The effect of trehalose on patients with lysosomal storage diseases

Investigation of the effects of trehalose on quality of life status and paraclinical findings of patients with lysosomal storage diseases (Niemann-Pick, and Mucopolysaccharidosis type III/Sanfilippo syndrome)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

IRCT

Registry ID

IRCT20201129049534N10

Enrollment

Registered

2024-12-28

Start date

2025-02-08

Completion date

Unknown

Last updated

2025-01-08

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

Conditions

Condition 1: Mucopolysaccharidosis type III/Sanfilippo syndrome. Condition 2: Niemann-Pick. Sanfilippo mucopolysaccharidoses Niemann-Pick disease

Interventions

Intervention group: Intravenous (IV) administration of Trehalose 15 gr per week for 12 weeks.

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients with lysosomal storage disorders (Niemann-Pick and Sanfilippo syndrome)

Exclusion criteria

Exclusion criteria: Special medical conditions

Design outcomes

Primary

Measure	Time frame
Assessment of quality of life. 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 Ped.QL and/or SF-36 questionnaire.	—

Secondary

Measure	Time frame
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.;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.;Alanine aminotransferase (ALT). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Aspartate aminotransferase (AST). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Alkaline Phosphatase (ALP). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Total bilirubin. Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Blood urea nitrogen (BUN). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: ELISA test.;Creatinine. Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: ELISA test.;Lactate Dehydrogenase (LDH). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Serum concentrations of heparan sulfate. Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: ELISA test.;Serum concentrations of lyso-sphingomyelin. Timepoint: A before-and-after study (At the beginning and end of the	—

Measure

Time frame

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.;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.;Alanine aminotransferase (ALT). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Aspartate aminotransferase (AST). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Alkaline Phosphatase (ALP). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Total bilirubin. Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Blood urea nitrogen (BUN). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: ELISA test.;Creatinine. Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: ELISA test.;Lactate Dehydrogenase (LDH). Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: Enzymatic photometric method.;Serum concentrations of heparan sulfate. Timepoint: A before-and-after study (At the beginning and end of the intervention trial (Day 0 and week 12). Method of measurement: ELISA test.;Serum concentrations of lyso-sphingomyelin. Timepoint: A before-and-after study (At the beginning and end of the

—

Countries

Iran (Islamic Republic of)

Contacts

Public ContactDr. Mohammad Bagherniya

Esfahan University of Medical Sciences

bagherniya@nutr.mui.ac.ir+98 31 3972 3154

Outcome results

None listed

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