ALN-APOC3 in Adult Participants With Dyslipidemia

Regeneron Pharmaceuticals

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Dyslipidemia

Treatments: Drug: Matching Placebo; Drug: ALN-APOC3

Study type: Interventional

Funder types: Industry

Identifiers

NCT06784349

ALN-APOC3-hLP-2447

Details and patient eligibility

About

This study is researching an experimental drug called ALN-APOC3 (called "study drug"). The study is focused on participants who have dyslipidemia (abnormal amounts of fats in the blood, including triglycerides and cholesterol), but who are otherwise healthy.

The aim of the study is to see how safe and effective the study drug is.

The study is looking at several other research questions, including:

• What side effects may happen from taking the study drug
• How much study drug is in the blood at different times
• How the study drug changes lipid levels in the blood

Full description

The study will be conducted in 2 parts, Part A and Part B. Participants in Part A will be excluded from participating in Part B.

Enrollment: 136 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. Judged by the investigator to be in in good health based on medical history, physical examination, vital signs, electrocardiograms (ECG's) and laboratory safety testing, as defined in the protocol

2. Participants can enter the study under one of the following options, as defined in the protocol:

   1. Currently not taking statin and have not been on statin therapy for the 3 months prior to screening, or
   2. On a stable dose of statin, taken continuously for 3 months

3. Fasting triglycerides concentrations ≥100 and <500 mg/dL (1.13 to 5.65 mmol/L) during screening visit, as defined in the protocol

4. Fasting LDL-C ≥70 (1.81 mmol/L) if on stable statin therapy OR if not on statin treatment, as defined in the protocol

Key Exclusion Criteria:

1. History of clinically significant cardiovascular (other than dyslipidemia), respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, psychiatric, or neurological disease, or any other concern, as assessed by the investigator that may confound the results of the study or poses an additional risk to the participant by study participation
2. Was hospitalized (ie, >24 hours) for any reason within 30 days of the screening visit
3. Is positive for human immunodeficiency virus (HIV) or hepatitis B surface antigen (HBsAg) at the screening visit, as defined in the protocol
4. Is positive for hepatitis C antibody and positive for qualitative hepatitis c virus (HCV) ribonucleic acid (RNA) test at the screening visit
5. Any malignancy, except for non-melanoma skin cancer or cervical/anus in-situ, that have been resected with no evidence of metastatic disease for 3 years prior to the screening visit

NOTE: Other protocol defined inclusion/exclusion criteria apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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136 participants in 5 patient groups, including a placebo group

ALN-APOC3 Ascending Cohorts

Experimental group

Description:

Part A: Ascending Cohorts Randomized as described in the protocol May include optional cohorts

Treatment:

Drug: Matching Placebo

Drug: ALN-APOC3

Placebo

Placebo Comparator group

Description:

Part A and Part B: Randomized as described in the protocol

Treatment:

Drug: Matching Placebo

ALN-APOC3 low-dose

Experimental group

Description:

Part B: Parallel Arm Randomized as described in the protocol

Treatment:

Drug: ALN-APOC3

ALN-APOC3 medium-dose

Experimental group

Description:

Part B: Parallel Arm Randomized as described in the protocol

Treatment:

Drug: ALN-APOC3

ALN-APOC3 high-dose

Experimental group

Description:

Part B: Parallel Arm Randomized as described in the protocol

Treatment:

Drug: ALN-APOC3