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

Volanesorsen (ISIS 304801) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS).

Study Overview

• Status: No longer available
• Conditions: Familial Chylomicronemia
• Intervention / Treatment: Drug: Volanesorsen

Detailed Description

The Program is intended to provide expanded access to volanesorsen for eligible patients with FCS who have limited or no available treatment options.

• Study Type: Expanded Access
• Expanded Access Type: Individual Patients

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Patients who participated in the APPROACH Open Label trial, must have completed the open label extension trial for volanesorsen for a length of time consistent with the study protocol (≥ 1 year). Patients in the APPROACH open Label trial for less than a year require approval from Akcea to participate in the EAP. Patients who did not participate in the APPROACH open label trial require approval from Akcea for entry into EAP.
• Patients not participating in the APPROACH open label trial must have a diagnosis of FCS as determined by the participating physician, as outlined in the Volanesorsen EAP protocol. Akcea will review each application to determine eligibility.
• Male patients and female patients of childbearing potential must continue to use appropriate contraception with their partners, or refrain from sexual activity

Exclusion Criteria:

• Patients who have any new conditions or worsening of existing conditions which in the opinion of the Physician would make the patient unsuitable for treatment with volanesorsen.
• Volanesorsen naïve patients with baseline platelet values ≤ 140,000/mm3
• Patients not willing to adhere to mandatory blood draws for platelet monitoring
• Patients who in the opinion of Akcea's medical team, are not eligible candidates for volanesorsen therapy.
• Any patient who plans to or becomes pregnant.
• Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Akcea Therapeutics
• Collaborators: CaligorRx, Inc.

Study record dates

Study Registration Dates

• First Submitted: May 21, 2018
• First Submitted That Met QC Criteria: May 21, 2018
• First Posted (Actual): June 1, 2018

Study Record Updates

• Last Update Posted (Actual): September 16, 2021
• Last Update Submitted That Met QC Criteria: September 10, 2021
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Keywords: Genetic Diseases, Inborn; Lipid Metabolism Disorders; Metabolic Diseases; Metabolism, Inborn Errors; Dyslipidemias; Hyperlipidemias; Familial Chylomicronemia; Familial Lipoprotein Lipase Deficiency; Hyperlipoproteinemias; Familial Hyperlipoproteinemia Type 1; Hyperlipoproteinemia Type 1; Hyperchylomicronemia, Familial; Lipoprotein Lipase Deficiency, Familial; Lipid Metabolism, Inborn Errors
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Hyperlipidemias; Dyslipidemias; Lipid Metabolism, Inborn Errors; Hyperlipoproteinemias; Hyperlipoproteinemia Type I
• Other Study ID Numbers: ISIS 304801