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

Familial Chylomicronemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT03544060
• Other study ID #: ISIS 304801
• Status: No longer available

Study information

• Verified date: September 2021
• Source: Akcea Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial Summary

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

Description:

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

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: All
• Age group: 18 Years and older

Eligibility

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• Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.

Related Conditions & MeSH terms

• Familial Chylomicronemia
• Hyperlipoproteinemia Type I

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Akcea Therapeutics	CaligorRx, Inc.