Ignite Creation Date:
2024-05-06 @ 11:15 AM
Last Modification Date:
2024-10-26 @ 12:42 PM
Study NCT ID:
NCT03471143
Status:
RECRUITING
Last Update Posted:
2024-03-12
First Post:
2018-03-07
Brief Title:
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease Type C
Sponsor:
Washington University School of Medicine
Organization:
Washington University School of Medicine
Study Overview
Official Title:
Phase 12a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease Type C
Status:
RECRUITING
Status Verified Date:
2024-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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 Niemann-Pick C1 disease NPC1 95 of cases or NPC2 genes 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 central nervous system decline including inability to coordinate balance gait extremity and eye movements Acute liver disease in the newborninfant period is frequently observed but subsequently resolves However chronic sub-clinical liver disease persists Intrathecal 2-Hydroxypropyl-β-Cyclodextrin HP-β-CD VTS-270 also known as adrabetadex has proven effective in reducing the signs and prolonging life in animal models and Phase 12a data support efficacy in NPC1 patients Adrabetadex VTS-270 also has been shown to be effective in treating liver disease in the NPC1 cat
This Phase 12a open-label multiple ascending dose trial will evaluate whether adrabetadex VTS-270 administered intravenously is effective in treating acute liver disease in NPC1 infants
This Phase 12a open-label multiple ascending dose trial will evaluate whether adrabetadex VTS-270 administered intravenously is effective in treating acute liver disease in NPC1 infants
Detailed Description:
In the first phase of the study infants will be treated for a total of 6 weeks treated twice weekly Infants will be admitted to the Neonatal Intensive Care Unit NICU for the first week of treatment Procedures during the first week of the study will include blood draws for genetic testing clinical and research blood draws urine collection abdominal ultrasound peripheral inserted central catheter PICC placement hearing screening and the first two IV adrabetadex VTS-270 infusions through the PICC line Weeks 2-6 will occur on an outpatient basis During week 2-6 the infant will receive 2 doses per week of adrabetadex VTS-270 with blood draws and urine collection during weeks 2 4 and 6 PICC line will be removed after final infusion
Subjects who demonstrate significant reduction either in the glycine-conjugated trihydroxycholanic acid biomarker or serum bilirubin direct bilirubin or direct bilirubin total bilirubin ratio will be allowed to crossover into the second phase of the study an open label phase of six months duration in which IV adrabetadex VTS-270 will be administered monthly for a total of six doses Month 1-6 procedures will occur on an outpatient basis Procedures during the second phase include a monthly intravenous line placement After each monthly visit the intravenous line will be removed
Subjects who demonstrate significant reduction either in the glycine-conjugated trihydroxycholanic acid biomarker or serum bilirubin direct bilirubin or direct bilirubin total bilirubin ratio will be allowed to crossover into the second phase of the study an open label phase of six months duration in which IV adrabetadex VTS-270 will be administered monthly for a total of six doses Month 1-6 procedures will occur on an outpatient basis Procedures during the second phase include a monthly intravenous line placement After each monthly visit the intravenous line will be removed
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 |
|---|---|---|---|
| 1U01HD090845-01 | NIH | None | httpsreporternihgovquickSearch1U01HD090845-01 |