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

• Status: Withdrawn
• Trial end date: 2018-12-31
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Akcea Therapeutics
• Collaborator: Ionis Pharmaceuticals, Inc.

Criteria

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%