Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
- Conditions
- Familial Chylomicronemia
- Registration Number
- NCT03544060
- Lead Sponsor
- 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).
- 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.
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
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- Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What are the molecular mechanisms of volanesorsen in reducing triglycerides in Familial Chylomicronemia Syndrome (FCS)?
How does volanesorsen compare to standard-of-care treatments for FCS in terms of efficacy and safety profiles?
Which biomarkers are used to select FCS patients for the volanesorsen Early Access Program and predict treatment response?
What are the known adverse events associated with volanesorsen therapy in FCS patients and how are they managed?
Are there any related antisense oligonucleotides or competitor drugs targeting APOC3 for FCS treatment?