NCT00418847

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jul 2004

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2004

Completed
2.5 years until next milestone

First Submitted

Initial submission to the registry

January 4, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

January 5, 2007

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2009

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2009

Completed
Last Updated

May 19, 2016

Status Verified

May 1, 2016

Enrollment Period

4.8 years

First QC Date

January 4, 2007

Last Update Submit

May 18, 2016

Conditions

Gangliosidoses GM2

Keywords

pediatricslysosomal storage diseasesGangliosidoses GM2Tay-Sachs diseaseSandhoff diseaseInfantile GM2-activator deficiencyMiglustat

Outcome Measures

Primary Outcomes (1)

  • Concentration of miglustat in plasma

    Periodic intervals up to 24 hours

Secondary Outcomes (5)

  • Changes in volume loss and signal intensity from baseline MRI

    12 months

  • Change in single-voxel N acetylaspartate (NAA) from baseline MRS

    1 month, 3 months, 6 months, 9 months, and 12 months

  • Change in neuropsychological testing from baseline

    6 months and 12 months

  • Change in nerve conduction

    6 months and 12 months

  • Change in neurological examination from baseline

    1 month, 3 months, 6 months, 9 months, and 12 months

Study Arms (1)

1

EXPERIMENTAL
Drug: miglustat

Interventions

miglustatDRUG

Target dose of 320 mg/m\^2/day (divided in 3 doses) will be based on the Body Surface Area (BSA). For children with a BSA \> 1.3, 200 mg TID will be administered. For children with a BSA of 0.8-1.3, 100 mg TID will be administered.

Also known as: Zavesca
1

Eligibility Criteria

Age6 Years - 20 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Related Publications (1)

  • Maegawa GH, Banwell BL, Blaser S, Sorge G, Toplak M, Ackerley C, Hawkins C, Hayes J, Clarke JT. Substrate reduction therapy in juvenile GM2 gangliosidosis. Mol Genet Metab. 2009 Sep-Oct;98(1-2):215-24. doi: 10.1016/j.ymgme.2009.06.005. Epub 2009 Jun 12.

    PMID: 19595619RESULT

MeSH Terms

Conditions

Gangliosidoses, GM2Lysosomal Storage DiseasesTay-Sachs DiseaseSandhoff Disease

Interventions

miglustat

Condition Hierarchy (Ancestors)

GangliosidosesSphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Study Officials

  • Joe TR Clarke, MD

    The Hospital for Sick Children, Toronto Canada

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 4, 2007

First Posted

January 5, 2007

Study Start

July 1, 2004

Primary Completion

April 1, 2009

Study Completion

April 1, 2009

Last Updated

May 19, 2016

Record last verified: 2016-05

Locations

CA(1)