ALN-APOC3 in Adult Participants With Dyslipidemia

NCT06784349 | Completed Phase 1 FDA Drug

• 1 other identifier: ALN-APOC3-hLP-2447
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Dyslipidemia

Keywords

Lipids; Triglycerides; Cholesterol

Outcome Measures

Primary Outcomes (3)

• Incidence of Treatment Emergent Adverse Events (TEAEs)

  Through Week 44

• Severity of TEAEs

  Through Week 44

• Percent changes in fasting concentrations of High-Density Lipoprotein Cholesterol (HDL-C)

  Part B Only

  Baseline to week 24

Secondary Outcomes (25)

• Concentration of combined ALN-APOC3 and metabolites in plasma

  Through Week 44

• Urinary excretion of combined ALN-APOC3 and metabolites

  Through 24 Hours, After Treatment

• Percent change in fasting concentrations of APOC3

  Baseline to Week 12

• Absolute change in fasting concentrations of APOC3

  Baseline to Week 12

• Percent change in fasting concentrations of APOC3

  Baseline to Week 24

Study Arms (5)

ALN-APOC3 Ascending Cohorts

EXPERIMENTAL

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

Drug: ALN-APOC3; Drug: Matching Placebo

Placebo

PLACEBO COMPARATOR

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

Drug: Matching Placebo

ALN-APOC3 low-dose

EXPERIMENTAL

Part B: Parallel Arm Randomized as described in the protocol

Drug: ALN-APOC3

ALN-APOC3 medium-dose

EXPERIMENTAL

Part B: Parallel Arm Randomized as described in the protocol

Drug: ALN-APOC3

ALN-APOC3 high-dose

EXPERIMENTAL

Part B: Parallel Arm Randomized as described in the protocol

Drug: ALN-APOC3

Interventions

ALN-APOC3 DRUG

Administered as per the protocol

ALN-APOC3 Ascending Cohorts; ALN-APOC3 high-dose; ALN-APOC3 low-dose; ALN-APOC3 medium-dose

Matching Placebo DRUG

Administered as per the protocol

ALN-APOC3 Ascending Cohorts; Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Judged by the investigator to be in good health based on medical history, physical examination, vital signs, Electrocardiograms (ECG's) and laboratory safety testing, as defined in the protocol
• Participants can enter the study under one of the following options, as defined in the protocol:
• Currently not taking statin and have not been on statin therapy for the 3 months prior to screening, or
• On a stable dose of statin, taken continuously for 3 months
• Fasting triglycerides concentrations ≥100 and \<500 mg/dL (1.13 to 5.65 mmol/L) during screening visit, as defined in the protocol
• Fasting LDL-C ≥70 (1.81 mmol/L) if on stable statin therapy OR if not on statin treatment, as defined in the protocol

You may not qualify if:

• 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
• Was hospitalized (ie, \>24 hours) for any reason within 30 days of the screening visit
• Is positive for Human Immunodeficiency Virus (HIV) or Hepatitis B surface Antigen (HBsAg) at the screening visit, as defined in the protocol
• Is positive for hepatitis C antibody and positive for qualitative Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) test at the screening visit
• 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

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (1)

Arensia Exploratory Medicine at the Republican Clinical Hospital

Chisinau, 2025, Moldova

Location

MeSH Terms

Conditions

Dyslipidemias

Condition Hierarchy (Ancestors)

Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Part A: Sequential Part B: Parallel

Study Record Dates

• First Submitted: December 3, 2024
• First Posted: January 20, 2025
• Study Start: January 27, 2025
• Primary Completion: February 25, 2026
• Study Completion: February 25, 2026
• Last Updated: March 16, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: When Regeneron has: * received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development * made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry) * the legal authority to share the data, and * ensured the ability to protect participant privacy
• Access Criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf

Locations

MO (1)