Familial Hypercholesterolemia
Conditions
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 .
Interventions
DRUGSHR-1209
SHR-1209 administered by subcutaneous injection Atorvastatin or Rosuvastatin combined with Ezetimibe oral
Sponsors
Jiangsu HengRui Medicine Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent Change from Baseline in LDL-C at Week 12 | Week 12 |
Secondary
| Measure | Time frame |
|---|---|
| Percent Change From Baseline in LDL-C at the Mean of Weeks 10 and 12 | Weeks 10 and 12 |
| Change From Baseline in LDL-C at the Mean of Weeks 10 and 12 | Weeks 10 and 12 |
| Percent Change From Baseline in the Total Cholesterol at the Mean of Weeks 10 and 12 | Weeks 10 and 12 |
| Percent Change From Baseline in the Total Cholesterol at Week 12 | Week 12 |
| Percent Change From Baseline in HDL-C at the Mean of Weeks 10 and 12 | Weeks 10 and 12 |
| Percent Change From Baseline in HDL-C at Week 12 | Week 12 |
| Percent Change From Baseline in Non-HDL-C at the Mean of Weeks 10 and 12 | Weeks 10 and 12 |
| Percent Change From Baseline in Non-HDL-C at Week 12 | Week 12 |
| Percent Change From Baseline in Triglycerides at the Mean of Weeks 10 and 12 | Weeks 10 and 12 |
| Change From Baseline in LDL-C at Week 12 | Week 12 |
| Percent Change From Baseline in Apo B at the Mean of Weeks 10 and 12 | Weeks 10 and 12 |
| Percent Change From Baseline in ApoB at Week 12 | Week 12 |
| Percent Change From Baseline in Apo A1 at the Mean of Weeks 10 and 12 | Weeks 10 and 12 |
| Percent Change From Baseline in Apo A1 at Week 12 | Week 12 |
| Percent Change From Baseline in Lipo(a) at the Mean of Weeks 10 and 12 | Weeks 10 and 12 |
| Percent Change From Baseline in Lipo(a) at Week 12 | Week 12 |
| Number of investigational product-related adverse events | Weeks 12 and 20 |
| Number of ADA and Nab | Week 20 |
| Percent Change From Baseline in Triglycerides at Week 12 | Week 12 |
Contacts
Primary ContactBin Zhang, Medial Manager
Backup ContactBo Zhu, Medical Director
Outcome results
None listed