Phase 3
N=370
A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT01968980 ↗Enrolled (actual)
370
Serious AEs
12.4%
Results posted
May 2017
Primary outcome: Primary: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 — -0.3; -54.2 percent change — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Bococizumab (PF-04950615;RN316) (Drug); Placebo (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Apr 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 |
-0.3; -54.2 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Total Cholesterol (TC) at Week 12 |
-0.0; -37.0 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non HDL-C) at Week 12 |
0.4; -49.9 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein B (ApoB) at Week 12 |
0.4; -47.5 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Lipoprotein (a) at Week 12 |
2.2; -26.4 | <0.001 sig |
| SECONDARY Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12 |
-0.3; 6.3 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 24 and 52 |
2.7; -50.1; 3.5; -45.3 | — |
| SECONDARY Percent Change From Baseline in Total Cholesterol (TC) at Week 24 and 52 |
1.6; -34.4; 1.2; -31.0 | — |
| SECONDARY Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non HDL-C) at Week 24 and 52 |
2.4; -46.7; 1.2; -41.8 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein B (ApoB) at Week 24 and 52 |
2.6; -44.8; 1.7; -39.3 | — |
| SECONDARY Percent Change From Baseline in Lipoprotein (a) at Week 24 and 52 |
14.6; -21.2; 1.3; -15.1 | — |
| SECONDARY Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 24 and 52 |
0.9; 6.6; 2.2; 4.7 | — |
| SECONDARY Percent Change From Baseline in Triglycerides (TG) at Week 12, 24 and 52 |
7.1; -8.7; 7.6; -9.0; 4.9; -3.4 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein A-I (ApoA-I) at Week 12, 24 and 52 |
-0.5; 4.8; -0.6; 3.9; 0.3; 3.0 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein A-II (ApoA-II) at Week 12, 24 and 52 |
-2.6; -1.6; -2.8; -1.8; -3.5; -2.4 | — |
| SECONDARY Percent Change From Baseline in Very Low Density Lipoprotein Cholesterol (VLDL-C) at Week 12, 24 and 52 |
7.1; -8.7; 7.6; -9.0; 4.9; -3.4 | — |
| SECONDARY Absolute Change From Baseline in Low Density Lipoprotein (LDL-C) at Week 12 |
150.0; 144.2; -3.0; -79.1 | — |
| SECONDARY Absolute Change From Baseline in Total Cholesterol (TC) at Week 12 |
227.2; 220.7; -2.8; -83.2 | — |
| SECONDARY Absolute Change From Baseline in Non- High Density Lipoprotein Cholesterol (Non HDL-C) at Week 12 |
178.6; 170.4; -2.3; -86.0 | — |
| SECONDARY Absolute Change From Baseline in Apolipoprotein B (ApoB) at Week 12 |
115.9; 112.8; -0.9; -53.7 | — |
| SECONDARY Absolute Change From Baseline in Lipoprotein (a) at Week 12 |
58.8; 59.7; -1.4; -14.0 | — |
| SECONDARY Absolute Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12 |
48.6; 50.3; -0.6; 2.8 | — |
| SECONDARY Absolute Change From Baseline in Ratio of Total Cholesterol to High Density Lipoprotein Cholesterol (TC/HDL-C Ratio) at Week 12, 24 and 52 |
4.9; 4.6; 0.0; -1.8; 0.0; -1.7 | — |
| SECONDARY Absolute Change From Baseline in Ratio of Apolipoprotein B to Apolipoprotein A-I (ApoB/ApoA-I Ratio) at Week 12, 24 and 52 |
0.8; 0.8; 0.0; -0.4; 0.0; -0.4 | — |
| SECONDARY Percentage of Participants Achieving Low Density Lipoprotein Cholesterol (LDL-C) Level Less Than or Equal to (<=) 100 Milligram Per Deciliter (2.59 Millimoles Per Liter) at Week 12, 24 and 52 |
13.1; 83.1; 13.1; 80.9; 16.6; 70.8 | — |
| SECONDARY Percentage of Participants Achieving Low Density Lipoprotein Cholesterol (LDL-C) Level Less Than or Equal to (<=) 70 Milligram Per Deciliter (1.81 Millimoles Per Liter) at Week 12, 24 and 52 |
1.1; 66.3; 1.1; 60.7; 0.6; 53.2 | — |
| SECONDARY Plasma PF-04950615 Concentrations at Week 12, 24 and 52 |
6.23; 7.00; 4.84 | — |
| SECONDARY Number of Participants With Adverse Events Related to Type 1 or 3 Hypersensitivity Reactions and Injection Site Reactions |
0; 0; 1; 38 | — |
| SECONDARY Percentage of Participants With Positive Anti-Drug Antibodies (ADA) and Neutralizing Antibodies (nAb) |
49.7; 33 | — |
Summary
This is a multicenter, randomized study in subjects with heterozygous familial hypercholesterolemia receiving highly effective statins to assess the safety, efficacy and tolerability of Bococizumab (PF-04950615; RN316) to lower LDL-C.
Eligibility Criteria
Inclusion Criteria
- Treated with a statin.
- Fasting LDL-C > 70 mg/dL and triglyceride <=400 mg/dL.
- High or very high risk of incurring a cardiovascular event.
- Heterozygous familial hypercholesterolemia.
Exclusion Criteria
- Pregnant or breastfeeding females.
- Cardiovascular or cerebrovascular event of procedures during the past 30 days.
- Congestive heart failure NYHA class IV.
- Poorly controlled hypertension.
Data sourced from ClinicalTrials.gov (NCT01968980). 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.