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Synergistic Enteral Regimen for Treatment of the Gangliosidoses

NCT02030015 · Status: TERMINATED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2021-04-14

Study results available
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Summary

The investigators hypothesize that a combination therapy using miglustat and the ketogenic diet for infantile and juvenile patients with gangliosidoses will create a synergy that 1) improves overall survival for patients with infantile or juvenile gangliosidoses, and 2) improves neurodevelopmental clinical outcomes of therapy, compared to data reported in previous natural history studies. The ketogenic diet is indicated for management of seizures in patients with seizure disorders. In this study, the ketogenic diet will be used to minimize or prevent gastrointestinal side-effects of miglustat. A Sandhoff disease mouse study has shown that the ketogenic diet may also improve central nervous system response to miglustat therapy (see Denny in "Citations" list below). Patients with infantile and juvenile gangliosidoses commonly suffer from seizure disorders, and use of the ketogenic diet in these patients may therefore also improve seizure management.

Conditions

  • GM1 Gangliosidoses
  • GM2 Gangliosidoses
  • Tay-Sachs Disease
  • Sandhoff Disease

Interventions

DRUG

miglustat

The Syner-G therapy regimen includes treating with orally-administered miglustat for the duration of the 60-month study.

OTHER

Ketogenic Diet

The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet for the 60-month duration of this study.

Sponsors & Collaborators

  • Rare Diseases Clinical Research Network

    collaborator NETWORK

  • National Center for Advancing Translational Sciences (NCATS)

    collaborator NIH

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

    collaborator NIH

  • Lysosomal Disease Network

    collaborator OTHER

  • University of Minnesota

    lead OTHER

Principal Investigators

  • Jeanine R. Jarnes, PharmD · University of Minnesota Fairview Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
204 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-12-22
Primary Completion
2019-07-31
Completion
2019-07-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02030015 on ClinicalTrials.gov