Familial Chylomicronemia
Conditions
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
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.
Interventions
DRUGVolanesorsen
Volanesorsen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.
Sponsors
CaligorRx, Inc.
Akcea Therapeutics
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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 * Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.
Outcome results
None listed