Trehalose and lysosomal storage diseases

Phase 3

• Conditions: Condition 1: Mucopolysaccharidosis type III/Sanfilippo syndrome. Condition 2: Niemann-Pick types A & B.; Sanfilippo mucopolysaccharidoses; Niemann-Pick disease; E76.22; E75.24

• Registration Number: IRCT20130829014521N16
• Lead Sponsor: Mashhad University of Medical Sciences

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 22 February 2021

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Patients with lysosomal storage disorders (Niemann-Pick type A & B and Sanfilippo syndrome)

Exclusion Criteria

Not provided

Study & Design

• Study Type: interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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.;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 Outcome Measures

Name	Time	Method
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.;Serum 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.