Investigation of the effects of trehalose on quality of life of patients with lysosomal storage disorders

Phase 2

• Conditions: Condition 1: Niemann-Pick. Condition 2: Mucopolysaccharidosis type III (Sanfilippo).; Other sphingolipidosis; Other mucopolysaccharidoses; E75.2; E76.2

• Registration Number: IRCT20130829014521N23
• Lead Sponsor: Vice-chancellor of research and technology of the Ministry of health and medical education

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 6 March 2024

Recruitment & Eligibility

• Status: Pending
• Sex: All
• Target Recruitment: 40

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

Study & Design

• Study Type: interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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.