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

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ClinicalTrials.gov Identifier: NCT03544060
Expanded Access Status : Available
First Posted : June 1, 2018
Last Update Posted : June 1, 2018
Sponsor:
Collaborator:
CaligorRx, Inc.
Information provided by (Responsible Party):
Akcea Therapeutics

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

Condition or disease Intervention/treatment
Familial Chylomicronemia 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
Official Title: Volanesorsen (ISIS 304801) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)



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

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03544060


Contacts
Contact: Director - Medical Affairs 833-327-8652 volanesorsenEAP@akceatx.com

Sponsors and Collaborators
Akcea Therapeutics
CaligorRx, Inc.
Investigators
Study Director: Rosa Jiminez, MD, PhD Akcea Therapeutics

Responsible Party: Akcea Therapeutics
ClinicalTrials.gov Identifier: NCT03544060     History of Changes
Other Study ID Numbers: ISIS 304801
First Posted: June 1, 2018    Key Record Dates
Last Update Posted: June 1, 2018
Last Verified: May 2018

Keywords provided by Akcea Therapeutics:
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

Additional relevant MeSH terms:
Hyperlipoproteinemia Type I
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases