NCT04958642

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

A Phase 2B/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 66 (actual)
• Sponsor: Mandos LLC · Industry
• Sex: All
• Age: 4 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here. This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords). In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control). In Part C, all participants will receive study drug.

Detailed description

Participants in Part C will receive adrabetadex until the investigator considers adrabetadex to no longer be beneficial to the participant, or the development program is discontinued.

Conditions

• Niemann-Pick Disease, Type C

Interventions

Type	Name	Description
DRUG	Adrabetadex	Mallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion.

Timeline

• Start date: 2015-12-23
• Primary completion: 2022-04-11
• Completion: 2022-04-11
• First posted: 2021-07-12
• Last updated: 2023-08-29
• Results posted: 2023-08-29

Locations

30 sites across 9 countries: United States, Australia, France, Germany, New Zealand, Singapore, Switzerland, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study