Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis

• Register: ClinicalTrials.gov
• Last refreshed on: 30 May 2016
• Main ID: NCT00418847
• Date of registration: 04/01/2007
• Prospective Registration: No
• Primary sponsor: The Hospital for Sick Children
• Public title: Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
• Scientific title: Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis: Single and Multiple Oral Doses
• Date of first enrolment: July 2004
• Target sample size: 5
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00418847
• Study type: Interventional
• Study design: Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Canada

Contacts

• Name: Joe TR Clarke, MD
• Affiliation: The Hospital for Sick Children, Toronto Canada

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of GM2 gangliosidosis confirmed by demonstration of profound deficiency of
ß-hexosaminidase A or A & B in peripheral blood leukocytes or cultured skin
fibroblasts

- Aged 6 to 20 years

- Onset of characteristic clinical symptoms of the disease before age 15 years

- Normal renal or hepatic function

Exclusion Criteria:

- Fertile patients who do not agree to use adequate contraception throughout the study
and for 3 months after cessation of miglustat treatment.

- Patients who cannot tolerate the study procedures, cannot be compliant to therapy or
who are unable to travel to the study center as required by this protocol.

- Patients receiving other investigational agents within 3 months of study initiation.

- Patients with disease that may affect absorption or elimination of drugs.

- Patients suffering from clinically significant diarrhea (>3 liquid stools per day for
> 7 days) without definable cause within 3 months of baseline visit, or who have a
history of significant gastrointestinal disorders.

- Patients with swallowing difficulties.

- Patients with a high probability of dying during the study.

- Patients who in the opinion of the investigator (for whatever reason) are thought to
be unsuitable for the study.



Age minimum: 6 Years
Age maximum: 20 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Gangliosidoses GM2

Intervention(s)

Drug: miglustat

Primary Outcome(s)

Concentration of miglustat in plasma [Time Frame: Periodic intervals up to 24 hours]

Secondary Outcome(s)

Change in single-voxel N acetylaspartate (NAA) from baseline MRS [Time Frame: 1 month, 3 months, 6 months, 9 months, and 12 months]

Change in nerve conduction [Time Frame: 6 months and 12 months]

Change in neurological examination from baseline [Time Frame: 1 month, 3 months, 6 months, 9 months, and 12 months]

Change in neuropsychological testing from baseline [Time Frame: 6 months and 12 months]

Changes in volume loss and signal intensity from baseline MRI [Time Frame: 12 months]

Secondary ID(s)

1000004763

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Actelion