A Study of PCSK9 Inhibitor AK102 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)

March 1, 2023 updated by: Akeso

A Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Safety and Efficacy of AK102 in Patients With Heterozygous Familial Hypercholesterolemia

This is a double-blind, randomized, placebo-controlled, multicenter study to evaluate the safety and efficacy of AK102 in patients with heterozygous familial hypercholesterolemia (HeFH).The primary objective of this study is to evaluate the efficacy of AK102 in patients with HeFH.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
109

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
      • Beijing, China, 100000
        • Peking Union Medical College Hospital
    • Beijing
      • Beijing, Beijing, China, 100029
        • Beijing Anzhen Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Subjects with heterozygous familial hypercholesterolemia diagnosed by genetic confirmation or clinical diagnosis criteria.
  • Stable on pre-existing, lipid-lowering therapies (statins with or without ezetimibe) for at least 4 weeks with no planned medication or dose change for the duration of study participation.
  • Fasting Low-Density Lipoprotein Cholesterol (LDL-C) ≥ 70 mg/dL in patients with history of Atherosclerotic Cardiovascular Disease (ASCVD) or Fasting Low-Density Lipoprotein Cholesterol (LDL-C) ≥ 100 mg/dL in patients without history of Atherosclerotic Cardiovascular Disease (ASCVD).
  • Fasting triglycerides ≤ 400 mg/dL.
  • Body weight ≥ 40kg.

Key Exclusion Criteria:

  • Subjects with homozygous FH (clinically or by genotyping).
  • Receipt of LDL apheresis within 12 months prior to the first dose of Investigational product.
  • Receipt of Lomitapide or Mipomersen within 5 months prior to the first dose of Investigational product.
  • Prior use of PCSK9 inhibitors.
  • Creatine kinase (CK) >3 times of the upper limit of normal (ULN).
  • Aspartate Aminotransferase (AST) ≥ 2 x ULN.
  • Estimated Glomerular Filtration Rate (eGFR)≤ 30 mL/min/1.73m^2.
  • Thyroid-Stimulating Hormone (TSH)> 1.5 x ULN or <1 x LLN.
  • Type 1 diabetes, or type 2 diabetes that is or poorly controlled（HbA1c> 8.5%).
  • Subjects with untreated or active chronic hepatitis B or active hepatitis C virus infections.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: AK102 450 mg
Participants received AK102 450 mg subcutaneous injection once every 4 weeks (Q4W) for 12 weeks
Drug: AK102
Administered by subcutaneous injection
Drug: Statins and/or Ezetimibe
Lipid-lowering therapies
Experimental: AK102 300 mg
Participants received AK102 300 mg subcutaneous injection once every 4 weeks (Q4W) for 12 weeks
Drug: AK102
Administered by subcutaneous injection
Drug: Statins and/or Ezetimibe
Lipid-lowering therapies
Experimental: AK102 150 mg
Participants received AK102 150 mg subcutaneous injection once every 2 weeks (Q2W) for 12 weeks
Drug: AK102
Administered by subcutaneous injection
Drug: Statins and/or Ezetimibe
Lipid-lowering therapies
Placebo Comparator: Placebo Q4W
Participants received placebo subcutaneous injection once every 4 weeks (Q4W) for 12 weeks
Drug: Placebo
Administered by subcutaneous injection
Drug: Statins and/or Ezetimibe
Lipid-lowering therapies
Placebo Comparator: Placebo Q2W
Participants received placebo subcutaneous injection once every 2 weeks (Q2W) for 12 weeks
Drug: Placebo
Administered by subcutaneous injection
Drug: Statins and/or Ezetimibe
Lipid-lowering therapies

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Percent change from baseline in low-density lipoprotein cholesterol (LDL-C) at Week 12
Time Frame: At baseline and week 12
At baseline and week 12

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percent change from baseline in low-density lipoprotein cholesterol (LDL-C)
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Percent change from baseline in high-density lipoprotein cholesterol (HDL-C)
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Percent change from baseline in non High-density lipoprotein (non-HDL) cholesterol
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Percent change from baseline in serum Triglyceride (TG) cholesterol
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Percent change from baseline in Apolipoprotein B (Apo B)
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Percent change from baseline in Apolipoprotein A-I (ApoA-I)
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Percent change from baseline in Lipoprotein(a) [Lp-(a)]
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Percent change from baseline in Total Cholesterol(TC)
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Incidence of treatment-emergent adverse events
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Serum concentrations of AK102
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks
Number of subjects who develop detectable anti-drug antibodies (ADAs)
Time Frame: From baseline through 12 weeks
The immunogenicity of AK102 will be assessed by summarizing the number of subjects who develop detectable anti-drug antibodies.
From baseline through 12 weeks
Change from baseline in proprotein convertase subtilisin/kexin type 9 (PCSK9)
Time Frame: From baseline through 12 weeks
From baseline through 12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Akeso

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
AD Pharmaceuticals Co., Ltd.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Shuyang Zhang, MD, Peking Union Medical College Hospital
  • Principal Investigator: Yujie Zhou, MD, Beijing Anzhen Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 18, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 26, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 26, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 17, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 20, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 22, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 2, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 1, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AK102-201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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