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.
Know the risks and potential benefits of clinical studies and talk to your health care provider before participating.
Read our disclaimer for details.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
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
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Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.