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

Investigation of the effects of trehalose on quality of life status and paraclinical findings of patients with lysosomal storage disorders (Niemann-Pick and mucopolysaccharidosis type III (Sanfilippo))

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

IRCT

Registry ID

IRCT20130829014521N23

Enrollment

Registered

2023-10-27

Start date

2024-04-08

Completion date

Unknown

Last updated

2024-03-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Condition 1: Niemann-Pick. Condition 2: Mucopolysaccharidosis type III (Sanfilippo). Other sphingolipidosis Other mucopolysaccharidoses

Interventions

Intervention group: IV Trehalose 15 gr per week for 12 weeks.

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients with Niemann-Pick and mucopolysaccharidosis type III Informed consents of the patients or their parents(for children)

Exclusion criteria

Exclusion criteria: Special medical conditions

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
The size of liver and spleen. Timepoint: Baseline and after 12 weeks. Method of measurement: Sonography.;Plasma Sphingomyelin Level. Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Serum heparan sulfate level. Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Heparan sulfate and glycosaminoglycans levels in the urine. Timepoint: Baseline and after 12 weeks. Method of measurement: Urine Analysis.;Lung HRCT (High-resolution computed tomography). Timepoint: Baseline and after 12 weeks. Method of measurement: HRCT.;Complete Blood Count (CBC). Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Liver enzymes including glutamic-oxaloacetic transaminase (SGOT) and Serum glutamic pyruvic transaminase (SGPT). Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Creatinine. Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Lactate dehydrogenase (LDH). Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.	—

Measure

Time frame

The size of liver and spleen. Timepoint: Baseline and after 12 weeks. Method of measurement: Sonography.;Plasma Sphingomyelin Level. Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Serum heparan sulfate level. Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Heparan sulfate and glycosaminoglycans levels in the urine. Timepoint: Baseline and after 12 weeks. Method of measurement: Urine Analysis.;Lung HRCT (High-resolution computed tomography). Timepoint: Baseline and after 12 weeks. Method of measurement: HRCT.;Complete Blood Count (CBC). Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Liver enzymes including glutamic-oxaloacetic transaminase (SGOT) and Serum glutamic pyruvic transaminase (SGPT). Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Creatinine. Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.;Lactate dehydrogenase (LDH). Timepoint: Baseline and after 12 weeks. Method of measurement: Blood test.

—

Countries

Iran (Islamic Republic of)

Contacts

Public ContactMohammad Baghernia

Esfahan University of Medical Sciences

Bagherniya@yahoo.com+98 31 3792 3154

Outcome results

None listed

Source: IRCT (via WHO ICTRP) · Data processed: Feb 4, 2026