Effects of Miglustat on systemic and neurological symptoms of Sandhoff and Tay-Sachs diseases

Survey of Miglustat therapeutic effects on neurological and systemic symptoms of infantile type of Sandhoff and Tay-Sachs diseases

Status

Recruiting

Phases

Phase 4

Study type

Interventional

Source

IRCT

Registry ID

IRCT20181023041435N1

Enrollment

Registered

2018-11-04

Start date

2018-11-22

Completion date

Unknown

Last updated

2019-01-14

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

Conditions

Condition 1: Sandhoff. Condition 2: Tay-Sachs. GM2 gangliosidosis GM2 gangliosidosis

Interventions

Intervention group: Cases of infantile form of Sandhoff and Tay-Sachs diseases.

Eligibility

Sex/Gender

All

Age

6 Months to 24 Months

Inclusion criteria

Inclusion criteria: Clinically and genetically confirmed cases of Sandhoff (SD)/Tay-Sachs (TSD) diseases Enrolled patients who are referred to Myelin Disorders Clinic, Children’s Medical Center with clinically suspicion to TSD and SD

Exclusion criteria

Exclusion criteria: Diarrhea Renal impairement Loss of follow up Other systemic diseases Concomitant drug therapy which may affect neurological system function

Design outcomes

Primary

Measure	Time frame
Hospitallization frequency. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: Checklist.;Pneumonia aspiration frequency. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: Checklist.;Seizure Frequency. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: checklist.;Nasogastric tube insertion. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: checklist.;Motor function. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: Gross Motor Function Classification System.;Quality of life. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: Infant/ Toddler Quality Of Life Quastionnaire.	—

Measure

Time frame

Hospitallization frequency. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: Checklist.;Pneumonia aspiration frequency. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: Checklist.;Seizure Frequency. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: checklist.;Nasogastric tube insertion. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: checklist.;Motor function. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: Gross Motor Function Classification System.;Quality of life. Timepoint: before intervention and 4, 8, 12 months after intervention and 1-year without intervention. Method of measurement: Infant/ Toddler Quality Of Life Quastionnaire.

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Countries

Iran (Islamic Republic of)

Contacts

Public ContactAlireza Tavassoli

Tehran University of Medical Sciences

alirezatavasoli236@gmail.com+98 21 6147 2751

Outcome results

None listed

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