A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) (BALANCE)
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| ClinicalTrials.gov Identifier: NCT04568434 |
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Recruitment Status :
Completed
First Posted : September 29, 2020
Last Update Posted : March 27, 2024
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Sponsor:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
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Brief Summary:
The purpose of the study is to evaluate the efficacy of Olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Familial Chylomicronemia Syndrome | Drug: Olezarsen Drug: Placebo | Phase 3 |
This is a multi-center, double-blind, Phase 3 study in up to 60 patients with FCS. Participants will be 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 is approximately 74 weeks, which includes 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 may have the option of enrolling in an open label extension study.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 66 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Double (Participant, Investigator) |
| Primary Purpose: | Treatment |
| Official Title: | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS) |
| Actual Study Start Date : | November 16, 2020 |
| Actual Primary Completion Date : | July 14, 2023 |
| Actual Study Completion Date : | October 17, 2023 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Familial lipoprotein lipase deficiency
MedlinePlus related topics:
Family Issues
| Arm | Intervention/treatment |
|---|---|
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Experimental: Olezarsen
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.
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Drug: Olezarsen
Olezarsen will be administered by SC injection.
Other Names:
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Placebo Comparator: Placebo
Olezarsen-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49.
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Drug: Placebo
Olezarsen-matching placebo will be administered by SC injection. |
Primary Outcome Measures :
- Percent Change from Baseline in Fasting TG at 6 Months (average of Weeks 23, 25, and 27) compared to placebo [ Time Frame: Baseline and Month 6 ]
Secondary Outcome Measures :
- Percent Change from Baseline in Fasting TG at 12 Months (average of Weeks 51 and 53) Compared to Placebo [ Time Frame: Baseline and Month 12 ]
- Percent Change in Fasting apoC-III from Baseline at 6 Months and 12 Months Compared to Placebo [ Time Frame: Baseline, Month 6 and Month 12 ]
- Change from Baseline in the Proportion of Participants who Achieve ≥ 40% Reduction in Fasting TG at 6 Months Compared to Placebo [ Time Frame: Baseline and Month 6 ]
- Percent Change from Baseline in Fasting Apolipoprotein B-48 (apoB-48) at 6 Months and 12 Months Compared to Placebo [ Time Frame: Baseline, Month 6 and Month 12 ]
- Percent Change in Fasting non-HDL-C from Baseline at 6 Months and 12 Months Compared to Placebo [ Time Frame: Baseline, Month 6 and Month 12 ]
- Adjudicated Acute Pancreatitis Event Rate During the Treatment Period Compared to Placebo in Patients with a History of Pancreatitis within 10 Years Prior to Screening [ Time Frame: Week 1 through Week 53 and Week 13 through Week 53 ]
- Adjudicated Acute Pancreatitis Event Rate During the Treatment Period Compared to Placebo [ Time Frame: Week 1 through Week 53 and Week 13 through Week 53 ]
- Change from Baseline in the Proportion of Participants Who Achieve ≥ 70% Reduction in Fasting TG at 6 Months Compared to Placebo [ Time Frame: Baseline and Month 6 ]
- Proportion of Participants Who Achieve Fasting TG ≤ 880 mg/dL at 6 Months Compared to Placebo [ Time Frame: Week 1 through Week 53 ]
- Adjudicated Acute Pancreatitis Event Rate During the Treatment Period Compared to Placebo, in Participants with ≥ 2 Events of Adjudicated Acute Pancreatitis in 5 Years Prior to Enrollment [ Time Frame: Week 1 through Week 53 and Week 13 through Week 53 ]
- Proportion of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 6 Months Compared to Placebo [ Time Frame: At Month 6 ]
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Inclusion Criteria:
- 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:
- 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04568434
Locations
Show 49 study locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
| Responsible Party: | Ionis Pharmaceuticals, Inc. |
| ClinicalTrials.gov Identifier: | NCT04568434 |
| Other Study ID Numbers: |
ISIS 678354-CS3 2020-002536-67 ( EudraCT Number ) |
| First Posted: | September 29, 2020 Key Record Dates |
| Last Update Posted: | March 27, 2024 |
| Last Verified: | March 2024 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | Yes |
Keywords provided by Ionis Pharmaceuticals, Inc.:
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FCS |
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type I Syndrome Disease Pathologic Processes Lipid Metabolism, Inborn Errors Metabolism, Inborn Errors |
Genetic Diseases, Inborn Hyperlipoproteinemias Hyperlipidemias Dyslipidemias Lipid Metabolism Disorders Metabolic Diseases |