Clinical Trial: Pediatrics, Neurology

Niemann-Pick Type C1 Disease (PRO00003302)

Full Name

A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS270 (2-hydroxypropyl-β-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease


There are three parts to this study (Parts A, B and C). Part A of the study will evaluate the safest dose of VTS-270 that children should receive every two weeks. In Part A, 3 different doses will be evaluated to see which one is best to study further in the next part of the study (Part B). In Part A, participants will have a 75% chance of receiving VTS-270, and a 25% chance of receiving no treatment (“control”). In the second part of the study (Part B), 67% of participants will receive VTS-270, and 33% will receive no treatment (“control”). The dose of VTS-270 to be used in Part B was determined by the data of Part A. Therefore, all participants in the treatment group in Part B will receive the same dose. Part C is considered the “open label extension” phase of the study. Twelve participants will be enrolled in Part A, all of whom are expected to continue into Part B, with a planned total treatment time of 12 months. After Part A enrollment is complete, new patients will be enrolled into Part B, wherein they will receive a total of 12 months of treatment with the dose selected from Part A. The drug is administered through an intrathecal lumbar puncture. Intrathecal means it is given directly into the cerebrospinal fluid that bathes the brain and spinal cord. Since participants are receiving an intrathecal lumbar puncture, they will also receive “conscious sedation” (procedures used to calm your child). Participants assigned to the no treatment (“control”) arm will not receive VTS-270 but will go through the same procedures as those receiving the drug every two weeks. This means that participants assigned to no treatment (“control”), will receive a “sham” intrathecal lumbar puncture. Participation in this study will last approximately 14 months. Hearing will be closely monitored in this study. Hearing loss could occur with a single dose or could occur after receiving multiple doses.


Inclusion Criteria

Must be 6 to 21 years of age at time of screening with onset of neurological symptoms prior to 15 years of age

Must have a diagnosis of Niemann-Pick Type C1

Must be able to undergo a lumbar puncture and intrathecal (into the spinal canal) drug administration under conscious sedation or general anesthesia

Must have a Total NPC Clinical Severity Score of 10 or greater

Must not have a body weight less than 15 kg (33 pounds)

Must not have uncontrolled seizures

Must not be pregnant or nursing a baby

Doctor(s) Running This Study