Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease

Exclusion Criteria

• •1. Is from a vulnerable population, as defined by the US CFR Title 45, Part 46, Section 46.111(b) and other local and national regulations, including but not limited to, employees (temporary, part-time, full time, etc) or a family member of the research staff conducting the study, or of the sponsor, or of the contract research organization, or of the institutional review board (IRB)/independent ethics committee (IEC).
• •2. Has a history of sensitivity or allergy to any product containing HP-β-CD.
• •3. A history of hypersensitivity reactions or allergy to the anesthetic and/or sedative agents to be used for the lumbar puncture procedure.
• •4. Taken an anticoagulant in the 2 weeks prior to the Baseline Visit or plan to use anticoagulants during the study.
• •5. Change in antiepileptic treatment between the Screening Visit and the Baseline Visit.
• •6. Received treatment for any investigational product (exclusive of VTS-270) within 4 weeks of the Baseline Visit or at least 5 half-lives, whichever criteria is longest.
• •7. A suspected infection of the central nervous system or any systemic infection.
• •8. A spinal deformity that is likely to impact the ability to perform repeated LPs.
• •9. Evidence of obstructive hydrocephalus or normal pressure hydrocephalus.
• •10. Undergoing intravenous treatment with VTS-270. Note: prior or current treatment with IT VTS-270 is not exclusionary.
• •11. A known bleeding disorder.
• •12. Has any of the following laboratory abnormalities ( greater than 1.5 times the upper limit of normal) at the Screening Visit:
• •* Neutropenia
• •* Thrombocytopenia
• •* Activated partial thromboplastin time
• •* Prothrombin time prolongation.
• •13. Has any other clinically significant disease, disorder or laboratory abnormality, which, in the opinion of the investigator, might put the participant at risk due to participation in the study, or may influence the results of the study or the participant's ability to complete the study.
• •14. Is participating in or plans to participate in any other interventional research study from the time of screening and throughout this study.
• •15. Also excluded are participant, who in the opinion of the investigator, are unable to comply with the protocol or who have a medical condition (eg, cardiovascular, respiratory, hematologic, neurologic, psychiatric, renal) that would potentially increase the risk of study participation.