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
- Are there results?
- None posted in the registry
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- Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy.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
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Volanesorsen (ISIS 304801) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
- ClinicalTrials.gov ID
- NCT03544060
- Acronym / sponsor ID
- ISIS 304801
- Conditions
- Familial Chylomicronemia
- Keywords
- Familial Chylomicronemia, Familial Lipoprotein Lipase Deficiency, Hyperlipoproteinemias, Familial Hyperlipoproteinemia Type 1, Hyperlipoproteinemia Type 1, Hyperchylomicronemia, Familial, Lipoprotein Lipase Deficiency, Familial, Hyperlipidemias
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Akcea TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- CaligorRx, Inc.
- First posted
- 1 Jun 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VolanesorsenAlso: WaylivraVolanesorsen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Volanesorsen in people with Familial Chylomicronemia, run by Akcea Therapeutics.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2021.