A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-05-13

Study Completion Date

2021-03-15

Brief Summary

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AK102 is being developed for the treatment of HoFH. The study will be conducted in 2 parts, part 1 is open label, single arm study to evaluate the safety, tolerability and efficacy of PCSK9 inhibitor AK102, and part 2 is double blind, randomized, placebo controlled study to evaluate the efficacy and safety of PCSK9 inhibitor AK102. The treatment period will last 12 week.

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Detailed Description

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Conditions

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Homozygous Familial Hypercholesterolemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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AK102

450mg AK102, Q4W, subcutaneous injection

Group Type EXPERIMENTAL

AK102

Intervention Type DRUG

450mg, Q4W, subcutaneous injection

Statins

Intervention Type DRUG

Lipid-lowering therapies

Ezetimibe

Intervention Type DRUG

Lipid-lowering therapies

placebo

Placebo, Q4W, subcutaneous injection

Group Type PLACEBO_COMPARATOR

Statins

Intervention Type DRUG

Lipid-lowering therapies

Ezetimibe

Intervention Type DRUG

Lipid-lowering therapies

Interventions

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AK102

450mg, Q4W, subcutaneous injection

Intervention Type DRUG

Statins

Lipid-lowering therapies

Intervention Type DRUG

Ezetimibe

Lipid-lowering therapies

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Males and females, ≥18 years of age with a diagnosis of homozygous familial hypercholesterolemia by genetic confirmation or a clinical diagnosis based on a history of an untreated low-density lipoprotein cholesterol (LDL-C) concentration \>500 mg/deciliter (dL) \[13 millimoles/liter (mmol/L)\] together with either xanthoma before 10 years of age or evidence of heterozygous familial hypercholesterolemia in both parents
* Stable on pre-existing, lipid-lowering therapies (statins in combination with ezetimibe) for at least 4 weeks with no planned medication or dose change for the duration of study participation
* Fasting central lab LDL-C concentration \>130 mg/dL (3.4 mmol/L) and triglyceride concentration \<400 mg/dL (4.5 mmol/L).
* Body weight of 40 kilograms (kg) or greater at screening

Exclusion Criteria

* Received LDL plasma replacement therapy within 8 weeks before Investigational product administration
* Received Lomitapide or Mipomersen within 5 months before Investigational product administration
* Received prior treatment with PCSK9 inhibitors or AK102.
* Unexplained creatine kinase (CK) ≥ 5 times the upper limit of normal (ULN)
* Subjects with untreated chronic hepatitis B or chronic hepatitis B virus (HBV) DNA exceeding 500 IU/ mL or active hepatitis C virus (HCV) should be excluded. Subjects with non-active HBsAg carriers, treated and stable hepatitis B (HBV DNA \<500 IU/ mL) , and cured hepatitis C can be enrolled. Subjects with positive HCV antibodies are eligible only if the HCV RNA test results are negative.
* Known allergic reactions to any ingredients of AK102
* Any other condition(s) that would compromise the safety of the patient or compromise the quality of the clinical study as judged by the Investigator and/or Medical Monitor.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No