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 NPC diagnosis
- Have a late-infantile or juvenile form of the disease
This means that the first neurological signs of the disease appeared between 2 and 15 years of age.
There are other criteria, more related to clinical progression, which must be evaluated by the clinician.
Co-medication
If you are taking miglustat at full dose, you are not eligible for this study. If your dose is reduced, you can discuss participation with your clinical team, but it will be necessary to stop your miglustat treatment in order to participate in the NAVIGATE study.
Therapies for NPC such as Miplyffa (arimoclomol) or Aqneursa (levacetylleucine) will be accepted if they are approved in your country and prescribed by your doctor. Other treatments such as cannabinoids or tanganil are accepted in the study as a concomitant treatment. In all cases, treatment should be started at least 3 months before screening and the dose will not change during the study.
What is included:
- 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