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

Akcea Therapeutics

Status and phase: Withdrawn; Phase 2

Conditions: Homozygous Familial Hypercholesterolemia

Treatments: Drug: AKCEA-ANGPTL3-LRX

Study type: Interventional

Funder types: Industry

Identifiers

NCT03455777

ISIS 703802-CS4

Details and patient eligibility

About

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).

Sex: All

Ages: 18+ years old

Volunteers: No 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%

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

AKCEA-ANGPTL3-LRX Dose 1

Experimental group

Treatment:

Drug: AKCEA-ANGPTL3-LRX