Eligibility criteria
Participants must fulfil the following inclusion criteria
To be eligible, you need to:
- Be 4 years of age or older
Children under the age of 18 need a parent or guardian to sign the Informed Consent Form (ICF) and agree to take part in the study. - Have a clinical and genetic diagnosis for either GM1 or GM2 Gangliosidosis (genetic or enzymatic diagnosis).
- Have a late-infantile or juvenile form of the disease
This means that the first neurological signs of the disease appeared between 1 and 10 years of age.
There are other criteria, more related to clinical progression, that must be evaluated by the clinician.
If you are currently or have participated in another clinical trial, and you are interested in participating in NAVIGATE, there needs to be a period of time between the last dose of the previous study and the first dose of nizubaglustat. Please discuss this with your clinician.
Those who qualify will receive:
- Reimbursement of select expenses (all the operational aspects such as travel, lodging, and meals to attend the visits will be arranged and communicated to the families)
- Study-related care
- Study medication