Status:
COMPLETED
Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Lead Sponsor:
The Hospital for Sick Children
Collaborating Sponsors:
Actelion
Conditions:
Gangliosidoses GM2
Eligibility:
All Genders
6-20 years
Phase:
PHASE2
Brief Summary
The purpose of the study is to investigate the pharmacokinetics of Zavesca (miglustat, OGT918) when given as single and multiple doses in juvenile patients with GM2 gangliosidosis.
Detailed Description
The GM2 gangliosidoses are a group of neuro-degenerative lysosomal storage diseases resulting from accumulation of GM2 and related glycolipids in the central nervous system (CNS). Tay-Sachs and Sandho...
Eligibility Criteria
Inclusion
- Diagnosis of GM2 gangliosidosis confirmed by demonstration of profound deficiency of β-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
- 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.
Key Trial Info
Start Date :
July 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2009
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT00418847
Start Date
July 1 2004
End Date
April 1 2009
Last Update
May 19 2016
Active Locations (1)
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1
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8