Familial ChylomicronemiaNCT03544060
Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
A study testing Volanesorsen in people with Familial Chylomicronemia, run by Akcea Therapeutics.
Trial summary
The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Familial Chylomicronemia
- What is being tested?
- Volanesorsen
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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- Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.
About this study
Official title: Volanesorsen (ISIS 304801) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS).
Read the 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.
- Conditions
- Familial Chylomicronemia
- Being tested
- Volanesorsen
- 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
- Medical subject headings
- Hyperlipoproteinemia Type I, Hyperlipoproteinemias, Familial hyperchylomicronemia syndrome, Hyperlipidemias, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VolanesorsenDrug
Also called Waylivra
Volanesorsen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial ChylomicronemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Akcea Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- CaligorRx, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03544060
- Sponsor's protocol ID
- ISIS 304801
- Status last verified
- Sep 2021
- First submitted
- 21 May 2018
- Last updated
- 16 Sep 2021
- Results posted
- No