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

• Status: Completed
• Trial end date: 2009-04-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The Hospital for Sick Children
• Collaborator: Actelion
• Treatments: 1-Deoxynojirimycin; Miglustat

Criteria

Inclusion Criteria:

• 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 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.