Status:

WITHDRAWN

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

Lead Sponsor:

Akcea Therapeutics

Collaborating Sponsors:

Ionis Pharmaceuticals, Inc.

Conditions:

Homozygous Familial Hypercholesterolemia

Eligibility:

All Genders

18+ years

Phase:

PHASE2

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 Hyperc...

Eligibility Criteria

Inclusion

  • 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

  • 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%

Key Trial Info

Start Date :

April 12 2018

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

December 31 2018

Estimated Enrollment :

Patients enrolled

Trial Details

Trial ID

NCT03455777

Start Date

April 12 2018

End Date

December 31 2018

Last Update

December 3 2018

Active Locations (1)

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1

Clinical Site

Québec, Quebec, Canada, G1V4W2