A Study to Determine the Safety, Tolerability, and Efficacy of SHR-1209 in Patients With Familial Hypercholesterolemia

Open-label, Single-arm, Multicentre Study to Evaluate Efficacy and Safety of SHR-1209 in Subjects With Homozygous Familial Hypercholesterolemia

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 .

Study Overview

• Status: Unknown
• Conditions: Familial Hypercholesterolemia
• Intervention / Treatment: Drug: SHR-1209

• Study Type: Interventional
• Enrollment (Anticipated): 8
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Bin Zhang, Medial Manager; Phone Number: +86 13671548369; Email: zhangbin@hrglobe.cn
• Study Contact Backup: Name: Bo Zhu, Medical Director; Phone Number: +86 13380043088; Email: zhubo@hrglobe.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment group; SHR-1209 administered by subcutaneous injection Atorvastatin or Rosuvastatin combined with Ezetimibe oral	Drug: SHR-1209; SHR-1209 administered by subcutaneous injection Atorvastatin or Rosuvastatin combined with Ezetimibe oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percent Change from Baseline in LDL-C at Week 12	Week 12

Secondary Outcome Measures

Outcome Measure	Time Frame
Change From Baseline in LDL-C at Week 12	Week 12
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
Percent Change From Baseline in Triglycerides 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

Collaborators and Investigators

• Sponsor: Jiangsu HengRui Medicine Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Anticipated): September 1, 2020
• Primary Completion (Anticipated): June 30, 2021
• Study Completion (Anticipated): August 31, 2021

Study Registration Dates

• First Submitted: June 29, 2020
• First Submitted That Met QC Criteria: June 29, 2020
• First Posted (Actual): July 2, 2020

Study Record Updates

• Last Update Posted (Actual): July 2, 2020
• Last Update Submitted That Met QC Criteria: June 29, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Hyperlipidemias; Dyslipidemias; Lipid Metabolism, Inborn Errors; Hyperlipoproteinemias; Hypercholesterolemia; Hyperlipoproteinemia Type II
• Other Study ID Numbers: SHR-1209-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No