Ignite Creation Date:
2024-05-06 @ 2:47 AM
Last Modification Date:
2024-10-26 @ 11:23 AM
Study NCT ID:
NCT02124083
Status:
COMPLETED
Last Update Posted:
2018-02-22
First Post:
2014-04-25
Brief Title:
Phase 12 Study of Vorinostat Therapy in 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:
Phase 12 Study of Vorinostat Therapy in Niemann-Pick Disease Type C1
Status:
COMPLETED
Status Verified Date:
2018-01-13
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:
Niemann-Pick disease type C NPC is a lethal autosomal recessive lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence The causative genes NPC1 about 95 of cases and NPC2 about 5 of cases are involved in the intracellular trafficking of lipids and cholesterol Mutations on either of these genes lead to progressive accumulation of unesterified cholesterol and other lipids in the central nervous system CNS Vorinostat is a histone deacetylase inhibitor that has been shown in vivo to increase mutant NPC1 protein levels and to reverse cellular accumulation of unesterified cholesterol Vorinostat has been labeled by the FDA for treatment of cutaneous T-cell lymphoma In this Phase I non-randomized open-label single-center study we plan to study whether Vorinostat can be repurposed to treat patients with NPC1 Our primary objective is to determine the safety and tolerability of Vorinostat in NPC1 disease Our secondary objectives will be to determine biochemical efficacy of Vorinostat to increase expression of NPC1 protein and normalize lipid and protein biomarkers This study will enroll up to 12 NPC1 patients and test the safety of two dose levels 200 and 400 mg Drug will be administered on a 3 days on4 days off schedule for 3 months at each dose level Patients will be evaluated at the NIH Clinical Center at 0 3 and 6 months Safety will be assessed by adverse events AEs clinical laboratory tests and physical examinations Biochemical efficacy will be assessed by measurement of serum and cerebral spinal fluid biomarkers Clinical efficacy will be evaluated by audiologic testing assessment ataxia and swallowing studies
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 The causative genes NPC1 about 95 of cases and NPC2 about 5 of cases are involved in the intracellular trafficking of lipids and cholesterol Mutations on either of these genes lead to progressive accumulation of unesterified cholesterol and other lipids in the central nervous system CNS Vorinostat is a histone deacetylase inhibitor that has been shown in vivo to increase mutant NPC1 protein levels and to reverse cellular accumulation of unesterified cholesterol Vorinostat has been labeled by the FDA for treatment of cutaneous T-cell lymphoma In this Phase I non-randomized open-label single-center study we plan to study whether Vorinostat can be repurposed to treat patients with NPC1 Our primary objective is to determine the safety and tolerability of Vorinostat in NPC1 disease Our secondary objectives will be to determine biochemical efficacy of Vorinostat to increase expression of NPC1 protein and normalize lipid and protein biomarkers This study will enroll up to 12 NPC1 patients and test the safety of two dose levels 200 and 400 mg Drug will be administered on a 3 days on4 days off schedule for 3 months at each dose level Patients will be evaluated at the NIH Clinical Center at 0 3 and 6 months Safety will be assessed by adverse events AEs clinical laboratory tests and physical examinations Biochemical efficacy will be assessed by measurement of serum and cerebral spinal fluid biomarkers Clinical efficacy will be evaluated by audiologic testing assessment ataxia and swallowing studies
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 |
|---|---|---|---|
| 14-CH-0102 | OTHER | The National Institutes of Health | None |