Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS) (NCT06360237) | Clinical Trial Compass
APPROVED_FOR_MARKETINGNot Applicable
Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
United States
Plain-language summary
The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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.
Exclusion Criteria:
* 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.