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

NCT06360237 | Unknown

• 1 other identifier: ISIS 678354
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Familial Chylomicronemia Syndrome

Keywords

Familial Chylomicronemia; Familial Lipoprotein Lipase Deficiency; Hyperlipoproteinemias; Familial Hyperlipoproteinemia Type 1; Hyperlipoproteinemia Type 1; Hyperchylomicronemia, Familial; Lipoprotein Lipase Deficiency, Familial; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Lipid Metabolism, Inborn Errors; Metabolic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn

Interventions

Olezarsen DRUG

Olezarsen 80 mg administered once monthly by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.

Also known as: ISIS-678354

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)

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.

Sponsors & Collaborators

• Ionis Pharmaceuticals, Inc.: lead

Study Sites (1)

Expanded Access Site

Carlsbad, California, 92010, United States

Location

Related Links

• Related Info

MeSH Terms

Conditions

Familial hyperchylomicronemia syndrome; Hyperlipoproteinemia Type I; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Lipid Metabolism, Inborn Errors; Metabolic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn

Interventions

olezarsen

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 6, 2024
• First Posted: April 11, 2024
• Last Updated: December 27, 2024

Record last verified: 2024-12

Locations

US (1)