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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03544060
Expanded Access Status : Available
First Posted : June 1, 2018
Last Update Posted : March 25, 2021
CaligorRx, Inc.
Information provided by (Responsible Party):
Akcea Therapeutics

Tracking Information
First Submitted Date May 21, 2018
First Posted Date June 1, 2018
Last Update Posted Date March 25, 2021
Descriptive Information
Brief Title Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
Official Title Volanesorsen (ISIS 304801) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
Brief Summary The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS).
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
Condition Familial Chylomicronemia
Intervention Drug: Volanesorsen
Volanesorsen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.
Other Name: Waylivra
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Available
Eligibility Criteria

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.
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Contact: Akcea Therapeutics 617-207-0289
Listed Location Countries Not Provided
Removed Location Countries  
Administrative Information
NCT Number NCT03544060
Other Study ID Numbers ISIS 304801
Responsible Party Akcea Therapeutics
Study Sponsor Akcea Therapeutics
Collaborators CaligorRx, Inc.
Investigators Not Provided
PRS Account Akcea Therapeutics
Verification Date March 2021