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Study Investigating Potential Treatment for people with Niemann-Pick type C or GM1/GM2 gangliosidoses (Tay-Sachs, Sandhoff or GM2AB variant) disease

Pediatric | Adult | Clinical Trial | NPC | Tay-Sachs

This study is looking at a study drug called nizubaglustat and how it might improve symptoms in people with Niemann-Pick type C or GM1/GM2 gangliosidoses (Tay-Sachs, Sandhoff or GM2AB variant) disease.

This study will be over 18 months and is being done to see how safe and effective nizubaglustat is in treating these diseases compared to placebo. Placebo looks the same as the study drug but does not have any medicine-related effects on the body.

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Participation Requirements

  • Sex:

    Any

  • Eligible Ages:

    4 and up

Participation Criteria

Inclusion Criteria:
- Be at least 4 years of age
- Confirmed diagnosis of Niemann-Pick type C or GM1/GM2 gangliosidoses
- Able to take the study medication (dissolvable oral tablet)
- Must be willing to comply to pregnancy prevention requirements
- Willing and able to complete all study assessments
- Willing to be open and honest with providing information on all current drugs/therapies being used to manage symptoms
Exclusion Criteria:
- Body weight < 10kg
- Presence of another brain/liver disease
- Not on a stable dose of anti seizure medication
- Pregnant or planned pregnancy
- Prior use of another study drug including gene therapy or stem cell transplant
- Current thoughts of self harm
- Taking/have taken the drug miglustat within the last year

Study Location

University of Alberta
University of Alberta
Edmonton, Alberta
Canada

Contact Study Team

Primary Contact

Casey Brodie

[email protected]
780-492-9277
Study Sponsored By
University of Alberta
Participants Required
More Information
Study ID: Pro00156506