Familial Chylomicronemia SyndromePhase 3BALANCENCT04568434
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
A Phase 3 study testing Olezarsen in people with Familial Chylomicronemia Syndrome, run by Ionis Pharmaceuticals, Inc. at 47 sites in 11 countries.
Trial summary
The purpose of the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Familial Chylomicronemia Syndrome
- What is being tested?
- Olezarsen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 66 participants
- Where
- 47 sitesin 11 countries
- Study dates
- 18 Nov 2020to 17 Oct 2023
- Phase
- Phase 3
- 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
- A diagnosis of genetically confirmed Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia)
- Fasting TG ≥ 880 mg/dL (10 millimoles per liter (mmol/L) at Screening
- History of pancreatitis. Patients without a documented history of pancreatitis are also eligible but their enrollment will be capped at 35%
- Stable doses of statins, omega-3 fatty acids, fibrates, or other lipid-lowering medications are allowed
Who cannot join
- Acute coronary syndrome within 6 months of Screening
- Major surgery within 3 months of Screening
- Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion, or could interfere with participating in or completing the study
Trial locations and contacts
47 sites in 11 countries.
47 sites in 11 countries
United States17 sites
University of Michigan- Endocrinology & Metabolism
Ann Arbor, Michigan, 48105
Excel Medical Clinical Trials, LLC
Boca Raton, Florida, 33434
University of Texas Southwestern
Dallas, Texas, 75390
NorthShore University Health System
Evanston, Illinois, 60201
Moses H. Cone Memorial Hospital
Greensboro, North Carolina, 27401
Baylor College of Medicine
Houston, Texas, 77030
Diabetes/Lipid Management & Research Center
Huntington Beach, California, 92648
Ascension St. Vincent Cardiovascular Research Institute
Indianapolis, Indiana, 46290
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66160
West Virginia University Heart and Vascular Institute
Morgantown, West Virginia, 26506
Vanderbilt University
Nashville, Tennessee, 37232
New York University (NYU) Langone Medical Center
New York, New York, 10016
Columbia University Medical Center
New York, New York, 10032
York Clinical Research LLC
Norfolk, Virginia, 23510
Advocate Health and Hospitals Corporation - Lutheran General Hospital
Park Ridge, Illinois, 60068
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of California, San Francisco (UCSF) - Medical Center
San Francisco, California, 94143
Spain5 sites
Hospital Abente y Lago
A Coruña, 15001
Hospital Clinic Barcelona
Barcelona, 08036
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital Universitario Virgen del Rocio
Seville, 41013
Fundacio Pere Virgili
Tarragona, 43204
Canada4 sites
Ecogene-21
Chicoutimi, Quebec, G7H 7K9
Nathalie Saint-Pierre
Montreal, Quebec, H2W 1R7
Institute de Recherches Cliniques de Montreal
Montreal, H2W 1R7
Clinique des Maladies Lipidiques de Quebec Inc.
Québec, Quebec, G1V 4W2
Italy4 sites
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162
Azienda Ospedaliera Universitaria Federico II
Naples, 80131
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, 90127
Azienda Ospedaliero Universitaria Policlinico Umberto I
Roma, 00161
United Kingdom4 sites
The Royal Free Hospital
London, NW3 2QG
St. Thomas' Hospital
London, SE1 7EH
Manchester University NHS Foundation Trust (MFT)
Manchester, MI39WL
Sandwell General Hospital
West Bromwich, B71 4HJ
France3 sites
Hôpital Louis Pradel - HCL
Bron, 69677
CHU Dijon - Bocage
Dijon, 21000
Assistance Publique - Hopitaux de Marseille
Marseille, 13385
Netherlands3 sites
Academic Medical Center - Department of Vascular Medicine
Amsterdam, 1105 AZ
Erasmus MC
Rotterdam, 3015 GD
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
Portugal3 sites
Centro Hospitalar de Lisboa Ocidental. E.P.E, - Hospital Santa Cruz
Carnaxide, 2790-134
Hospital da Senhora da Oliveira - Guimaraes
Creixomil, 4835-044
Centro Hospitalar de Lisboa Ocidental, EPE - Hospital Egas Moniz
Lisbon, 1349-019
Sweden2 sites
Sahlgrenska University Hospital
Gothenburg, 413 45
Karolinska University Hospital
Solna, 171 76
Slovakia1 site
Metabolicke centrum MUDr Katariny Raslovej s. r. o.
Bratislava, 83301
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
Read the detailed description
This was a multi-center, double-blind, Phase 3 study in up to 60 patients with FCS. Participants were randomized in a 2:1 ratio to receive Olezarsen or matching placebo in a 53-week treatment period. The length of participation in the study was approximately 74 weeks, which included an up to 8-week screening period, a 53-week treatment period, and a 13-week post-treatment evaluation period. Following the treatment period, eligible patients had the option to enroll in the Open-label Extension (OLE) Study ISIS 678354-CS13 (NCT05130450).
- Conditions
- Familial Chylomicronemia Syndrome
- Being tested
- Olezarsen, Placebo
- Keywords
- FCS
- Medical subject headings
- Familial hyperchylomicronemia syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OlezarsenDrug
Also called ISIS 678354, AKCEA-APOCIII-LRx
Olezarsen was administered by SC injection.
PlaceboDrug
Olezarsen-matching placebo was administered by SC injection.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Participants received olezarsen-matching placebo, once every 4 weeks by subcutaneous (SC) injection, during Weeks 1 to 49 of the 53-week treatment period. Receives: Drug: Placebo |
| Olezarsen 50 mg | Experimental | Participants received olezarsen, 50 milligrams (mg), once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period. Receives: Drug: Olezarsen |
| Olezarsen 80 mg | Experimental | Participants received olezarsen 80 mg, once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period. Receives: Drug: Olezarsen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline in Fasting TG at Month 6
Measured: Baseline, Month 6
Secondary outcomes (14)
Percent Change From Baseline in Fasting TG at Month 12
Measured: Baseline, Month 12
Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12
Measured: Baseline, Months 6 and 12
Percentage of Participants With ≥ 40% Reduction in Fasting TG at Month 6
Percentages are rounded off to the nearest single decimal place.
Measured: Month 6
Percent Change From Baseline in Fasting Apolipoprotein B-48 (apoB-48) at Months 6 and 12
Measured: Baseline, Months 6 and 12
Percent Change From Baseline in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Months 6 and 12
Measured: Baseline, Months 6 and 12
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53) in Participants With Prior History of Pancreatitis
All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the Acute Pancreatitis Adjudication Committee (PAC) Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the treatment period.
Measured: During the treatment period Week 1 through Week 53
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53)
All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the PAC Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the treatment period.
Measured: During the treatment period Week 1 through Week 53
Adjudicated Acute Pancreatitis Mean Event Per 100 Participant-Years Rate During Week 13 Through Week 53 in Participants With Prior History of Pancreatitis
All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the PAC Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the specified duration.
Measured: Week 13 through Week 53
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Week 13 to Week 53
All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the PAC Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the specified duration.
Measured: Week 13 through Week 53
Percentage of Participants With ≥ 70% Reduction in Fasting TG at Month 6
Percentages are rounded off to the nearest single decimal place.
Measured: Month 6
Percentage of Participants With Fasting TG ≤ 880 mg/dL at Month 6
Percentages are rounded off to the nearest single decimal place.
Measured: Month 6
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Treatment Period in Participants With ≥ 2 Events in 5 Years Prior to Enrollment
All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the PAC Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the treatment period.
Measured: During the treatment period Week 1 through Week 53
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years From Week 13 to Week 53 in Participants With ≥ 2 Events in 5 Years Prior to Enrollment
All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the PAC Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the specified duration.
Measured: Week 13 to Week 53
Percentage of Participants With Fasting TG ≤ 500 mg/dL at Month 6
Percentages are rounded off to the nearest single decimal place.
Measured: Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2020
- Study start18 Nov 2020
- Primary completion14 Jul 2023
- Study completion17 Oct 2023
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.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Familial Chylomicronemia SyndromeStudies of the same condition, open and under way.
- More trials run by Ionis Pharmaceuticals, Inc.The sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 66 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04568434
- Sponsor's protocol ID
- ISIS 678354-CS3
- Other IDs
- 2020-002536-67
- Acronym
- BALANCE
- Status last verified
- Feb 2025
- First submitted
- 23 Sep 2020
- Last updated
- 6 Mar 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.
- Expanded access
- Available
Publications
Stroes ESG, Alexander VJ, Karwatowska-Prokopczuk E, Hegele RA, Arca M, Ballantyne CM, Soran H, Prohaska TA, Xia S, Ginsberg HN, Witztum JL, Tsimikas S; Balance Investigators. Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome. N Engl J Med. 2024 May 16;390(19):1781-1792. doi: 10.1056/NEJMoa2400201. Epub 2024 Apr 7.
PubMed 38587247Sikora Kessler A, Vera-Llonch M, Karwatowska-Prokopczuk E, Alexander VJ, Ann Q, Mejia Herrera C, Paparrodopoulos S, Xia S, Stroes ESG, Baum SJ, Tsimikas S; Balance Investigators. Olezarsen reduces all-cause health services utilization and improves the treatment experience of patients with familial chylomicronemia syndrome. J Clin Lipidol. 2026 Feb;20(2):299-309. doi: 10.1016/j.jacl.2025.12.018. Epub 2025 Dec 19.
PubMed 41545246
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
- ClinicalTrials.gov ID
- NCT04568434
- Acronym / sponsor ID
- BALANCE / ISIS 678354-CS3
- Conditions
- Familial Chylomicronemia Syndrome
- Keywords
- FCS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 66 participants (actual)
- Lead sponsor
- Ionis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 18 Nov 2020 (actual)
- Primary completion
- 14 Jul 2023 (actual)
- Study completion
- 17 Oct 2023 (actual)
- First posted
- 29 Sep 2020 (actual)
- Results
- Posted 6 Mar 2025
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 the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
Detailed description
This was a multi-center, double-blind, Phase 3 study in up to 60 patients with FCS. Participants were randomized in a 2:1 ratio to receive Olezarsen or matching placebo in a 53-week treatment period. The length of participation in the study was approximately 74 weeks, which included an up to 8-week screening period, a 53-week treatment period, and a 13-week post-treatment evaluation period. Following the treatment period, eligible patients had the option to enroll in the Open-label Extension (OLE) Study ISIS 678354-CS13 (NCT05130450).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboParticipants received olezarsen-matching placebo, once every 4 weeks by subcutaneous (SC) injection, during Weeks 1 to 49 of the 53-week treatment period. |
| Olezarsen 50 mg | Experimental | Drug: OlezarsenParticipants received olezarsen, 50 milligrams (mg), once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period. |
| Olezarsen 80 mg | Experimental | Drug: OlezarsenParticipants received olezarsen 80 mg, once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- A diagnosis of genetically confirmed Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia)
- Fasting TG ≥ 880 mg/dL (10 millimoles per liter (mmol/L) at Screening
- History of pancreatitis. Patients without a documented history of pancreatitis are also eligible but their enrollment will be capped at 35%
- Stable doses of statins, omega-3 fatty acids, fibrates, or other lipid-lowering medications are allowed
Key exclusion criteria (3 listed)
- Acute coronary syndrome within 6 months of Screening
- Major surgery within 3 months of Screening
- Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion, or could interfere with participating in or completing the study
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 |
|---|---|---|
| OlezarsenAlso: ISIS 678354, AKCEA-APOCIII-LRxOlezarsen was administered by SC injection. | Drug | Olezarsen 50 mg, Olezarsen 80 mg |
| PlaceboOlezarsen-matching placebo was administered by SC injection. | Drug | Placebo |
Primary outcome
Percent Change From Baseline in Fasting TG at Month 6
Time frame: Baseline, Month 6
14 secondary outcomes
Percent Change From Baseline in Fasting TG at Month 12
Time frame: Baseline, Month 12
Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12
Time frame: Baseline, Months 6 and 12
Percentage of Participants With ≥ 40% Reduction in Fasting TG at Month 6
Time frame: Month 6
Percent Change From Baseline in Fasting Apolipoprotein B-48 (apoB-48) at Months 6 and 12
Time frame: Baseline, Months 6 and 12
Percent Change From Baseline in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Months 6 and 12
Time frame: Baseline, Months 6 and 12
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53) in Participants With Prior History of Pancreatitis
Time frame: During the treatment period Week 1 through Week 53
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53)
Time frame: During the treatment period Week 1 through Week 53
Adjudicated Acute Pancreatitis Mean Event Per 100 Participant-Years Rate During Week 13 Through Week 53 in Participants With Prior History of Pancreatitis
Time frame: Week 13 through Week 53
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Week 13 to Week 53
Time frame: Week 13 through Week 53
Percentage of Participants With ≥ 70% Reduction in Fasting TG at Month 6
Time frame: Month 6
Percentage of Participants With Fasting TG ≤ 880 mg/dL at Month 6
Time frame: Month 6
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Treatment Period in Participants With ≥ 2 Events in 5 Years Prior to Enrollment
Time frame: During the treatment period Week 1 through Week 53
Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years From Week 13 to Week 53 in Participants With ≥ 2 Events in 5 Years Prior to Enrollment
Time frame: Week 13 to Week 53
Percentage of Participants With Fasting TG ≤ 500 mg/dL at Month 6
Time frame: Month 6
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Mar 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 38587247Stroes ESG, Alexander VJ, Karwatowska-Prokopczuk E, Hegele RA, Arca M, Ballantyne CM, Soran H, Prohaska TA, Xia S, Ginsberg HN, Witztum JL, Tsimikas S; Balance Investigators. Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome. N Engl J Med. 2024 May 16;390(19):1781-1792. doi: 10.1056/NEJMoa2400201. Epub 2024 Apr 7.
- Derived from this study: PubMed 41545246Sikora Kessler A, Vera-Llonch M, Karwatowska-Prokopczuk E, Alexander VJ, Ann Q, Mejia Herrera C, Paparrodopoulos S, Xia S, Stroes ESG, Baum SJ, Tsimikas S; Balance Investigators. Olezarsen reduces all-cause health services utilization and improves the treatment experience of patients with familial chylomicronemia syndrome. J Clin Lipidol. 2026 Feb;20(2):299-309. doi: 10.1016/j.jacl.2025.12.018. Epub 2025 Dec 19.
- Individual participant dataSharing planned: Yes. Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.
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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.
47 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Olezarsen in people with Familial Chylomicronemia Syndrome, run by Ionis Pharmaceuticals, Inc. at 47 sites in 11 countries.
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 4 inclusion and 3 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 6 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 47 sites in United States, Spain, Canada, Italy, United Kingdom and 6 more countries. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 6 Mar 2025). 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?
Percent Change From Baseline in Fasting TG at Month 6 (time frame: Baseline, Month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04568434, which also lists 2 linked publications.
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 6 Mar 2025.