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

NCT03544060 | Unknown

• 1 other identifier: ISIS 304801
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Familial Chylomicronemia

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

Volanesorsen DRUG

Volanesorsen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.

Also known as: Waylivra

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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• Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.

Sponsors & Collaborators

• Akcea Therapeutics: lead
• CaligorRx, Inc.: collaborator

MeSH Terms

Conditions

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

Interventions

ISIS 304801

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: May 21, 2018
• First Posted: June 1, 2018
• Last Updated: September 16, 2021

Record last verified: 2021-09