Prediction
## Amended Eligibility Criteria (Version 17)
**Inclusion Criteria:**
To be included in the study, a participant must meet the following criteria:
1.
Is male or female and at least 4 years of age at time of screening.
2.
Has a confirmed diagnosis of NPC and exhibits neurologic symptoms.
3.
Patient or parent/guardian must provide written informed consent to participate in the study.
In addition to parental consent, assent to participate must also be sought from minor children.
4.
Has the ability to undergo LP and IT drug administration.
5.
If taking miglustat (Zavesca®), must have been on a stable dose for the past 6 weeks and be willing to remain on a stable dose for the duration of participation in the study.
Alternatively, subjects may elect to discontinue miglustat use and be eligible for entry into the study after undergoing a minimum 6-week washout period prior to study Day 1.
6.
If there is a history of seizures, the condition must be adequately controlled, ie, the pattern of seizure activity must be stable and the subject must be on a stable dose and regimen of antiepileptic medication during the 1 month prior to Screening, with no change in dose or regimen up to and including the day of Screening.
7.
**After 21 June 2021, the investigator must assess whether the subject is benefiting from treatment with intrathecal adrabetadex.
For those subjects who appear to benefit from treatment, the investigator must review with them the risks associated with adrabetadex, including hearing loss, and the data from the randomized, controlled trial that demonstrated no significant differences between subjects treated with adrabetadex and sham-treated subjects on any efficacy measures.
Subjects must be discontinued from the study if the investigator does not consider them to be benefiting from treatment and/or they do not understand the risks associated with adrabetadex, including hearing loss.
This discussion with the subject should be documented in the subject’s medical record and only those subjects (or legally authorized representative) who have demonstrated an understanding of the risk/benefit of adrabetadex treatment should be permitted to continue in the study.**
**Exclusion Criteria:**
Patients will be excluded from the study if they meet any of the following criteria:
1.
Weighs less than 15 kg.
2.
Has a history of hypersensitivity reactions to any product containing HP-β-CD or has a history of hypersensitivity reactions or allergy to anesthesia/sedation.
3.
Has received treatment with any investigational product (other than adrabetadex) within 1 month before Day 1 of treatment.
4.
Is pregnant or nursing.
5.
Has systemic infection or uncontrolled psychosis.
6.
Has known history of a bleeding disorder.
7.
Has used anticoagulants within 2 months of entry into the study.
8.
Per protocol, or in the opinion of the investigator:
* Has laboratory values that would preclude participation
* Has suspected infection of the central nervous system (CNS)
* Has a spinal deformity that could impact performance of repeated LP
* Has a serious skin infection in the lumbar region or evidence of obstructive or normal pressure hydrocephalus
* Is unable to comply with the study requirements
* Has a medical condition that might increase the risk of participation