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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

APPROVED_FOR_MARKETING

Study Classification

EXPANDED_ACCESS

Brief Summary

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

Detailed Description

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The Expanded Access Program (EAP) is intended to provide pre-approval access to olezarsen for eligible patients with FCS who have limited or no available treatment options. This program is open in the United States and operates under the individual patient (also referred to as single patient) IND expanded access route in which the patient's treating physician serves as the sponsor. Expanded access requests must be from the patient's treating physicians and submitted according to the instructions at https://www.ionispharma.com/patients/expanded-access-policy/

Conditions

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Familial Chylomicronemia Syndrome

Keywords

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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

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Olezarsen

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

Intervention Type DRUG

Other Intervention Names

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ISIS-678354

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Locations

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Expanded Access Site

Carlsbad, California, United States

Site Status

Countries

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United States

Other Identifiers

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ISIS 678354

Identifier Type: -

Identifier Source: org_study_id