Familial Chylomicronemia SyndromePhase 2NCT03360747
Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)
A Phase 2 study testing AKCEA-ANGPTL3-LRx in people with Familial Chylomicronemia Syndrome and Lipoprotein Lipase Deficiency, run by Akcea Therapeutics at 1 site in Canada.
Trial summary
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
- What is being tested?
- AKCEA-ANGPTL3-LRx
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteCanada
- Study dates
- 21 Dec 2017to 4 Sep 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Genetically confirmed chylomicronemia syndrome.
- Fasting triglycerides greater than or equal to (>=) 750 milligrams per deciliter (mg/dL) [8.4 millimoles per liter (mmol/L)] at Screening.
Who cannot join
- Diabetes mellitus if newly diagnosed or if glycated hemoglobin (HbA1c) >= 9.0%.
- Active pancreatitis within 2 weeks of screening.
- Acute coronary syndrome within 6 months of screening.
- Major surgery within 3 months of screening.
- Treatment with Glybera therapy within 2 years of screening.
- Previous treatment with AKCEA-ANGPTL3-LRx.
- Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of AKCEA-ANGPTL3-LRx (ISIS 703802) Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.
- Conditions
- Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
- Being tested
- AKCEA-ANGPTL3-LRx
- Keywords
- Familial Lipoprotein Lipase Deficiency, Hyperlipoproteinemias, Familial Hyperlipoproteinemia Type 1, Hyperlipoproteinemia Type I, 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
- Familial hyperchylomicronemia syndrome, Hyperlipoproteinemia Type I, Hyperlipoproteinemias, 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.
AKCEA-ANGPTL3-LRxDrug
Also called ISIS 703802, IONIS-ANGPTL3-LRx, Vupanorsen
AKCEA-ANGPTL3-LRx at dose 20 mg, administered via SC injection QW.
| Study group | Role | What they receive |
|---|---|---|
| AKCEA-ANGPTL3-LRx 20 mg | Experimental | Participants received a subcutaneous (SC) injection of AKCEA-ANGPTL3-LRx, 20 milligrams (mg), weekly (QW) for 13-weeks of treatment period. Participants were followed up to Week 26. Receives: Drug: AKCEA-ANGPTL3-LRx |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Measured: Baseline to Month 3
Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Measured: Baseline to Month 3
Secondary outcomes (8)
Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3)
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Measured: Baseline to Month 3
Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3)
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Measured: Baseline to Month 3
Fasting Lipid and Lipoprotein Measurements at Month 3
Fasting lipid and lipoprotein measurements included non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C and LDL-C. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Measured: Month 3
Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters
Other fasting lipid measurements included total cholesterol (TC), non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Measured: Baseline to Month 3
Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters
Other fasting lipid measurements included TC, non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Measured: Baseline to Month 3
Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG)
Participants consumed standardized pre-cooked meals (lunches and dinners and instructions for breakfasts and snacks) for 2 days prior to the postprandial assessments. Change from Baseline to Day 92 in maximum postprandial TG was assessed.
Measured: Baseline to Day 92
Number of Participants Who Experienced Abdominal Pain During the Treatment Period
Measured: Days 1, 29, 57 and 92
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any unfavorable and unintended sign (including a clinically-significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product. A TEAE was defined as any AE starting on or after the first dose of the study drug.
Measured: From time of informed consent to end of follow-up period (Up to Week 26)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2017
- Study start21 Dec 2017
- Primary completion12 Jun 2018
- Study completion4 Sep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Familial Chylomicronemia SyndromeStudies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Akcea Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03360747
- Sponsor's protocol ID
- ISIS 703802-CS3
- Status last verified
- Dec 2020
- First submitted
- 18 Nov 2017
- Last updated
- 7 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of AKCEA-ANGPTL3-LRx (ISIS 703802) Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
- ClinicalTrials.gov ID
- NCT03360747
- Acronym / sponsor ID
- ISIS 703802-CS3
- Conditions
- Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
- Keywords
- Familial Lipoprotein Lipase Deficiency, Hyperlipoproteinemias, Familial Hyperlipoproteinemia Type 1, Hyperlipoproteinemia Type I, Hyperchylomicronemia, Familial, Lipoprotein Lipase Deficiency, Familial, Hyperlipidemias, Dyslipidemias
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Akcea TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Study start
- 21 Dec 2017 (actual)
- Primary completion
- 12 Jun 2018 (actual)
- Study completion
- 4 Sep 2018 (actual)
- First posted
- 4 Dec 2017 (actual)
- Results
- Posted 7 Jan 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AKCEA-ANGPTL3-LRx 20 mg | Experimental | Drug: AKCEA-ANGPTL3-LRxParticipants received a subcutaneous (SC) injection of AKCEA-ANGPTL3-LRx, 20 milligrams (mg), weekly (QW) for 13-weeks of treatment period. Participants were followed up to Week 26. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Genetically confirmed chylomicronemia syndrome.
- Fasting triglycerides greater than or equal to (>=) 750 milligrams per deciliter (mg/dL) [8.4 millimoles per liter (mmol/L)] at Screening.
Key exclusion criteria (7 listed)
- Diabetes mellitus if newly diagnosed or if glycated hemoglobin (HbA1c) >= 9.0%.
- Active pancreatitis within 2 weeks of screening.
- Acute coronary syndrome within 6 months of screening.
- Major surgery within 3 months of screening.
- Treatment with Glybera therapy within 2 years of screening.
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 |
|---|---|---|
| AKCEA-ANGPTL3-LRxAlso: ISIS 703802, IONIS-ANGPTL3-LRx, VupanorsenAKCEA-ANGPTL3-LRx at dose 20 mg, administered via SC injection QW. | Drug | AKCEA-ANGPTL3-LRx 20 mg |
Primary outcome
Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)
Time frame: Baseline to Month 3
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)
Time frame: Baseline to Month 3
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
8 secondary outcomes
Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3)
Time frame: Baseline to Month 3
Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3)
Time frame: Baseline to Month 3
Fasting Lipid and Lipoprotein Measurements at Month 3
Time frame: Month 3
Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters
Time frame: Baseline to Month 3
Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters
Time frame: Baseline to Month 3
Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG)
Time frame: Baseline to Day 92
Number of Participants Who Experienced Abdominal Pain During the Treatment Period
Time frame: Days 1, 29, 57 and 92
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: From time of informed consent to end of follow-up period (Up to Week 26)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects
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A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AKCEA-ANGPTL3-LRx in people with Familial Chylomicronemia Syndrome and Lipoprotein Lipase Deficiency, run by Akcea Therapeutics at 1 site in Canada.
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 2 inclusion and 7 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: finished, as of the sponsor's last update on 7 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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.
What does the primary outcome measure?
Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG) (time frame: Baseline to Month 3). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
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 7 Jan 2021.