Back to resultsVolanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
Primary Purpose
Familial Chylomicronemia
Status
No longer available
Phase
Locations
Study Type
Expanded Access
Intervention
Volanesorsen
Sponsored by
About this trial
This is an expanded access trial for Familial Chylomicronemia focused on measuring 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
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- Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT03544060
First Posted
May 21, 2018
Last Updated
September 10, 2021
Sponsor
Akcea Therapeutics
Collaborators
CaligorRx, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03544060
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)
Study Type
Expanded Access
2. Study Status
Record Verification Date
September 2021
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Akcea Therapeutics
Collaborators
CaligorRx, Inc.
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Volanesorsen
Other Intervention Name(s)
Waylivra
Intervention Description
Volanesorsen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
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.
12. IPD Sharing Statement
Learn more about this trial
Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
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