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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03544060
Expanded Access Status : No longer available
First Posted : June 1, 2018
Last Update Posted : September 16, 2021
Sponsor:
Collaborator:
CaligorRx, Inc.
Information provided by (Responsible Party):
Akcea Therapeutics

Study Description
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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 Design
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Study Type : Expanded Access
Expanded Access Type : Individual Patients
Official Title: Volanesorsen (ISIS 304801) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Family Issues

Interventions
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Intervention Details:
  • Drug: Volanesorsen
    Volanesorsen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.
    Other Name: Waylivra
Eligibility Criteria
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Information from the National Library of Medicine

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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.
Contacts and Locations
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No Contacts or Locations Provided
More Information
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Responsible Party: Akcea Therapeutics
ClinicalTrials.gov Identifier: NCT03544060    
Other Study ID Numbers: ISIS 304801
First Posted: June 1, 2018    Key Record Dates
Last Update Posted: September 16, 2021
Last Verified: September 2021
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:
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Hyperlipoproteinemia Type I
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
 Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases