Phase 2
N=9
Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Homozygous Familial Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT02265952 ↗Enrolled (actual)
9
Serious AEs
17.7%
Results posted
Dec 2019
Primary outcome: Primary: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) to Week 4 in the Main Study Period — -49.17 Percent Change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- REGN1500 250 mg SC/15 mg/kg IV/450 mg SC (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Regeneron Pharmaceuticals
- Primary completion
- Nov 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) to Week 4 in the Main Study Period |
-49.17 | — |
| SECONDARY Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) to Week 4 in the Main Study Period |
-157.34 | — |
| SECONDARY Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Week 2 to Week 4 in the Main Study Period |
-63.63 | — |
| SECONDARY Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Week 2 to Week 4 in the Main Study Period |
-30.05 | — |
| SECONDARY Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) Over Time in the Main Study Period |
-39.60; -67.59; -93.71; -129.97; -157.34; -179.18 | — |
| SECONDARY Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) Over Time in the Main Study Period |
-12.76; -24.00; -30.13; -41.38; -49.17; -46.88 | — |
| SECONDARY Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period |
261.34; -58.73; -88.16; -116.20; -87.80; -144.99 | — |
| SECONDARY Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period |
261.34; -22.49; -36.74; -44.35; -37.37; -50.74 | — |
| SECONDARY Absolute Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) From Baseline (Week 0) up to Week 26 in the Main Study Period |
-53.0; -81.0; -96.3; -104.6; -100.7; -96.8 | — |
| SECONDARY Percent Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) From Baseline (Week 0) up to Week 26 in the Main Study Period |
-24.38; -38.63; -45.89; -42.28; -43.12; -42.66 | — |
| SECONDARY Absolute Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period |
-48.1; -65.9; -40.9; -79.5; -78.6; -57.8 | — |
| SECONDARY Percent Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period |
-30.59; -41.27; -29.02; -50.90; -48.59; -36.68 | — |
| SECONDARY Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% From Baseline (Week 0) in the Main Study Period |
100 | — |
| SECONDARY Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% From Baseline (Week 26) in the Open Label Extension (OLE) Period |
100 | — |
| SECONDARY Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% From Baseline (Week 0) in the Main Study Period |
77.8 | — |
| SECONDARY Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% From Baseline (Week 26) in the Open Label Extension (OLE) Period |
87.5 | — |
| SECONDARY Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 0) Over Time in the Main Study Period |
-5.81; -9.92; -11.99; -16.02; -15.07; -11.66 | — |
| SECONDARY Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 0) Over Time in the Main Study Period |
-14.37; -23.34; -28.75; -36.96; -35.24; -29.74 | — |
| SECONDARY Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period |
-21.47; -27.60; -26.51; -27.57; -32.81; -32.72 | — |
| SECONDARY Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period |
-8.78; -12.08; -11.44; -13.29; -13.94; -14.11 | — |
| SECONDARY Absolute Change in Apolipoprotein CIII (Apo CIII) From Baseline (Week 0) Over Time in the Main Study Period |
-3.729; -4.296; -4.301; -6.000; -8.380; -3.772 | — |
| SECONDARY Percent Change in Apolipoprotein CIII (Apo CIII) From Baseline (Week 0) Over Time in the Main Study Period |
-46.59; -55.43; -56.57; -75.44; -91.48; -49.07 | — |
Summary
This is an open-label, single-arm study to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by REGN1500 in patients with homozygous familial hypercholesterolemia (HoFH).
Eligibility Criteria
Inclusion Criteria
- Men and women ≥18 years of age at the time of the screening visit
- Diagnosis of homozygous familial hypercholesterolemia (HoFH)
- Willing to consistently maintain usual diet for the duration of the study
Exclusion Criteria
- Background medical lipid modifying therapy that has not been stable for at least 4 weeks (6 weeks for fibrates) prior to the screening visit
- Having undergone lipid apheresis within 4 weeks prior to the screening visit
- Use of another investigational drug or therapy within 30 days or at least 5 half-lives (whichever is longer) prior to the screening visit
- Previous participation in any clinical trial of REGN1500
Note: The information listed above is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial therefore not all inclusion/ exclusion criteria are listed.
Data sourced from ClinicalTrials.gov (NCT02265952). 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.