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

Phase 2

Withdrawn

• Conditions: Homozygous Familial Hypercholesterolemia
• Interventions: Drug: AKCEA-ANGPTL3-LRX

• Registration Number: NCT03455777
• Lead Sponsor: Akcea Therapeutics

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 available

Study Timeline

• Study First Submitted: 2018-02-22
• Study First Submitted QC: 2018-03-05
• Study First Posted: 2018-03-07
• Study Start: 2018-04-12
• Primary Completion: 2018-09-30
• Status Verified: 2018-11
• Last Update Submitted: 2018-11-29
• Last Update Posted: 2018-12-03
• Study Completion: 2018-12-31

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AKCEA-ANGPTL3-LRX Dose 1	AKCEA-ANGPTL3-LRX	-

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Clinical Site; 🇨🇦 Québec, Quebec, Canada