A Phase 3 interventional study of evinacumab and Placebo in Homozygous Familial Hypercholesterolemia, sponsored by Regeneron Pharmaceuticals. Completed at 30 sites in 11 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2021-05-18.
Sponsored by Regeneron Pharmaceuticals · Phase 3, Interventional, and Treatment
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Among 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States — 7 open to participants now, 9 under way, 4 of them phase 3. Homozygous Familial Hypercholesterolemia · United States.
Among the 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States, this one is the 11th largest by enrollment of the 31 interventional ones that give a count, is one of 3 that started in 2018, is one of 16 in phase 3, and was the 20th registered.
68 studies on the registry are indexed under Homozygous Familial Hypercholesterolemia; 13 are open to participants now.
Browse Homozygous Familial Hypercholesterolemia studies →7 of the 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States are open now. Recruiting first, the newest first:
Arrowhead Pharmaceuticals · Sites in United States, Denmark, Poland
Ages 12–17
University of Wisconsin, Madison · Sites in United States
Ages 18+
Novartis Pharmaceuticals · Sites in United States, Austria, China and 8 more countries
Ages 2–11
CRISPR Therapeutics AG · Sites in United States, Australia, New Zealand and 1 more country
Ages 18–75
Novartis Pharmaceuticals · Sites in United States, Argentina, Brazil and 23 more countries
Ages 2–100
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 12 Homozygous Familial Hypercholesterolemia studies recruiting now, 6 (50%) set no upper age limit and 5 (42%) stop at 75 or younger; 8 take children under 18; none take healthy volunteers.
This study: 12 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
36 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 2 · Phase 2 11 · Phase 3 18
First in 2003; this study’s year is marked. 3 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 65 is above the median of 21 across 51 interventional studies indexed under Homozygous Familial Hypercholesterolemia.
Median enrollment 29 across the 31 interventional studies, 60 across the 5 observational. Of the 26 that completed or were terminated, 19 (73%) have results posted.
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32 of 36 have an industry lead sponsor. The 10 most frequent:
Regeneron Pharmaceuticals is the lead sponsor of 412 studies on the registry; 90 are open to participants now.
Of its 126 completed or terminated interventional studies of FDA-regulated products, 79 (63%) have results posted.
Regeneron Pharmaceuticals has 5 studies filed under Homozygous Familial Hypercholesterolemia — fewer than ten, so there is no comparison to draw.
3 of the studies in Homozygous Familial Hypercholesterolemia with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07037771Phase 3Arrowhead PharmaceuticalsA Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE) | 2027-08-20 |
| NCT04815005University of PennsylvaniaHoFH, the International Clinical Collaborators Registry | 2026-12-31 |
| NCT07491172CRISPR Therapeutics AGA Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias | 2027-06 |
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Among the 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial hypercholesterolemia (HoFH). The secondary objectives of the study are to evaluate the effect of evinacumab IV on other lipid parameters, evaluate the effect of evinacumab on LDL-C goal attainment, assess the effect of evinacumab on eligibility for apheresis (using German and US apheresis criteria), evaluate the safety and tolerability of evinacumab in patients with HoFH, assess the pharmacokinetics (PK) of evinacumab in patients with HoFH and evaluate the potential development of anti-evinacumab antibodies.
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other protocol defined inclusion/exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
TIA (transient ischaemic attack)
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: evinacumab
Drug: Placebo
IV administration of evinacumab
Also known as: REGN1500
IV administration of placebo
Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (Intent-to-Treat [ITT] Estimand)
Percent change was calculated as 100x(calculated LDL-C value at Week 24 - calculated LDL-C value at baseline)/calculated LDL-C value at baseline. The baseline LDL-C value was the last calculated LDL-C value obtained before the first dose of double-blind-study drug. The calculated LDL-C at week 24 was the LDL-C value obtained within the week 24 efficacy analysis window, regardless of adherence to treatment and subsequent therapies (intent-to-treat \[ITT\] estimand). The ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand)
Percent change in Apo B from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand)
Percent change from baseline in non-HDL-C at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Percent Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand)
Percent change in TC from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Percentage of Participants With ≥30% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand)
Percentage of participants who achieved reduction in calculated LDL-C ≥30% at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: At Week 24
Percentage of Participants With ≥50% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand)
Percentage of participants who achieved reduction in calculated LDL-C ≥ 50% at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: At Week 24
Absolute Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (ITT Estimand)
Absolute change in calculated LDL-C from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Percentage of Participants Who Met United States (US) Apheresis Eligibility Criteria at Week 24 (ITT Estimand)
US apheresis eligibility criteria included participants who had inadequate response to diet and LMTs after 6 months of treatment and with functional Homozygous familial hypercholesterolemia (HoFH) or Heterozygous familial hypercholesterolemia (HeFH) (with 0-1 risk factor) with LDL-C ≥ 300 mg/dL (7.77 mmol/L). Percentage of participants who met US apheresis eligibility criteria at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: At Week 24
Percentage of Participants With Low-Density Lipoprotein Cholesterol (LDL-C) <100 Milligrams Per Deciliter (mg/dL) (2.59 Millimoles Per Liter [mmol/L]) at Week 24 (ITT Estimand)
Percentage of participants with LDL-C value \<100 mg/dL (2.59 mmol/L) in the DBTP at Week 24 was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analysed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: At Week 24
Percentage of Participants Who Met European Union (EU) Apheresis Eligibility Criteria at Week 24 (ITT Estimand)
EU apheresis eligibility criteria included participants who had inadequate response to diet and Lipid modifying therapies (LMTs) after 3 months of treatment, Primary prevention: Participants with Familial hypercholesterolemia (FH) with LDL-C \>160 mg/dL (4.2 mmol/L) and Cardiovascular (CV) events in close relatives. Secondary prevention: Participants with progressive CV events with LDL-C \> 120 to 130 mg/dL (3.1-3.4 mmol/L). Percentage of participants who met EU apheresis eligibility criteria at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: At Week 24
Percentage of Participants With Calculated Low-Density Lipoprotein Cholesterol (LDL-C) <70 mg/dL (1.81 mmol/L) at Week 24 (ITT Estimand)
Percentage of participants with LDL-C \<70 mg/dL (1.81 mmol/L) at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: At Week 24
Percent Change in Fasting Triglycerides (TG) From Baseline to Week 24 (ITT Estimand)
Percent change from baseline in TG at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24 (ITT Estimand)
Percent change in Lp(a) from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Absolute Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand)
Absolute change in Apo B from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Absolute Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand)
Absolute change in non-HDL-C from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Absolute Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand)
Absolute change in TC from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
Percent Change in Apolipoprotein CIII (Apo CIII) From Baseline to Week 24 (ITT Estimand)
Percent change in Apo CIII from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
Time frame: Week 24
This study was conducted at 30 centers that enrolled participants in 11 countries in Europe, Asia, North America, and Australia. Randomization was stratified by apheresis treatment status and by region (Japan, Rest of the World \[ROW\]).
| Milestone | Placebo IV Q4W (DBTP) | Evinacumab 15 mg/kg (DBTP) | Placebo IV Q4W (OLTP) | Evinacumab 15 mg/kg (OLTP) |
|---|---|---|---|---|
| Started | 22 | 43 | 0 | 0 |
| Completed | 21 | 43 | 0 | 0 |
| Not completed | 1 | 0 | 0 | 0 |
| Withdrew: Withdrawal by subject | 1 | 0 | 0 | 0 |
| Milestone | Placebo IV Q4W (DBTP) | Evinacumab 15 mg/kg (DBTP) | Placebo IV Q4W (OLTP) | Evinacumab 15 mg/kg (OLTP) |
|---|---|---|---|---|
| Started | 0 | 0 | 20 | 44 |
| Completed | 0 | 0 | 19 | 43 |
| Not completed | 0 | 0 | 1 | 1 |
| Withdrew: Noncompliance with protocol by the participant | 0 | 0 | 1 | 0 |
| Withdrew: Pregnancy | 0 | 0 | 0 | 1 |
Percent change was calculated as 100x(calculated LDL-C value at Week 24 - calculated LDL-C value at baseline)/calculated LDL-C value at baseline. The baseline LDL-C value was the last calculated LDL-C value obtained before the first dose of double-blind-study drug. The calculated LDL-C at week 24 was the LDL-C value obtained within the week 24 efficacy analysis window, regardless of adherence to treatment and subsequent therapies (intent-to-treat \[ITT\] estimand). The ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percent Change | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (Intent-to-Treat [ITT] Estimand) | 1.9 ± 6.5 | -47.1 ± 4.6 |
Percent change in Apo B from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percent Change | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand) | -4.5 ± 4.8 | -41.4 ± 3.3 |
Percent change from baseline in non-HDL-C at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percent Change | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand) | 2.0 ± 5.4 | -49.7 ± 3.8 |
Percent change in TC from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percent Change | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percent Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand) | 1.0 ± 4.2 | -47.4 ± 3.0 |
Percentage of participants who achieved reduction in calculated LDL-C ≥30% at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percentage of Participants | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percentage of Participants With ≥30% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand) | 18.2 | 83.7 |
Percentage of participants who achieved reduction in calculated LDL-C ≥ 50% at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percentage of Participants | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percentage of Participants With ≥50% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand) | 4.5 | 55.8 |
Absolute change in calculated LDL-C from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Milligrams per Deciliter (mg/dL) | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Absolute Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (ITT Estimand) | -2.6 ± 17.6 | -134.7 ± 12.4 |
US apheresis eligibility criteria included participants who had inadequate response to diet and LMTs after 6 months of treatment and with functional Homozygous familial hypercholesterolemia (HoFH) or Heterozygous familial hypercholesterolemia (HeFH) (with 0-1 risk factor) with LDL-C ≥ 300 mg/dL (7.77 mmol/L). Percentage of participants who met US apheresis eligibility criteria at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percentage of Participants | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percentage of Participants Who Met United States (US) Apheresis Eligibility Criteria at Week 24 (ITT Estimand) | 22.7 | 7.0 |
Percentage of participants with LDL-C value \<100 mg/dL (2.59 mmol/L) in the DBTP at Week 24 was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analysed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percentage of Participants | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percentage of Participants With Low-Density Lipoprotein Cholesterol (LDL-C) <100 Milligrams Per Deciliter (mg/dL) (2.59 Millimoles Per Liter [mmol/L]) at Week 24 (ITT Estimand) | 22.7 | 46.5 |
EU apheresis eligibility criteria included participants who had inadequate response to diet and Lipid modifying therapies (LMTs) after 3 months of treatment, Primary prevention: Participants with Familial hypercholesterolemia (FH) with LDL-C \>160 mg/dL (4.2 mmol/L) and Cardiovascular (CV) events in close relatives. Secondary prevention: Participants with progressive CV events with LDL-C \> 120 to 130 mg/dL (3.1-3.4 mmol/L). Percentage of participants who met EU apheresis eligibility criteria at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percentage of Participants | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percentage of Participants Who Met European Union (EU) Apheresis Eligibility Criteria at Week 24 (ITT Estimand) | 77.3 | 32.6 |
Percentage of participants with LDL-C \<70 mg/dL (1.81 mmol/L) at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percentage of Participants | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percentage of Participants With Calculated Low-Density Lipoprotein Cholesterol (LDL-C) <70 mg/dL (1.81 mmol/L) at Week 24 (ITT Estimand) | 4.5 | 27.9 |
Percent change from baseline in TG at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percent Change | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percent Change in Fasting Triglycerides (TG) From Baseline to Week 24 (ITT Estimand) | -4.6 ± 7.0 | -55.0 ± 3.1 |
Percent change in Lp(a) from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percent Change | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24 (ITT Estimand) | -3.6 ± 5.8 | -5.5 ± 4.0 |
Absolute change in Apo B from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| mg/dL | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Absolute Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand) | -8.0 ± 9.1 | -74.4 ± 6.3 |
Absolute change in non-HDL-C from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| mg/dL | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Absolute Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand) | -0.4 ± 17.4 | -148.0 ± 12.3 |
Absolute change in TC from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| mg/dL | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Absolute Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand) | -0.4 ± 17.2 | -161.6 ± 12.2 |
Percent change in Apo CIII from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Percent Change | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W |
|---|---|---|
| Percent Change in Apolipoprotein CIII (Apo CIII) From Baseline to Week 24 (ITT Estimand) | 5.8 ± 5.5 | -84.1 ± 3.9 |
Collected over All Adverse Events (AEs) were collected from signature of the informed consent form up until end of study (Week 68) regardless of seriousness or relationship to investigational product (IP).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo IV Q4W (DBTP) | 0/21 (0%) | 0/21 (0%) | 13/21 (61.9%) |
| Evinacumab 15 mg/kg (DBTP) | 0/44 (0%) | 2/44 (4.5%) | 20/44 (45.5%) |
| Placebo IV Q4W (OLTP) | 0/20 (0%) | 0/20 (0%) | 12/20 (60%) |
| Evinacumab 15 mg/kg (OLTP) | 0/44 (0%) | 7/44 (15.9%) | 19/44 (43.2%) |
| Event | Placebo IV Q4W (DBTP) | Evinacumab 15 mg/kg (DBTP) | Placebo IV Q4W (OLTP) | Evinacumab 15 mg/kg (OLTP) |
|---|---|---|---|---|
| Acute myocardial infarctionCardiac disorders | 0/21 | 0/44 | 0/20 | 1/44 |
| Angina pectorisCardiac disorders | 0/21 | 0/44 | 0/20 | 1/44 |
| Angina unstableCardiac disorders | 0/21 | 0/44 | 0/20 | 1/44 |
| Cardiac failure congestiveCardiac disorders | 0/21 | 0/44 | 0/20 | 1/44 |
| Coronary artery diseaseCardiac disorders | 0/21 | 0/44 | 0/20 | 1/44 |
| GlaucomaEye disorders | 0/21 | 0/44 | 0/20 | 1/44 |
| PyelonephritisInfections and infestations | 0/21 | 0/44 | 0/20 | 1/44 |
| UrosepsisInfections and infestations | 0/21 | 1/44 | 0/20 | 0/44 |
| Cardiac procedure complicationInjury, poisoning and procedural complications | 0/21 | 0/44 | 0/20 | 1/44 |
| Carotid artery restenosisInjury, poisoning and procedural complications | 0/21 | 0/44 | 0/20 | 1/44 |
| Event | Placebo IV Q4W (DBTP) | Evinacumab 15 mg/kg (DBTP) | Placebo IV Q4W (OLTP) | Evinacumab 15 mg/kg (OLTP) |
|---|---|---|---|---|
| NasopharyngitisInfections and infestations | 5/21 | 7/44 | 1/20 | 5/44 |
| HeadacheNervous system disorders | 5/21 | 4/44 | 1/20 | 5/44 |
| Influenza like illnessGeneral disorders | 0/21 | 5/44 | 0/20 | 2/44 |
| Urinary tract infectionInfections and infestations | 2/21 | 0/44 | 0/20 | 1/44 |
| ToothacheGastrointestinal disorders | 2/21 | 2/44 | 1/20 | 1/44 |
| Aspartate aminotransferase increasedInvestigations | 2/21 | 0/44 | 0/20 | 0/44 |
| MyalgiaMusculoskeletal and connective tissue disorders | 2/21 | 0/44 | 0/20 | 0/44 |
| NauseaGastrointestinal disorders | 1/21 | 1/44 | 0/20 | 3/44 |
| Back painMusculoskeletal and connective tissue disorders | 0/21 | 1/44 | 0/20 | 3/44 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 0/21 | 3/44 | 0/20 | 0/44 |
| Age, Continuous(Years) | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W | Total |
|---|---|---|---|
| Mean | 36.7 ± 11.52 | 44.3 ± 16.78 | 41.7 ± 15.54 |
| Age, Customized(Participants) | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W | Total |
|---|---|---|---|
| ≥12 years to <18 years of age | 1 | 1 | 2 |
| ≥18 years to <45 years of age | 16 | 23 | 39 |
| ≥45 years to <65 years of age | 5 | 11 | 16 |
| ≥65 years to <75 years of age | 0 | 7 | 7 |
| ≥75 years of age | 0 | 1 | 1 |
| Sex: Female, Male(Participants) | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W | Total |
|---|---|---|---|
| Female | 11 | 24 | 35 |
| Male | 11 | 19 | 30 |
| Ethnicity (NIH/OMB)(Participants) | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 1 | 2 |
| Not Hispanic or Latino | 20 | 38 | 58 |
| Unknown or Not Reported | 1 | 4 | 5 |
| Race(Participants) | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W | Total |
|---|---|---|---|
| White | 17 | 31 | 48 |
| Black or African American | 0 | 2 | 2 |
| Asian | 4 | 6 | 10 |
| American Indian or Alaska Native | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Not Reported | 0 | 2 | 2 |
| Other | 1 | 2 | 3 |
| Calculated Low-density Lipoprotein Cholesterol (LDL-C)(Milligrams per Deciliter (mg/dL)) | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W | Total |
|---|---|---|---|
| Mean | 246.5 ± 153.71 | 259.5 ± 172.40 | 255.1 ± 165.21 |
| Apolipoprotein B (Apo B)(mg/dL) | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W | Total |
|---|---|---|---|
| Mean | 175.9 ± 98.76 | 169.1 ± 82.75 | 171.4 ± 87.78 |
| Non-high-density Lipoprotein Cholesterol (non-HDL-C)(mg/dL) | Placebo IV Q4W | Evinacumab 15 mg/kg IV Q4W | Total |
|---|---|---|---|
| Mean | 269.9 ± 157.81 | 281.9 ± 172.61 | 277.8 ± 166.60 |
4 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jan 2018 to primary completion in Jun 2019, 16 months.
Results were first posted in May 2021, 1 year 11 months after primary completion. A results delay was requested in May 2020.
Of 20 phase 3 interventional studies under Homozygous Familial Hypercholesterolemia, half reached primary completion within 22 months of starting (the median), counting those still running; a quarter within 16 and three quarters within 34. This study's 16 months is below that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Apr 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Evinacumab
Homozygous Familial Hypercholesterolemia →