Phase 3
Completed N=482
Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)
Heterozygous Familial Hypercholesterolemia · Elevated Cholesterol
Source: ClinicalTrials.gov NCT03397121 ↗
Enrolled (actual)
482
Serious AEs
10.6%
Results posted
Oct 2020
Primary outcomePrimary: Percent Change in LDL-C From Baseline To Day 510 — -41.15; 8.37 percent change — p=<.0001
◆ Published Evidence
Highly cited
809citations · ~135 / year
Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia.
Summary
This is a Phase III, placebo-controlled, double-blind, randomized study in participants with HeFH and elevated LDL-C to evaluate the efficacy, safety, and tolerability of subcutaneous (SC) injection(s) of inclisiran. The study will be multicenter and international.
Linked Publications (2)
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Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia.
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A systematic review and meta-analysis of tolerability, cardiac safety and efficacy of inclisiran for the therapy of hyperlipidemic patients.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change in LDL-C From Baseline To Day 510 |
-41.15; 8.37 | <.0001 sig |
| PRIMARY Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 540 |
-38.08; 6.22 | <0.0001 sig |
| SECONDARY Absolute Change in LDL-C From Baseline to Day 510 |
-58.95; 9.94 | <0.0001 sig |
| SECONDARY Time-adjusted Absolute Change in LDL-C From Baseline After Day 90 and up to Day 540 |
-56.58; 6.17 | <0.0001 sig |
| SECONDARY Percentage Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Day 510 |
-60.68; 17.66 | <0.0001 sig |
| SECONDARY Percentage Change in Total Cholesterol From Baseline to Day 510 |
-25.11; 6.66 | <0.0001 sig |
| SECONDARY Percent Change in Apolipoprotein B (Apo-B) From Baseline To Day 510 |
-33.14; 2.93 | <0.0001 sig |
| SECONDARY Percent Change in Non-high-density Lipoprotein (HDL)-C From Baseline To Day 510 |
-34.93; 7.43 | <0.0001 sig |
Eligibility Criteria
Inclusion Criteria
Participants may be included if they meet all of the following inclusion criteria prior to randomization:
- Male or female participants ≥18 years of age.
- History of HeFH with a diagnosis of HeFH by genetic testing; and/or a documented history of untreated LDL-C of >190 mg/dL, and a family history of familial hypercholesterolemia, elevated cholesterol or early heart disease that may indicate familial hypercholesterolemia.
- Serum LDL-C ≥2.6 millimoles (mmol)/liter (L) (≥100 mg/dL) at screening.
- Fasting triglyceride 2 years postmenopausal (defined as 1 year or longer since last menstrual period) AND more than 55 years of age.
- Postmenopausal women (as defined above) and less than 55 years of age with a negative pregnancy test within 24 hours of randomization.
- Women who are surgically sterilized at least 3 months prior to enrollment.
- Males who are unwilling to use an acceptable method of birth control during the entire study period (condom with spermicide).
- Treatment with other investigational products or devices within 30 days or 5 half-lives of the screening visit, whichever is longer.
- Treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9.
The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Data sourced from ClinicalTrials.gov (NCT03397121) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.