Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With... | NCT03455777

Studies
Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

NCT03455777

Sponsor

Akcea Therapeutics

Status

Withdrawn

Last Update Posted

Dec 3, 2018Actual

Enrollment

0Actual

Phase

Phase 2

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

AKCEA-ANGPTL3-LRX

Countries

Canada

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2018

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Study withdrawn due to lack of available patients meeting entry criteria

Expanded Access Info

Start Date

Apr 12, 2018Actual

Primary Completion Date

Sep 30, 2018Estimated

Completion Date

Dec 31, 2018Estimated

First Submitted Date

Feb 22, 2018

First Submission Date that Met QC Criteria

Mar 5, 2018

First Posted Date

Mar 7, 2018Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 29, 2018

Last Update Posted Date

Dec 3, 2018Actual

Name	Class
Ionis Pharmaceuticals, Inc.	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3- LRX for reduction of low density lipoprotein cholesterol (LDL-C) levels in patients with Homozygous Familial Hypercholesterolemia (HoFH).

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
AKCEA-ANGPTL3-LRX Dose 1	Experimental		Drug: AKCEA-ANGPTL3-LRX

Interventions

Name	Type	Description	Arm Group Labels	Other Names
AKCEA-ANGPTL3-LRX	Drug	Single open-label cohort	AKCEA-ANGPTL3-LRX Dose 1

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Reduction of low density lipoprotein cholesterol (LDL-C).	Percent change in LDL-C from Baseline to week 14.	14 weeks

Secondary Outcomes

Measure	Description	Time Frame
Effect of ISIS 703802 on plasma angiopoietin like 3 (ANGPTL3).	Absolute and percentage change in ANGPTL3 protein from Baseline to Week 14 will be summarized.	7 and 14 Weeks
Effect of ISIS 703802 on lipid parameters.

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Body mass index (BMI) ≤ 35 kg/m2,
Genetic confirmation of two mutant alleles at the LDLR, APOB, PCSK9, or LDLRAP1 gene locus OR an untreated LDL-C > 500 mg/dL (13 mmol/L) or treated LDL-C ≥ 300 mg/dL (2.59 mmol/L) together with either cutaneous or tendon xanthoma before age 10 years OR familial medical history of genetically confirmed heterozygous FH in both parents OR untreated elevated LDL-C and TC > 250 mg/dL consistent with the disease,
Patients must be on stable LDL-C lowering agents or on regular apheresis

Exclusion Criteria:

Myocardial infarction, percutaneous transluminal coronary intervention, or coronary artery bypass graft surgery within 12 weeks prior to Screening, or cerebrovascular accident within 24 weeks prior to Screening.
Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
Clinical Site		Québec	Quebec	G1V4W2	Canada

Condition Browse Module

MeSH Terms

ID	Term
D000090542	Homozygous Familial Hypercholesterolemia

Ancestor Terms

ID	Term
D006938	Hyperlipoproteinemia Type II
D008052	Lipid Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn

Browse Leaves

Not provided

Browse Branches

Not provided