Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03510715 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia | PHASE3 | COMPLETED | 18 | — | — | Aug 31, 2018 | Feb 17, 2020 | Dec 29, 2020 | 10 | Brazil, Canada +8 |
| NCT02476006 | Safety, Tolerability, and Effect of Alirocumab in High Cardiovascular Risk Patients With Severe Hypercholesterolemia Not Adequately Controlled With Conventional Lipid-modifying Therapies (ODYSSEY APPRISE) | PHASE3 | COMPLETED | 998 | — | — | Jun 23, 2015 | Apr 12, 2019 | Mar 28, 2022 | 153 | Austria, Belgium +14 |
| NCT02979015 | A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous (SC) Administered Alirocumab in Healthy Chinese Subjects | PHASE1 | COMPLETED | 35 | — | — | Nov 29, 2016 | Nov 27, 2017 | Dec 5, 2017 | 1 | China |
| NCT01785329 | Pharmacokinetic of Alirocumab SAR236553 (REGN727) Administered Subcutaneously at 3 Different Injection Sites in Healthy Subjects | PHASE1 | COMPLETED | 60 | — | — | Feb 1, 2013 | Jul 1, 2013 | Aug 19, 2013 | 1 | United Kingdom |
| NCT01670734 | Pharmacokinetic and Tolerability of Alirocumab SAR236553 (REGN727) in Patients With Hepatic Impairment and in Healthy Subjects | PHASE1 | COMPLETED | 25 | — | — | Sep 1, 2012 | May 1, 2013 | Jun 28, 2013 | 2 | France, Moldova |
| NCT01443650 | Injection Site Tolerability, Safety, Pharmacokinetics, Pharmacodynamics in Different Single-Dose Treatments of Alirocumab SAR236553 (REGN727) in Healthy Subjects | PHASE1 | COMPLETED | 36 | — | — | Jul 1, 2011 | Nov 1, 2011 | Jun 28, 2013 | 1 | United States |
| NCT01448304 | Study of the Tolerability, Safety, Pharmacokinetics and Pharmacodynamics of Alirocumab SAR236553 (REGN727) | PHASE1 | COMPLETED | 24 | — | — | Jun 1, 2011 | Sep 1, 2011 | Jun 28, 2013 | 1 | United States |
| NCT01448239 | Injection Site Tolerability, Safety, Pharmacokinetics and Pharmacodynamics Study After a Single Dose Subcutaneous Treatment of Alirocumab SAR236553 (REGN727) | PHASE1 | COMPLETED | 24 | — | — | Feb 1, 2011 | May 1, 2011 | Jun 28, 2013 | 1 | United States |
Adjusted least square (LS) means and standard errors were obtained from the mixed model analysis with repeated measures (MMRM) to account for missing data using all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis).
Adverse Event (AE) was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. Treatment-emergent AEs (TEAEs) were defined as AEs that that developed or worsened or became serious during the TEAE period (time from the first injection of study drug up to the day of the last injection of study drug + 14 days). A Serious Adverse Event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.
| Arm | Type | Description |
|---|---|---|
| Alirocumab | EXPERIMENTAL | Participants with BW less than (\<) 50 kilograms (kg) received subcutaneous (SC) injection of alirocumab 75 mg Q2W for 48 weeks. Alirocumab dose was up-titrated to 150 mg Q2W from Week 12 in case of increase in BW with BW greater than or equal to \[\>=\] 50 kg. Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks. |
| Placebo | PLACEBO_COMPARATOR | Subcutaneous injection of a single dose of matching placebo |
| alirocumab SAR236553 (REGN727) - Dose A | EXPERIMENTAL | alirocumab SAR236553 (REGN727) - Dose A - Injection in healthy subjects through subcutaneous administration in the abdomen. alirocumab SAR236553 (REGN727) is a fully human monoclonal antibody that binds PCSK9 (proprotein convertase subtilisin/kexin type 9) |
| alirocumab SAR236553 (REGN727) - Dose B | EXPERIMENTAL | alirocumab SAR236553 (REGN727) - Dose B - Injection in healthy subjects through subcutaneous administration in the upper arm. |
| alirocumab SAR236553 (REGN727) - Dose C | EXPERIMENTAL | alirocumab SAR236553 (REGN727) - Dose C - Injection in healthy subjects through subcutaneous administration in the thigh. |
| alirocumab SAR236553 (REGN727) - mild hepatic function | EXPERIMENTAL | Injection through subcutaneous (SC) administration in patients with mild hepatic function |
| alirocumab SAR236553 (REGN727) - moderate hepatic function | EXPERIMENTAL | Injection through subcutaneous (SC) administration in patients with moderate hepatic function |
| alirocumab SAR236553 (REGN727) - normal hepatic function | EXPERIMENTAL | Injection through subcutaneous (SC) administration in patients with normal hepatic function |
| alirocumab SAR236553 (REGN727) (Formulation A x 1) | EXPERIMENTAL | A single subcutaneous injection of Formulation A |
| alirocumab SAR236553 (REGN727) (Formulation B x 1) | EXPERIMENTAL | A single subcutaneous injection of Formulation B |
| alirocumab SAR236553 (REGN727) (Formulation A x 2) | EXPERIMENTAL | 2 single subcutaneous injections of Formulation A |
| Name | Type | Description |
|---|---|---|
| Alirocumab SAR236553 (REGN727) | DRUG | Pharmaceutical form: solution for injection in pre-filled syringe, Route of administration: subcutaneous (SC) |
| Atorvastatin | DRUG | Pharmaceutical form: tablet, Route of administration: oral |
| Simvastatin | DRUG | Pharmaceutical form: tablet, Route of administration: oral |
| Fluvastatin | DRUG | Pharmaceutical form: capsule, Route of administration: oral |
| Pravastatin | DRUG | Pharmaceutical form: tablet, Route of administration: oral |
| Lovastatin | DRUG | Pharmaceutical form: tablet, Route of administration: oral |
| Rosuvastatin | DRUG | Pharmaceutical form: tablet, Route of administration: oral |
| Ezetimibe | DRUG | Pharmaceutical form: tablet, Route of administration: oral |
| Cholestyramine | DRUG | Pharmaceutical form: oral suspension, Route of administration: oral |
| Nicotinic acid | DRUG | Pharmaceutical form: tablet, Route of administration: oral |
| Fenofibrate | DRUG | Pharmaceutical form: tablet, Route of administration: oral |
| Omega-3 fatty acids | DRUG | Pharmaceutical form: capsule, Route of administration: oral |
| placebo (for injection training only) | DRUG | Pharmaceutical form:solution Route of administration: subcutaneous |
| placebo | DRUG | Pharmaceutical form: Solution for injection Route of administration: Subcutaneous |
Inclusion criteria : * Participants genetically diagnosed with hoFH. * Participants treated with optimal dose of statin +/- other lipid modifying therapies (LMTs), or non-statin LMTs if statin-intolerant at stable dose(s) for at least 4 weeks. * A signed informed consent indicating parental permiss...