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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03933293
Recruitment Status : Not yet recruiting
First Posted : May 1, 2019
Last Update Posted : May 1, 2019
Sponsor:
Information provided by (Responsible Party):
Akeso

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Homozygous Familial Hypercholesterolemia Drug: AK102 Drug: Statins Drug: Ezetimibe Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 59 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2 Study to Evaluate the Safety and Efficacy of PCSK9 Inhibitor AK102 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
Estimated Study Start Date : April 2019
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: AK102
450mg AK102, Q4W, subcutaneous injection
Drug: AK102
450mg, Q4W, subcutaneous injection

Drug: Statins
Lipid background therapy

Drug: Ezetimibe
Lipid background therapy

Placebo Comparator: placebo
Placebo, Q4W, subcutaneous injection
Drug: Statins
Lipid background therapy

Drug: Ezetimibe
Lipid background therapy




Primary Outcome Measures :
  1. Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12 [ Time Frame: Week 12 ]
  2. Incidence of treatment-emergent adverse events as assessed by CTCAE V5.0(only for part 1) [ Time Frame: From baseline through 12 weeks ]

Secondary Outcome Measures :
  1. Percent Change From Baseline in High-density lipoprotein (HDL) cholesterol [ Time Frame: From baseline through 12 weeks ]
  2. Percent Change From Baseline in non High-density lipoprotein (non-HDL) cholesterol [ Time Frame: From baseline through 12 weeks ]
  3. Percent Change From Baseline in Serum Triglyceride (TG) [ Time Frame: From baseline through 12 weeks ]
  4. Percent Change From Baseline in Apolipoprotein B (Apo B) [ Time Frame: From baseline through 12 weeks ]
  5. Percent Change From Baseline in Apolipoprotein A-I (Apo A-I) [ Time Frame: From baseline through 12 weeks ]
  6. Percent Change From Baseline in Total Cholesterol(TC) [ Time Frame: From baseline through 12 weeks ]
  7. Change From Baseline in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) [ Time Frame: From baseline through 12 weeks ]
  8. Concentrations of AK102 in Serum [ Time Frame: Part 1: Day 1,Day 2, Day 4, Day 8, Day 15, Day 22, D29, D57. Part 2: Day 1, Day 29, Day 57 ]
  9. Number of subjects who develop detectable anti-drug antibodies (ADAs) [ Time Frame: From baseline through 12 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03933293


Contacts
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Contact: Xiaoping Jin, PhD +86 (0760) 8987 3999 clinicaltrials@akesobio.com

Locations
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China, Beijing
Peking Union Medical College Hospital Not yet recruiting
Beijing, Beijing, China, 100000
Contact: Peipei Chen, MD         
Principal Investigator: shuyang Zhang, MD         
Sponsors and Collaborators
Akeso
Investigators
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Principal Investigator: Shuyang Zhang, MD Peking Union Medical College Hospital

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Responsible Party: Akeso
ClinicalTrials.gov Identifier: NCT03933293     History of Changes
Other Study ID Numbers: AK102-202
First Posted: May 1, 2019    Key Record Dates
Last Update Posted: May 1, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Akeso:
HoFH
LDL-C
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Ezetimibe
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents