Ignite Creation Date:
2024-05-05 @ 10:26 PM
Last Modification Date:
2024-10-26 @ 10:18 AM
Study NCT ID:
NCT01109498
Status:
UNKNOWN
Last Update Posted:
2011-09-30
First Post:
2010-04-22
Brief Title:
Safety and Efficacy in LPL-Deficient Subjects of AMT-011 an Adeno-Associated Viral Vector Expressing Human Lipoprotein Lipase S447X
Sponsor:
Amsterdam Molecular Therapeutics
Organization:
Amsterdam Molecular Therapeutics
Study Overview
Official Title:
A Study to Determine the Safety and Efficacy in Lipoprotein Lipase-Deficient Subjects After Intramuscular Administration of AMT-011 an Adeno-Associated Viral Vector Expressing Human Lipoprotein LipaseS447X
Status:
UNKNOWN
Status Verified Date:
2010-04
Last Known Status:
ACTIVE_NOT_RECRUITING
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:
LPLD is a rare autosomal recessive disorder characterized by the presence of marked chylomicronemia and hence hypertriglyceridemia Clinically the most severe manifestation of chylomicronemia is acute pancreatitis which can be lethal There is no effective therapy available to modulate the course of the illness and prevent complications for these patients The current clinical management consists of severe reduction of dietary fat that is hard if not almost impossible to comply with LPLD subjects continue to experience pancreatitis attacks and are admitted to intensive care units on several occasions
Alipogene tiparvovec corrects or restores lipoprotein lipase LPL function long term and hence reverses some symptoms halts the disease progression and prevents further complications Alipogene tiparvovec gene therapy ensures that a catabolically beneficial variant of the human LPL gene LPLS447X is expressed and active in the relevant tissues in humans Delivery of the gene is realized via intramuscular injection of an adeno-associated viral vector pseudotyped with AAV1 capsids
Alipogene tiparvovec corrects or restores lipoprotein lipase LPL function long term and hence reverses some symptoms halts the disease progression and prevents further complications Alipogene tiparvovec gene therapy ensures that a catabolically beneficial variant of the human LPL gene LPLS447X is expressed and active in the relevant tissues in humans Delivery of the gene is realized via intramuscular injection of an adeno-associated viral vector pseudotyped with AAV1 capsids
Detailed Description:
The CT-AMT-011-01 study is an open-label dose-escalating study evaluating the safety and efficacy of a single intramuscular administration of AMT-011 at multiple sites The study will be performed in the Community Genomic medicineCenter CGMC Chicoutimi Canada under the supervision of their medical ethical committee and according to the local biosafety procedures The study participants will be treated under the responsibility of a Principal Investigator specialised in the treatment of lipid disorders A total number of 14 subjects will be administered Participants will be screened 3 weeks prior to administration of AMT-011 and will be evaluated for 12 weeks post administration in this study After the study subjects will be followed up long term with particular emphasis on the safety and efficacy aspects of LPL gene therapy using AMT-011 Subjects will be evaluated at the clinical site at 19 weeks 26 weeks 39 weeks 1 year 15 years 2 years 3 years 4 years and 5 years after administration of AMT-011 The TG values that are obtained at week 26 will be used for secondary efficacy analysis
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