Ignite Creation Date:
2024-05-05 @ 5:13 PM
Last Modification Date:
2024-10-26 @ 9:29 AM
Study NCT ID:
NCT00410566
Status:
TERMINATED
Last Update Posted:
2015-03-19
First Post:
2006-12-11
Brief Title:
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency Niemann-Pick Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
A Phase I Single-Center Single Dose Dose Escalation Study of Recombinant Human Acid Sphingomyelinase rhASM in Adults With Acid Sphingomyelinase Deficiency ASMD
Status:
TERMINATED
Status Verified Date:
2015-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Terminated by sponsor - Single dose safety objective achieved
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency
Detailed Description:
ASM deficiency ASMD also known as Niemann-Pick A and B disease is a rare genetic disorder in which reduced activity of the lysosomal enzyme ASM leads to the accumulation of sphingomyelin primarily in macrophages throughout the body This deficiency results in characteristic features such as hepatosplenomegaly thrombocytopenia interstitial lung disease growth retardation coronary artery disease fatigue and in severe cases neurodegeneration with death in early childhood There is no specific treatment for this disease This Phase 1 safety study will seek to enroll a minimum of 12 and a maximum of 30 eligible adults patients with ASMD with each patient participating for approximately 7 weeks
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None