VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
Status:
Active, not recruiting
Trial end date:
2021-10-01
Target enrollment:
Participant gender:
Summary
Due to different study designs, the sponsor separated Part C into a separate registration
(NCT04958642), leaving Parts A/B here in NCT02534844.
This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick
Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords).
In Parts A/B, two out of every three patients will receive the study drug. The third patient
will receive 1 to 2 small needle pricks at the location where the LP and IT injection is
normally made (sham control).
In Part C, all participants will receive study drug, as described in the Part C registration
record.
Start date for this record is the first day a participant was enrolled in Parts A/B. The
trial is actually continuing until the last primary outcome measure of safety data are
collected from Part C participants. The last primary outcome measure of safety, along with
final adverse events results will be posted in the separate Part C registration record.