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

NCT ID: NCT03455777

Last Updated: 2018-12-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2018-04-12

Study Completion Date

2018-12-31

Brief Summary

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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

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Conditions

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Homozygous Familial Hypercholesterolemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AKCEA-ANGPTL3-LRX Dose 1

Group Type EXPERIMENTAL

AKCEA-ANGPTL3-LRX

Intervention Type DRUG

Single open-label cohort

Interventions

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AKCEA-ANGPTL3-LRX

Single open-label cohort

Intervention Type DRUG

Other Intervention Names

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ISIS 703802

Eligibility Criteria

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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%
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role collaborator

Akcea Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Clinical Site

Québec, Quebec, Canada

Site Status

Countries

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Canada

Other Identifiers

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ISIS 703802-CS4

Identifier Type: -

Identifier Source: org_study_id

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