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

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)

Status

Withdrawn

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03455777

Enrollment

Registered

2018-03-07

Start date

2018-04-12

Completion date

2018-12-31

Last updated

2018-12-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Homozygous Familial Hypercholesterolemia

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).

Interventions

DRUGAKCEA-ANGPTL3-LRX

Single open-label cohort

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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%

Design outcomes

Primary

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

Secondary

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

Countries

Canada

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026