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

Homozygous Familial Hypercholesterolemia Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT03455777
• Other study ID #: ISIS 703802-CS4
• Status: Withdrawn
• Phase: Phase 2
• Start date: April 12, 2018
• Est. completion date: December 31, 2018

Study information

• Verified date: November 2018
• Source: Akcea Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: December 31, 2018
• Est. primary completion date: September 30, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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%

Related Conditions & MeSH terms

• Homozygous Familial Hypercholesterolemia
• Hypercholesterolemia
• Hyperlipoproteinemia Type II

Intervention

Drug:
• AKCEA-ANGPTL3-LRX
Single open-label cohort

Locations

Country	Name	City	State
Canada	Clinical Site	Québec	Quebec

Sponsors (2)

Lead Sponsor	Collaborator
Akcea Therapeutics	Ionis Pharmaceuticals, Inc.

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Reduction of low density lipoprotein cholesterol (LDL-C).	Percent change in LDL-C from Baseline to week 14.	14 weeks
Secondary	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
Secondary	Effect of ISIS 703802 on lipid parameters.	Absolute and percentage change from Baseline to Week 14 will be summarized.	7 and 14 Weeks
Secondary	Evaluate plasma trough levels of ISIS 703802.	Plasma trough levels of ISIS 703802 during treatment period and those during post-treatment follow up period will be descriptively summarized.	14 Weeks
Secondary	The safety of ISIS 703802 by the incidence of treatment-emergent adverse events	The safety of ISIS 703802 will be assessed by determining adverse effects.	14 Weeks