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 type A & B and mucopolysaccharidosis type III (Sanfilippo))
Design
Single-arm clinical trial (without control group), phase 2, on 50 patients
Settings and conduct
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.
Participants/Inclusion and exclusion criteria
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).
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
Acronym
IRCT registration information
IRCT registration number:IRCT20130829014521N23
Registration date:2023-10-27, 1402/08/05
Registration timing:prospective
Last update:2023-10-27, 1402/08/05
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
2024-01-21, 1402/11/01
Expected recruitment end date
2026-01-21, 1404/11/01
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 type A & B 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:
50
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
Akbar Hospital
Full name of responsible person
Amirhossein Sahebkar
Street address
Shahid Fakouri Blvd, Akbar Hospital
City
Mashhad
Province
Razavi Khorasan
Postal code
٩١٧٧٨٩٧١۵٧
Phone
+98 51 3871 3801
Email
sahebkara@mums.ac.ir
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Mohsen Tafaghodi
Street address
Ghoreshi Building, Daneshgah Street
City
Mashhad
Province
Razavi Khorasan
Postal code
9138813944
Phone
+98 51 3841 2081
Email
vcresearch@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
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
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