Protocol summary
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Study aim
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Reduction of Symptoms in LSD patients by Trehalose therapeutic effect
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Design
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This study has been designed as a non randomized controlled trial without control group and placebo.
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Settings and conduct
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Endocrinology and metabolism research institute, Imam Reza hospital
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Participants/Inclusion and exclusion criteria
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Patients with definitive diagnosis of Nieman-Pick and Sanfilippo syndrome
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Intervention groups
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Intravenous (IV) administration of Trehalose for 12 weeks in the intervention group
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Main outcome variables
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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
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Reason for update
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Acronym
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Trehalose Therapeutic Effects in Lysosomal Storage Diseases (TTELSD
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IRCT registration information
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IRCT registration number:
IRCT20130829014521N16
Registration date:
2020-03-03, 1398/12/13
Registration timing:
prospective
Last update:
2021-01-09, 1399/10/20
Update count:
1
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Registration date
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2020-03-03, 1398/12/13
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Registrant information
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Recruitment status
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Recruitment complete
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Funding source
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Expected recruitment start date
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2020-06-20, 1399/03/31
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Expected recruitment end date
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2021-02-18, 1399/11/30
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Actual recruitment start date
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empty
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Actual recruitment end date
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empty
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Trial completion date
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empty
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Scientific title
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Investigation of the therapeutic effects of Trehalose in lysosomal storage diseases (Niemann-Pick types A & B, and Mucopolysaccharidosis type III/Sanfilippo syndrome)
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Public title
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Trehalose and lysosomal storage diseases
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Purpose
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Treatment
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Inclusion/Exclusion criteria
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Inclusion criteria:
Patients with lysosomal storage disorders (Niemann-Pick type A & B and Sanfilippo syndrome)
Exclusion criteria:
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Age
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No age limit
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Gender
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Both
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Phase
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3
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Groups that have been masked
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No information
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Sample size
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Target sample size:
10
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Randomization (investigator's opinion)
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N/A
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Randomization description
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Blinding (investigator's opinion)
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Not blinded
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Blinding description
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Placebo
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Not used
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Assignment
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Single
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Other design features
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Ethics committees
1
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Ethics committee
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Approval date
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2019-12-06, 1398/09/15
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Ethics committee reference number
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IR.MUMS.REC.1398.257
Health conditions studied
1
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Description of health condition studied
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Mucopolysaccharidosis type III/Sanfilippo syndrome
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ICD-10 code
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E76.22
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ICD-10 code description
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Sanfilippo mucopolysaccharidoses
2
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Description of health condition studied
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Niemann-Pick types A & B
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ICD-10 code
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E75.24
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ICD-10 code description
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Niemann-Pick disease
Primary outcomes
1
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Description
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Assessment of liver and spleen size
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Timepoint
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A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12)
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Method of measurement
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Liver and spleen size can be evaluated with sonography
2
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Description
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Psychopathology and behavioral assessment
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Timepoint
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A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12)
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Method of measurement
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Filling out the questionnaire
Secondary outcomes
1
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Description
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Evaluation of plasma sphingomyelin levels
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Timepoint
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A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12)
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Method of measurement
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Sphingomyelin assay kit
2
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Description
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Serum concentrations of heparan sulfate and glycosaminoglycans (GAGs)
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Timepoint
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A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12)
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Method of measurement
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Sphingomyelin and glycosaminoglycans (GAGs) assay kit
Intervention groups
1
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Description
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Intervention group: Intravenous (IV) administration of Trehalose for 12 weeks
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Category
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Treatment - Drugs
1
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Sponsor
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Grant name
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Grant code / Reference number
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Is the source of funding the same sponsor organization/entity?
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Yes
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Title of funding source
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Mashhad University of Medical Sciences
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Proportion provided by this source
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100
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Public or private sector
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Public
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Domestic or foreign origin
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Domestic
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Category of foreign source of funding
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empty
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Country of origin
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Type of organization providing the funding
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Academic
Sharing plan
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Deidentified Individual Participant Data Set (IPD)
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No - There is not a plan to make this available
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Justification/reason for indecision/not sharing IPD
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"There is no further information"
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Study Protocol
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No - There is not a plan to make this available
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Statistical Analysis Plan
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No - There is not a plan to make this available
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Informed Consent Form
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No - There is not a plan to make this available
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Clinical Study Report
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Undecided - It is not yet known if there will be a plan to make this available
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Analytic Code
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Undecided - It is not yet known if there will be a plan to make this available
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Data Dictionary
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No - There is not a plan to make this available