Ignite Creation Date:
2024-05-06 @ 7:25 AM
Last Modification Date:
2024-10-26 @ 11:48 AM
Study NCT ID:
NCT02534844
Status:
COMPLETED
Last Update Posted:
2023-02-22
First Post:
2015-08-18
Brief Title:
VTS-270 to Treat Niemann-Pick Type C1 NPC1 Disease
Sponsor:
Mandos LLC
Organization:
Mandos LLC
Study Overview
Official Title:
A Phase 2b3 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
Status Verified Date:
2023-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Due to different study designs the sponsor separated Part C into a separate registration NCT04958642 leaving Parts AB 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 AB 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 AB 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
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 AB 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 AB 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
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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2015-002548-15 | EUDRACT_NUMBER | None | None |