Ignite Creation Date:
2024-05-06 @ 12:55 PM
Last Modification Date:
2024-10-26 @ 1:06 PM
Study NCT ID:
NCT03887533
Status:
TERMINATED
Last Update Posted:
2022-08-30
First Post:
2019-03-22
Brief Title:
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease Type C1
Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease Type C1
Status:
TERMINATED
Status Verified Date:
2021-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Enrollment was poor due to COVID-19 pandemic
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background
For people who have Niemann-Pick disease type C1 NPC1 cholesterol and other fats have trouble moving out of liver and other tissue cells This makes the cells sick Researchers want to find out if a drug called VTS-270 can help
Objective
To test if VTS-270 is safe and effective in treating chronic liver disease associated with NPC1
Eligibility
People ages 3-60 with NPC1
Design
Participants may be screened by phone or under another protocol
Participants will have visits once a month for 12 months If they have intrathecal injections the study may last 15 months or more The first visit will last about 5 days Others will last 2-3 days
Participants will get VTS-270 injected into a vein at each visit They can also choose to have intrathecal injections These are like spinal taps
Some visits will also include
Physical exam
Urine tests
Blood tests A small tube or needle will be inserted into the participants vein to collect blood The small tube will also be used to give the VTS-270
Hearing tests For one test participants will have electrodes taped to their head These will record brain waves
Breathing tests
Ultrasound of abdomen Sounds waves will take pictures of the participant s body
Chest x-ray This is a picture of the lungs
For people who have Niemann-Pick disease type C1 NPC1 cholesterol and other fats have trouble moving out of liver and other tissue cells This makes the cells sick Researchers want to find out if a drug called VTS-270 can help
Objective
To test if VTS-270 is safe and effective in treating chronic liver disease associated with NPC1
Eligibility
People ages 3-60 with NPC1
Design
Participants may be screened by phone or under another protocol
Participants will have visits once a month for 12 months If they have intrathecal injections the study may last 15 months or more The first visit will last about 5 days Others will last 2-3 days
Participants will get VTS-270 injected into a vein at each visit They can also choose to have intrathecal injections These are like spinal taps
Some visits will also include
Physical exam
Urine tests
Blood tests A small tube or needle will be inserted into the participants vein to collect blood The small tube will also be used to give the VTS-270
Hearing tests For one test participants will have electrodes taped to their head These will record brain waves
Breathing tests
Ultrasound of abdomen Sounds waves will take pictures of the participant s body
Chest x-ray This is a picture of the lungs
Detailed Description:
Niemann-Pick disease type C NPC is a lethal autosomal recessive lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence NPC results from mutation of either the NPC1 approximately 95 of cases or NPC2 genes Biochemically NPC is characterized by the endolysosomal storage of unesterified cholesterol and lipids in both the central nervous system and peripheral tissues such as the liver Individuals with NPC demonstrate progressive cerebellar ataxia and dementia Acute cholestatic liver disease is frequently observed in the neonatalinfantile period but subsequently resolves However chronic sub-clinical liver disease persists Intrathecal 2-hydroxypropyl-beta-cyclodextrin HPbetaCD VTS-270 has proven effective in reducing signs and prolonging life in NPC1 animal models and Phase 12a data support efficacy in NPC1 patients Parenteral administration of VTS-270 has also been shown to be effective in treating liver disease in the NPC1 cat
In this Phase 12a open-label randomized parallel dose single-center study we will examine whether VTS-270 can be used to treat chronic subacute liver disease in NPC1 patients Our primary objective is to determine the safety and tolerability of intravenous VTS-270 in NPC1 disease Secondary objectives will be to evaluate the efficacy of VTS-270 to reduce plasma cholestane-3beta5alpha6beta-triol an NPC1-specific pharmacodynamic biomarker and to normalize the degree of liver injury Exploratory testing will include lipid and protein biomarkers This study will evaluate three dose levels 500 1000 and 1500 mgkg administered monthly for twelve months Safety will be assessed by adverse event recording clinical laboratory testing and physical examination Clinical efficacy will be evaluated by assessment of liver chemistries determination of liver size and liver STIFFNESS Biochemical efficacy will be assessed by measurement of plasma cholestane-3beta5alpha6beta-triol and other biomarkers
In this Phase 12a open-label randomized parallel dose single-center study we will examine whether VTS-270 can be used to treat chronic subacute liver disease in NPC1 patients Our primary objective is to determine the safety and tolerability of intravenous VTS-270 in NPC1 disease Secondary objectives will be to evaluate the efficacy of VTS-270 to reduce plasma cholestane-3beta5alpha6beta-triol an NPC1-specific pharmacodynamic biomarker and to normalize the degree of liver injury Exploratory testing will include lipid and protein biomarkers This study will evaluate three dose levels 500 1000 and 1500 mgkg administered monthly for twelve months Safety will be assessed by adverse event recording clinical laboratory testing and physical examination Clinical efficacy will be evaluated by assessment of liver chemistries determination of liver size and liver STIFFNESS Biochemical efficacy will be assessed by measurement of plasma cholestane-3beta5alpha6beta-triol and other biomarkers
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 |
|---|---|---|---|
| 19-CH-0028 | None | None | None |