosis of Nieman-Pick and Sanfilippo syndrome
Intervention groups
Intravenous (IV) administration of Trehalose for 12 weeks in the intervention group
Main outcome variables
1-Psychopathology and behavioral assessment. 2-biochemical assays to evaluate the levels of serum enzymes such as sphyngomyelinase. 3-serum and urinary concentrations of glycosaminoglycans (GAGs) such as heparan sulfate. 4-Sonographic assessment of liver and spleen size
General information
Reason for update
Acronym
Trehalose Therapeutic Effects in Lysosomal Storage Diseases (TTELSD
IRCT registration information
IRCT registration number:IRCT20130829014521N16
Registration date:2020-03-03, 1398/12/13
Registration timing:prospective
Last update:2020-03-03, 1398/12/13
Update count:1
Registration date
2020-03-03, 1398/12/13
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
Recruitment complete
Funding source
Expected recruitment start date
2020-06-20, 1399/03/31
Expected recruitment end date
2021-02-18, 1399/11/30
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Investigation of the therapeutic effects of Trehalose in lysosomal storage diseases (Niemann-Pick types A & B, and Mucopolysaccharidosis type III/Sanfilippo syndrome)
Public title
Trehalose and lysosomal storage diseases
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Patients with lysosomal storage disorders (Niemann-Pick type A & B and Sanfilippo syndrome)
Exclusion criteria:
Age
No age limit
Gender
Both
Phase
3
Groups that have been masked
No information
Sample size
Target sample size:
10
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 of Mashhad University of Medical Sciences
Street address
Vice Chancellor for Research, Mashhad University of Medical Sciences, Ghoreishi building, Daneshgah Street
City
Mashhad
Province
Razavi Khorasan
Postal code
9138813944
Approval date
2019-12-06, 1398/09/15
Ethics committee reference number
IR.MUMS.REC.1398.257
Health conditions studied
1
Description of health condition studied
Mucopolysaccharidosis type III/Sanfilippo syndrome
ICD-10 code
E76.22
ICD-10 code description
Sanfilippo mucopolysaccharidoses
2
Description of health condition studied
Niemann-Pick types A & B
ICD-10 code
E75.24
ICD-10 code description
Niemann-Pick disease
Primary outcomes
1
Description
Assessment of liver and spleen size
Timepoint
A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12)
Method of measurement
Liver and spleen size can be evaluated with sonography
2
Description
Psychopathology and behavioral assessment
Timepoint
A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12)
Method of measurement
Filling out the questionnaire
Secondary outcomes
1
Description
Evaluation of plasma sphingomyelin levels
Timepoint
A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12)
Method of measurement
Sphingomyelin assay kit
2
Description
Serum and urinary concentrations of heparan sulfate and glycosaminoglycans (GAGs)
Timepoint
A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12)
Method of measurement
Sphingomyelin and glycosaminoglycans (GAGs) assay kit
Intervention groups
1
Description
Intervention group: Intravenous (IV) administration of Trehalose for 12 weeks
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Endocrinology and metabolism research institute, Akbar hospital
Full name of responsible person
Dr.Amirhossein Sahebkar
Street address
Shahid Fakouri blvd.
City
Mashhad
Province
Razavi Khorasan
Postal code
٩١٩۵٩٨٣١٣۴
Phone
+98 51 3180 1000
Email
Sahebkara@mums.ac.ir
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
دکتر محسن تفقدی
Street address
Vice-Chancellor for Research, Mashhad University of Medical Sciences, Ghoreishi building, Daneshgah Street, Mashhad,
City
Mashhad
Province
Razavi Khorasan
Postal code
9138813944
Phone
+98 51 3841 1538
Email
vcresraech@mums.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Mashhad University of Medical Sciences
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
Mashhad University of Medical Sciences
Full name of responsible person
Dr.Amirhossein Sahebkar
Position
Associate professor
Latest degree
Ph.D.
Other areas of specialty/work
Pharmaceutical biotechnology
Street address
School of Pharmacy, East door of Ferdowsi University, Azadi Square, Mashhad
City
Tehran
Province
Tehran
Postal code
9177899191
Phone
+98 51 1882 9260
Fax
+98 51 1882 3251
Email
sahebkara@mums.ac.ir
Person responsible for scientific inquiries
Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Dr. Amirhossein Sahebkar
Position
Associate professor
Latest degree
Ph.D.
Other areas of specialty/work
pharmaceutical biotechnology
Street address
School of Medicine, East door of Ferdowsi University, Azadi square, Mashhad
City
Mashhad
Province
Razavi Khorasan
Postal code
9177899191
Phone
+98 51 1800 2288
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
Dr. Amirhossein Sahebkar
Position
Associate professor
Latest degree
Ph.D.
Other areas of specialty/work
pharmaceutical biotechnology
Street address
School of Medicine, East door of Ferdowsi University, Azadi square, Mashhad
City
Mashhad
Province
Razavi Khorasan
Postal code
9177899191
Phone
+98 51 1800 2288
Email
sahebkara@mums.ac.ir
Sharing plan
Deidentified Individual Participant Data Set (IPD)
No - There is not a plan to make this available
Justification/reason for indecision/not sharing IPD
"There is no further information"
Study Protocol
No - There is not a plan to make this available
Statistical Analysis Plan
No - There is not a plan to make this available
Informed Consent Form
No - There is not 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