NCT02534844

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

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

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 56 (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 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.

Detailed description

Non-clinical studies and a Phase 1 clinical trial suggest that intrathecal administration of VTS-270 in patients with neurologic manifestations of Niemann-Pick Type C1 (NPC1) disease has the potential to slow the rate of progression of their neurologic disease. Niemann-Pick Type C1 (NPC1) disease is a rare, neurodegenerative, inherited, autosomal recessive lysosomal lipid storage disorder primarily in children and teenagers. The disease is characterized by the inability to properly metabolize cholesterol and other lipids within the cell due to mutations in the NPC1 gene, causing unesterified cholesterol to accumulate in the brain, liver and spleen. This study plans to enroll about 51 participants with NPC1 disease. It will be conducted in three parts: Parts A, B, and C. * Part A will evaluate 3 different dose levels of VTS-270 in 12 participants to determine the dose level for Parts B and C. * In Part B, 39 more participants will join the original 12 to evaluate the safety and effectiveness of the dose selected from Part A compared to sham control. * Part C will be an open-label extension phase of the study for Part B participants who either complete Part B or have met rescue therapy criteria, as well as participants entering Part C from other trials. Participants in Part C will receive treatment with VTS-270 until the product is licensed or the program is terminated (anticipated within 5 years). Final results will be posted in the Part C registration record (NCT04958642).

Conditions

• Niemann-Pick Disease, Type C

Interventions

Type	Name	Description
DRUG	Parts A/B: Adrabetadex	900 - 1800 milligram (mg) of adrabetadex administered every 2 weeks via lumbar intrathecal infusion
OTHER	Parts A/B: Sham Control	No experimental drug is administered to participants - intrathecal administrations are simulated by skin prick

Timeline

• Start date: 2015-10-01
• Primary completion: 2018-03-28
• Completion: 2018-03-28
• First posted: 2015-08-28
• Last updated: 2023-02-22
• Results posted: 2023-02-22

Locations

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

Regulatory

• FDA-regulated drug study