Trehalose and lysosomal storage diseases

Investigation of the therapeutic effects of Trehalose in lysosomal storage diseases (Niemann-Pick types A & B, and Mucopolysaccharidosis type III/Sanfilippo syndrome)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

IRCT

Registry ID

IRCT20130829014521N16

Enrollment

Registered

2020-03-03

Start date

2020-06-20

Completion date

Unknown

Last updated

2021-02-22

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 types A & B. Sanfilippo mucopolysaccharidoses Niemann-Pick disease

Interventions

Intervention group: Intravenous (IV) administration of Trehalose for 12 weeks.

Eligibility

Sex/Gender

All

Inclusion criteria

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

Exclusion criteria

Exclusion criteria:

Design outcomes

Primary

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.;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.	—

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.;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.

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Secondary

Measure	Time frame
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.	—

Measure

Time frame

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.

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Countries

Iran (Islamic Republic of)

Contacts

Public ContactDr.Amirhossein Sahebkar

Mashhad University of Medical Sciences

sahebkara@mums.ac.ir+98 51 1882 9260

Outcome results

None listed

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