Clinical trial NCT04455581: A Study to Determine the Safety, Tolerability, and Efficacy of SHR-1209 in Patients With Familial Hypercholesterolemia
Official title: Open-label, Single-arm, Multicentre Study to Evaluate Efficacy and Safety of SHR-1209 in Subjects With Homozygous Familial Hypercholesterolemia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Familial Hypercholesterolemia
Lead sponsor: Jiangsu HengRui Medicine Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2020-07-02
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The study is being conducted to evaluate the efficacy, safety and tolerability of SHR-1209 in subjects with familial hypercholesterolemia. 8 eligible patients (aged ≥18 years) with familial hypercholesterolemia, on stable maximum tolerable dose lipid-regulating therapy for at least 28 days, to receive subcutaneous SHR-1209, follow up 8 weeks. The primary endpoint was percentage change in LDL cholesterol from baseline at week 12 .


Eligibility criteria: Inclusion Criteria:

* Males and females ≥ 18 years of age
* Diagnosis of homozygous familial hypercholesterolemia
* Stable lipid-lowering therapies for at least 28 days
* LDL cholesterol ≥ 130 mg/dl (3.4 mmol/L)
* Triglyceride ≤ 400 mg/dL (4.5 mmol/L)
* Bodyweight of ≥ 40 kg at screening.

Exclusion Criteria:

* LDL or plasma apheresis within 8 weeks prior to randomization
* New York Heart Association (NYHA) class III or IV or last known left ventricular ejection fraction \< 30%
* Myocardial infarction, unstable angina, percutaneous coronary intervention, coronary artery bypass graft or stroke within 3 months of randomization, Planned cardiac surgery or revascularization, Uncontrolled cardiac arrhythmia
* Liver transplant history.
* Uncontrolled hypertension.
* Moderate to severe renal dysfunction.
* Active liver disease or hepatic dysfunction.
* Known sensitivity to any of the products to be administered during dosing

Primary outcomes:
measure: Percent Change from Baseline in LDL-C at Week 12
timeFrame: Week 12
Secondary outcomes:
measure: Change From Baseline in LDL-C at Week 12
timeFrame: Week 12
measure: Percent Change From Baseline in LDL-C at the Mean of Weeks 10 and 12
timeFrame: Weeks 10 and 12
measure: Change From Baseline in LDL-C at the Mean of Weeks 10 and 12
timeFrame: Weeks 10 and 12
measure: Percent Change From Baseline in the Total Cholesterol at the Mean of Weeks 10 and 12
timeFrame: Weeks 10 and 12
measure: Percent Change From Baseline in the Total Cholesterol at Week 12
timeFrame: Week 12
measure: Percent Change From Baseline in HDL-C at the Mean of Weeks 10 and 12
timeFrame: Weeks 10 and 12
measure: Percent Change From Baseline in HDL-C at Week 12
timeFrame: Week 12
measure: Percent Change From Baseline in Non-HDL-C at the Mean of Weeks 10 and 12
timeFrame: Weeks 10 and 12
measure: Percent Change From Baseline in Non-HDL-C at Week 12
timeFrame: Week 12
measure: Percent Change From Baseline in Triglycerides at the Mean of Weeks 10 and 12
timeFrame: Weeks 10 and 12
measure: Percent Change From Baseline in Triglycerides at Week 12
timeFrame: Week 12
measure: Percent Change From Baseline in Apo B at the Mean of Weeks 10 and 12
timeFrame: Weeks 10 and 12
measure: Percent Change From Baseline in ApoB at Week 12
timeFrame: Week 12
measure: Percent Change From Baseline in Apo A1 at the Mean of Weeks 10 and 12
timeFrame: Weeks 10 and 12
measure: Percent Change From Baseline in Apo A1 at Week 12
timeFrame: Week 12
measure: Percent Change From Baseline in Lipo(a) at the Mean of Weeks 10 and 12
timeFrame: Weeks 10 and 12
measure: Percent Change From Baseline in Lipo(a) at Week 12
timeFrame: Week 12
measure: Number of investigational product-related adverse events
timeFrame: Weeks 12 and 20
measure: Number of ADA and Nab
timeFrame: Week 20
Other outcomes:
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