Clinical trial NCT05325203: A Study to Evaluate the Efficacy and Safety of JS002 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH).
Official title: A Randomized, Double-blind, Placebo-controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of Recombinant Humanized Anti-PCSK9 Monoclonal Antibody Injection in Subjects With Heterozygous Familial Hypercholesterolemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Heterozygous Familial Hypercholesterolemia
Lead sponsor: Shanghai Junshi Bioscience Co., Ltd.
Sponsor class: OTHER
First posted: 2022-04-13
Enrollment: 135
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: JS002 is a recombinant human anti-PCSK9 monoclonal antibody.The study is a multicenter, randomized, double-blind, placebo-controlled Phase III clinical study in Chinese patients with heterozygous familial hypercholesterolemia (HeFH). Objective To evaluate the efficacy and safety of JS002 150 mg (Q2W) and 450 mg (Q4W) subcutaneous injection (SC).

Detailed description: A randomized, double-blind, placebo-controlled Phase III clinical study evaluating the efficacy and safety of JS002 in patients with heterozygous familial hypercholesterolemia. 120 subjects are planned to be enrolled. Each subject is required a maximum of 6 weeks of screening, 24 weeks of treatment, and 8 weeks of follow-up. To evaluate the lipid-lowering efficacy of subcutaneous injection of JS002 at 24 weeks compared with placebo in heterozygous familial hypercholesterolemia patients under optimized lipid lowing therapy . Subjects meeting the study inclusion criteria will be randomly assigned in a 2:1:2:1 ratio to JS002 150 mg Q2W or JS002 450 mg Q4W or matched placebo to receive the study drug (JS002) or placebo subcutaneously for 24 weeks.

treatment cohorts: JS002 150mg Cohort:JS002 150mg or placebo treatment(JS002 :Placebo=2:1) Q2W JS002 450mg Cohort:JS002 450mg or placebo treatment(JS002 :Placebo=2:1)Q4W

Eligibility criteria: Inclusion Criteria:

1. Signed informed consent.
2. Males and females ≥ 18 to ≤ 80 years of age
3. DLCN\>8 in HeFH
4. Stable lipid-lowering therapies for at least 4 weeks
5. Patients with ASCVD LDL cholesterol≥1.4mmol/L at screening Patients without ASCVD LDL cholesterol≥2.6mmol/L at screening
6. Triglyceride≤4.5 mmol/L（400 mg/dL）;

Exclusion Criteria:

1. HoFH or meet the diagnostic criteria of HoFH
2. New York Heart Association (NYHA) class III or IV or last known left ventricular ejection fraction \< 30%
3. History of uncontrolled arrhythmia within 90 days
4. Myocardial infarction, unstable angina, percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG) or stroke within 90 days of randomization
5. Planned cardiac surgery or revascularization.
6. Uncontrolled diabetes mellitius (HbA1c\>8.0%）.
7. Uncontrolled hypertension.
8. Other conditions that the researchers considered inappropriate to participate in the study.

Primary outcomes:
measure: Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24
timeFrame: Baseline and week 24
Secondary outcomes:
measure: Absolute Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24
timeFrame: Baseline and Week 24
measure: Percentage changes From Baseline in the Total Cholesterol at week 24
timeFrame: Baseline and Week 24
measure: Absolute changes From Baseline in the Total Cholesterol at week 24
timeFrame: Baseline and Week 24
measure: Percentage changes From Baseline in Non-HDL-C at Week 24
timeFrame: Baseline and Week 24
measure: Absolute changes From Baseline in Non-HDL-C at Week 24
timeFrame: Baseline and Week 24
measure: Percentage changes From Baseline in Apo B at Week 24
timeFrame: Baseline and Week 24
measure: Absolute changes From Baseline in Apo B at Week 24
timeFrame: Baseline and Week 24
measure: Percentage changes From Baseline in Lp(a) at Week 24
timeFrame: Baseline and Week 24
measure: Absolute changes From Baseline in Lp(a) at Week 24
timeFrame: Baseline and Week 24
measure: Percentage changes From Baseline in HDL-C at Week 24
timeFrame: Baseline and Week 24
measure: Absolute changes From Baseline in HDL-C at Week 24
timeFrame: Baseline and Week 24
measure: Percentage changes From Baseline in Apo A1 at Week 24
timeFrame: Baseline and Week 24
measure: Absolute changes From Baseline in Apo A1 at Week 24
timeFrame: Baseline and Week 24
measure: Percentage changes From Baseline in TG at Week 24
timeFrame: Baseline and Week 24
measure: Absolute changes From Baseline in TG at Week 24
timeFrame: Baseline and Week 24
measure: The ratio of TC/HDL - C
timeFrame: Baseline and Week 24
measure: The ratio of Apo B/Apo A1
timeFrame: Baseline and Week 24
measure: Percentage of Participants With 50% or Greater Reduction in LDL-C From Baseline at Week 24
timeFrame: Baseline and Week 24
Other outcomes:
measure: Number of subjects who develop detectable anti-drug antibodies (ADAs)
timeFrame: from baseline to 32 weeks
Locations:
city: Beijing
country: China
facility: Beijing Anzhen Hospital Capital Medical University City:Beijing
state: Beijing
Study officials:
References: