Usability Study of the Commercial Auto-injector Device and the New Auto-injector Device (SYDNEY) in Patients With High or Very High CV Risk With Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy

Sanofi

Status and phase: Completed; Phase 3

Conditions: Hypercholesterolaemia

Treatments: Drug: Atorvastatin; Device: Current auto-injector device (AI); Drug: Alirocumab SAR236553; Drug: Rosuvastatin; Device: New auto-injector device (SYDNEY)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03415178

MSC14864

U1111-1186-3466 (Other Identifier)

Details and patient eligibility

About

Primary Objective:

To collect real-use (usability) data assessing the robustness and user interaction of the new alirocumab auto-injector device (which is referred to as SYDNEY), in unsupervised settings.

Secondary Objective:

Device-related:

• To collect real-use (usability) data assessing the robustness and user interaction of SYDNEY and the current alirocumab auto-injector device (which is referred to as AI) in supervised settings.

Pharmacokinetics:

• To compare alirocumab pharmacokinetics (PK) administered using SYDNEY and AI.
• To evaluate alirocumab PK administered using SYDNEY.

Anti-drug antibodies:

• To evaluate the development of anti-drug (alirocumab) antibodies (ADA).

Efficacy/pharmacodynamics:

• To compare the percent and absolute change in low-density lipoprotein cholesterol (LDL-C) using SYDNEY and AI.
• To evaluate the percent and absolute change in LDL-C using SYDNEY.

Safety:

• To evaluate the safety and tolerability of alirocumab using both SYDNEY and AI.

Full description

Total study duration per participant was expected to be up to 18 weeks, with up to 2 weeks of screening period and 16 weeks of study treatment period.

Enrollment: 69 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion criteria :

• Participants were in either category A or B (below), and were not adequately controlled with a stable daily dose of atorvastatin (20 mg or 40 mg), or rosuvastatin (10 mg or 20 mg) for at least 4 weeks prior to the screening visit (Week -2), with or without other LMT:

  • A. Participants with heterozygous familial hypercholesterolemia (heFH) (diagnosis based on either genotyping or clinical criteria) OR
  • B. Non-FH Participants at high or very high cardiovascular (CV) risk. High and very high cardiovascular risk participants included participants with coronary heart disease (CHD), non-CHD cardiovascular disease (CVD), and other risk factors.

• Participant willing and able to self-inject for the duration of the study.

Exclusion criteria:

• LDL-C <70 milligrams per deciliter (mg/dL) (<1.81 millimoles per litre [mmol/L]) at the screening visit.
• Currently taking a daily dose of statin that was not atorvastatin 20 mg or 40 mg, or rosuvastatin 10 mg or 20 mg.
• Not on a stable dose of LMT (including statin) for at least 4 weeks, prior to the screening visit and from screening to randomization.
• Having previously used any device for the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor administration, or having participated in any clinical trial for a PCSK9 inhibitor.
• Fasting serum Triglyceride (TG) >400 mg/dL (>4.52 mmol/L) at the screening visit.

The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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69 participants in 2 patient groups

Auto-Injector Device (AI)

Experimental group

Description:

Alirocumab 300 milligram (mg) subcutaneous (SC) injection on Week 0 (Day 1), self-administered using AI device, on-site under supervision in the parallel arm treatment period of 4 weeks. From Week 4, participants switched to other arm of SYDNEY device to receive Alirocumab 300 mg, self- administered (unsupervised) using new auto-injector device (SYDNEY) every 4 weeks (Q4W) from Week 4 until Week 16 in the single arm treatment period added to lipid modifying therapy (LMT).

Treatment:

Drug: Rosuvastatin

Drug: Atorvastatin

Drug: Alirocumab SAR236553

Device: Current auto-injector device (AI)

New Auto-injector Device (SYDNEY)

Experimental group

Description:

Alirocumab 300 mg SC injection on Week 0 (Day 1), self-administered using new auto-injector device (SYDNEY), on-site under supervision in the parallel arm treatment period of 4 weeks. From Week 4, same treatment (Alirocumab 300 mg) with the same device (SYDNEY) was self-administered, (unsupervised) Q4W until Week 16 in the single arm treatment period added to LMT. Duration of single arm treatment period was 12 weeks, i.e. from Week 4 to 16.

Treatment:

Drug: Rosuvastatin

Drug: Atorvastatin

Device: New auto-injector device (SYDNEY)

Drug: Alirocumab SAR236553