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

November 29, 2018 updated by: Akcea Therapeutics

A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Patients With Homozygous Familial Hypercholesterolemia (HoFH)

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).

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Quebec
      • Québec, Quebec, Canada, G1V4W2
        • Clinical Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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%

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: AKCEA-ANGPTL3-LRX Dose 1
Drug: AKCEA-ANGPTL3-LRX
Single open-label cohort
Other Names:
  • ISIS 703802

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Effect of ISIS 703802 on plasma angiopoietin like 3 (ANGPTL3).
Time Frame: 7 and 14 Weeks
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.
Time Frame: 7 and 14 Weeks
Absolute and percentage change from Baseline to Week 14 will be summarized.
7 and 14 Weeks
Evaluate plasma trough levels of ISIS 703802.
Time Frame: 14 Weeks
Plasma trough levels of ISIS 703802 during treatment period and those during post-treatment follow up period will be descriptively summarized.
14 Weeks
The safety of ISIS 703802 by the incidence of treatment-emergent adverse events
Time Frame: 14 Weeks
The safety of ISIS 703802 will be assessed by determining adverse effects.
14 Weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Akcea Therapeutics

Collaborators

Ionis Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

April 12, 2018

Primary Completion (ANTICIPATED)

September 30, 2018

Study Completion (ANTICIPATED)

December 31, 2018

Study Registration Dates

First Submitted

February 22, 2018

First Submitted That Met QC Criteria

March 5, 2018

First Posted (ACTUAL)

March 7, 2018

Study Record Updates

Last Update Posted (ACTUAL)

December 3, 2018

Last Update Submitted That Met QC Criteria

November 29, 2018

Last Verified

November 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ISIS 703802-CS4

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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