Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

APPROVED_FOR_MARKETING

Conditions studied: Familial Chylomicronemia Syndrome

In brief

The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).

Key facts

Study ID

NCT06360237

Run by

Ionis Pharmaceuticals, Inc.

Last updated by the study team

2024-12-27

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Has a diagnosis of FCS as determined by the sponsoring physician. Ionis will review each application to determine eligibility based on documentation of validated genetic or clinical diagnosis.
• o Documented loss of function mutations (homozygous, compound / double heterozygous) in genes such as LPL, GPIHBP1, APOA5, APOC2 or LMF1) or clinically validated diagnosis of FCS.
• Resides in and is a resident of the United States.
• Willing to follow a diet comprising ≤20 g fat per day.

You may not qualify if…

• Has any new or worsening of existing conditions which, in the opinion of the physician, would make the patient unsuitable for treatment with olezarsen.
• Olezarsen naïve patients with baseline platelet count <100x109/L at qualification.
• Estimated GFR (eGFR) <30 mL/min/1.73 m2.
• Secondary factors are the cause of triglyceride elevations.
• Is currently hospitalized in an acute emergency setting.

Where it is running

• Expanded Access Site — Carlsbad, California, United States

Full record on ClinicalTrials.gov

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