Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-07-31

Study Completion Date

2007-08-31

Brief Summary

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We want to see if Zavesca (or miglustat) is safe and can be tolerated by patients with acute infantile onset GM2 gangliosidosis - classical Tay-Sachs and infantile onset Sandhoff disease. We know that miglustat inhibits the formation of GM2 ganglioside, the compound that is stored in the brains of children with Tay-Sachs and Sandhoff disease. Since it inhibits the synthesis of ganglioside, miglustat may be able to reduce or delay the onset of clinical symptoms.

Detailed Description

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Specific Aims

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.

Conditions

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GM2 Gangliosidoses Tay-Sachs Sandhoff Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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Zavesca (Miglustat)

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. 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.
2. Onset of characteristic clinical symptoms of the disease before the age of 9 months.
3. Normal renal and hepatic function.
4. Written informed consent from parent or legal guardian.

Exclusion Criteria

1. 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.
2. Patients receiving other investigational agents within 3 months of study initiation.
3. Patients who are anemic (hemoglobin \< 11 g/dl, and/or hematocrit \< 34%)
4. 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.
5. Patients with a high probability of dying during the 6-month assessment period of the study.
6. Patients who in the opinion of the investigator (for whatever reason) are thought to be unsuitable for the study.

Minimum Eligible Age

6 Months

Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Principal Investigators

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Cynthia J TIfft, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Children's National Research Institute

Locations

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Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

Countries

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United States

Other Identifiers

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3445

Identifier Type: -

Identifier Source: org_study_id