Phase 3
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
Bottom Line
View on ClinicalTrials.gov: NCT03397121 ↗Enrolled (actual)
482
Serious AEs
10.6%
Results posted
Oct 2020
Primary outcome: Primary: Percent Change in LDL-C From Baseline To Day 510 — -41.15; 8.37 percent change — p=<.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Inclisiran (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- The Medicines Company
- Primary completion
- Aug 2019
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 |
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.
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). 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.