Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
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|ClinicalTrials.gov Identifier: NCT00672022|
Recruitment Status : Completed
First Posted : May 6, 2008
Last Update Posted : May 6, 2008
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|Condition or disease||Intervention/treatment||Phase|
|GM2 Gangliosidoses Tay-Sachs Sandhoff Disease||Drug: Zavesca (Miglustat)||Phase 3|
The primary objective of the study is to investigate the pharmacokinetics of ZAVESCA® (miglustat, OGT918), when given as a single dose and at steady state, in infantile patients with GM2 gangliosidosis. The secondary objectives are to evaluate the tolerability and safety of single and multiple doses of miglustat and to monitor disease progression using physical and developmental assessments and disease-specific biomarkers.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset GM2 Gangliosidosis: Single and Steady State Oral Doses|
|Study Start Date :||July 2004|
|Actual Primary Completion Date :||August 2007|
|Actual Study Completion Date :||August 2007|
- Biomarkers (level of GM2 ganglioside, chitotriosidase activity, anti-GM2 antibodies) in plasma, serum and CSF will be measured at initial visit (run-in period), Week 13, and Week 25.
- Neurophysiologic Assessment - EEG and BEAR tests will be done at initial visit (run-in period), Week 13, and Week 25.
- Ophthalmology Assessment - comparision of the "cherry-red" macula changes will be made at initial visit (run-in period) and Week 25.
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|Ages Eligible for Study:||6 Months to 5 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Diagnosis of GM2 gangliosidosis, confirmed by demonstration of profound deficiency of -hexosaminidase A or A & B in peripheral blood leukocytes or in cultured skin fibroblasts, within the previous 1 year in non-bone marrow transplant recipients who are < 2 years of age, or prior to stem cell transplant in stably engrafted transplant patients who are < 5 years of age.
- Onset of characteristic clinical symptoms of the disease before the age of 9 months.
- Normal renal and hepatic function.
- Written informed consent from parent or legal guardian.
- Patients who are unable to comply with the study procedures of this protocol, including the refusal to swallow the food used to mask the taste of the study drug and whose parents are unwilling to administer the drug through a nasogastric or gastrostomy tube.
- Patients receiving other investigational agents within 3 months of study initiation.
- Patients who are anemic (hemoglobin < 11 g/dl, and/or hematocrit < 34%)
- Patients who have a history of significant gastrointestinal disorders, including clinically significant diarrhea (>3 liquid stools per day for > 7 days), without definable cause within 3 months of baseline visit.
- Patients with a high probability of dying during the 6-month assessment period of the study.
- Patients who in the opinion of the investigator (for whatever reason) are thought to be unsuitable for the study.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00672022
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010|
|Principal Investigator:||Cynthia J TIfft, MD, PhD||Children's National Research Institute|
|Other Study ID Numbers:||
|First Posted:||May 6, 2008 Key Record Dates|
|Last Update Posted:||May 6, 2008|
|Last Verified:||July 2005|
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