Ignite Creation Date:
2025-12-25 @ 1:24 AM
Ignite Modification Date:
2025-12-31 @ 11:44 PM
Study NCT ID:
NCT03933293
Status:
COMPLETED
Last Update Posted:
2023-03-02
First Post:
2019-04-28
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000090542', 'term': 'Homozygous Familial Hypercholesterolemia'}], 'ancestors': [{'id': 'D006938', 'term': 'Hyperlipoproteinemia Type II'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D006951', 'term': 'Hyperlipoproteinemias'}, {'id': 'D006949', 'term': 'Hyperlipidemias'}, {'id': 'D050171', 'term': 'Dyslipidemias'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D019161', 'term': 'Hydroxymethylglutaryl-CoA Reductase Inhibitors'}, {'id': 'D000069438', 'term': 'Ezetimibe'}], 'ancestors': [{'id': 'D000924', 'term': 'Anticholesteremic Agents'}, {'id': 'D000960', 'term': 'Hypolipidemic Agents'}, {'id': 'D000963', 'term': 'Antimetabolites'}, {'id': 'D045504', 'term': 'Molecular Mechanisms of Pharmacological Action'}, {'id': 'D020228', 'term': 'Pharmacologic Actions'}, {'id': 'D020164', 'term': 'Chemical Actions and Uses'}, {'id': 'D004791', 'term': 'Enzyme Inhibitors'}, {'id': 'D057847', 'term': 'Lipid Regulating Agents'}, {'id': 'D045506', 'term': 'Therapeutic Uses'}, {'id': 'D001384', 'term': 'Azetidines'}, {'id': 'D001385', 'term': 'Azetines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 10}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-05-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-03', 'completionDateStruct': {'date': '2021-03-15', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-03-01', 'studyFirstSubmitDate': '2019-04-28', 'studyFirstSubmitQcDate': '2019-04-30', 'lastUpdatePostDateStruct': {'date': '2023-03-02', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-05-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-03-15', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12', 'timeFrame': 'Week 12'}, {'measure': 'Incidence of treatment-emergent adverse events as assessed by CTCAE V5.0（only for part 1）', 'timeFrame': 'From baseline through 12 weeks'}], 'secondaryOutcomes': [{'measure': 'Percent Change From Baseline in High-density lipoprotein (HDL) cholesterol', 'timeFrame': 'From baseline through 12 weeks'}, {'measure': 'Percent Change From Baseline in non High-density lipoprotein (non-HDL) cholesterol', 'timeFrame': 'From baseline through 12 weeks'}, {'measure': 'Percent Change From Baseline in Serum Triglyceride (TG)', 'timeFrame': 'From baseline through 12 weeks'}, {'measure': 'Percent Change From Baseline in Apolipoprotein B (Apo B)', 'timeFrame': 'From baseline through 12 weeks'}, {'measure': 'Percent Change From Baseline in Apolipoprotein A-I (Apo A-I)', 'timeFrame': 'From baseline through 12 weeks'}, {'measure': 'Percent Change From Baseline in Total Cholesterol(TC)', 'timeFrame': 'From baseline through 12 weeks'}, {'measure': 'Change From Baseline in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9)', 'timeFrame': 'From baseline through 12 weeks'}, {'measure': 'Concentrations of AK102 in Serum', 'timeFrame': 'Part 1: Day 1,Day 2, Day 4, Day 8, Day 15, Day 22, D29, D57. Part 2: Day 1, Day 29, Day 57'}, {'measure': 'Number of subjects who develop detectable anti-drug antibodies (ADAs)', 'timeFrame': 'From baseline through 12 weeks'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['HoFH', 'LDL-C'], 'conditions': ['Homozygous Familial Hypercholesterolemia']}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* 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\n* 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\n* Fasting central lab LDL-C concentration \\>130 mg/dL (3.4 mmol/L) and triglyceride concentration \\<400 mg/dL (4.5 mmol/L).\n* Body weight of 40 kilograms (kg) or greater at screening\n\nExclusion Criteria:\n\n* Received LDL plasma replacement therapy within 8 weeks before Investigational product administration\n* Received Lomitapide or Mipomersen within 5 months before Investigational product administration\n* Received prior treatment with PCSK9 inhibitors or AK102.\n* Unexplained creatine kinase (CK) ≥ 5 times the upper limit of normal (ULN)\n* 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.\n* Known allergic reactions to any ingredients of AK102\n* 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.'}, 'identificationModule': {'nctId': 'NCT03933293', 'briefTitle': 'A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH', 'organization': {'class': 'INDUSTRY', 'fullName': 'Akeso'}, 'officialTitle': 'A Phase 2 Study to Evaluate the Safety and Efficacy of PCSK9 Inhibitor AK102 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)', 'orgStudyIdInfo': {'id': 'AK102-202'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AK102', 'description': '450mg AK102, Q4W, subcutaneous injection', 'interventionNames': ['Drug: AK102', 'Drug: Statins', 'Drug: Ezetimibe']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'placebo', 'description': 'Placebo, Q4W, subcutaneous injection', 'interventionNames': ['Drug: Statins', 'Drug: Ezetimibe']}], 'interventions': [{'name': 'AK102', 'type': 'DRUG', 'description': '450mg, Q4W, subcutaneous injection', 'armGroupLabels': ['AK102']}, {'name': 'Statins', 'type': 'DRUG', 'description': 'Lipid-lowering therapies', 'armGroupLabels': ['AK102', 'placebo']}, {'name': 'Ezetimibe', 'type': 'DRUG', 'description': 'Lipid-lowering therapies', 'armGroupLabels': ['AK102', 'placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '100000', 'city': 'Beijing', 'state': 'Beijing Municipality', 'country': 'China', 'facility': 'Peking Union Medical College Hospital', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}, {'zip': '100029', 'city': 'Beijing', 'state': 'Beijing Municipality', 'country': 'China', 'facility': 'Beijing Anzhen Hospital', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}], 'overallOfficials': [{'name': 'Shuyang Zhang, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Peking Union Medical College Hospital'}, {'name': 'Lvya Wang', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Beijing Anzhen Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Akeso', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'AD Pharmaceuticals Co., Ltd.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}