Ignite Creation Date:
2024-05-05 @ 5:32 PM
Last Modification Date:
2024-10-26 @ 9:33 AM
Study NCT ID:
NCT00477594
Status:
COMPLETED
Last Update Posted:
2016-09-09
First Post:
2007-05-22
Brief Title:
Open Label Extension of ISIS 301012 Mipomersen to Treat Familial Hypercholesterolemia
Sponsor:
Kastle Therapeutics LLC
Organization:
Kastle Therapeutics LLC
Study Overview
Official Title:
An Open-Label Extension Study to Assess the Long-term Safety and Efficacy of Mipomersen in Subjects With Familial Hypercholesterolemia
Status:
COMPLETED
Status Verified Date:
2016-08
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:
The purpose of this study is to evaluate the safety and efficacy of extended dosing of mipomersen in patients with familial hypercholesterolemia on lipid-lowering therapy who have completed either the 301012-CS8 NCT00280995 or 301012-CS9 NCT00281008 clinical drug trials
Detailed Description:
Familial Hypercholesterolemia FH is an autosomal dominant metabolic disorder characterized by markedly elevated low density lipoprotein LDL premature onset of atherosclerosis and development of xanthomata There are two distinct subpopulations that have a high unmet medical need due to the lack of alternative therapy homozygotes who have two defective LDL receptor LDL-R genes and heterozygotes with a history of cardiovascular disease CVD on maximally tolerated therapy Treatment for FH is directed at lowering plasma levels of LDL-C
Mipomersen is an antisense drug targeted to human apolipoprotein B apoB the principal apolipoprotein of atherogenic LDL-C and its metabolic precursor very low density lipoprotein VLDL Mipomersen is complimentary to the coding region of the messenger ribonucleic acid mRNA for apo-B Inhibition of apo-B would be expected to impair VLDL synthesis and result in lowered levels of LDL-C
In early clinical trials mipomersen has been shown to reduce levels of LDL-C to recommended target levels in some participants
This was an open-label extension study which consisted of a 2-week screening period up to 3 years of treatment with mipomersen and a 24-week post-treatment follow-up period Patients who participated in Cohorts A B or C in study 301012-CS9 were randomized in a 11 ratio to mipomersen 200 mg once a week QW or 200 mg mipomersen every other week QOW for up to 3 years Patients randomized to mipomersen 200 mg QOW were allowed to receive mipomersen 200 mg QW at the Investigators discretion after the first 52 weeks of the treatment period Patients who participated in study 301012-CS8 or Cohort D of study 301012-CS9 received 200 mg mipomersen QW for up to 3 years
Mipomersen is an antisense drug targeted to human apolipoprotein B apoB the principal apolipoprotein of atherogenic LDL-C and its metabolic precursor very low density lipoprotein VLDL Mipomersen is complimentary to the coding region of the messenger ribonucleic acid mRNA for apo-B Inhibition of apo-B would be expected to impair VLDL synthesis and result in lowered levels of LDL-C
In early clinical trials mipomersen has been shown to reduce levels of LDL-C to recommended target levels in some participants
This was an open-label extension study which consisted of a 2-week screening period up to 3 years of treatment with mipomersen and a 24-week post-treatment follow-up period Patients who participated in Cohorts A B or C in study 301012-CS9 were randomized in a 11 ratio to mipomersen 200 mg once a week QW or 200 mg mipomersen every other week QOW for up to 3 years Patients randomized to mipomersen 200 mg QOW were allowed to receive mipomersen 200 mg QW at the Investigators discretion after the first 52 weeks of the treatment period Patients who participated in study 301012-CS8 or Cohort D of study 301012-CS9 received 200 mg mipomersen QW for up to 3 years
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2007-001024-12 | EUDRACT_NUMBER | None | None |