A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia

Phase 2

Completed

• Conditions: Homozygous Familial Hypercholesterolemia
• Interventions: Biological: IBI306

• Registration Number: NCT04031742
• Lead Sponsor: Innovent Biologics (Suzhou) Co. Ltd.

Brief Summary

A study to evaluate safety and efficacy of IBI306 in subjects with homozygous familial hypercholesterolemia.

Detailed Description

This is a phase 2b/3 study to assess the efficacy and safety of IBI306 in subjects with homozygous familial hypercholesterolemia. Part 1 is an open-label, two-arm, multicenter pilot study to evaluate efficacy and safety of IBI306 in subjects with homozygous familial hypercholesterolemia. Part 2 is an open-label, single-arm, multicenter study to evaluate efficacy and safety of IBI306 in subjects with homozygous familial hypercholesterolemia.

Study Timeline

• Study First Submitted: 2019-07-22
• Study First Submitted QC: 2019-07-22
• Study First Posted: 2019-07-24
• Study Start: 2019-09-29
• Primary Completion: 2021-12-30
• Study Completion: 2021-12-30
• Status Verified: 2023-08
• Last Update Submitted: 2023-08-08
• Last Update Posted: 2023-08-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

1. Males and females ≥ 18 to ≤ 80 years of age
2. Diagnosis of homozygous familial hypercholesterolemia
3. LDL cholesterol ≥ 130 mg/dL (3.4mmol/L)
4. Triglyceride ≤ 400 mg/dL (4.5 mmol/L)
5. Bodyweight of ≥ 40 kg at screening

Exclusion Criteria

1. History of liver transplant
2. Uncontrolled hypertension
3. Moderate to severe renal dysfunction
4. Active liver disease or hepatic dysfunction
5. Known sensitivity to any of the products to be administered during dosing

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Part 1: IBI306	IBI306	Participants receive open-label IBI306 150 mg subcutaneously Q2W or 450 mg Q4W for 12 weeks.
Part 2: IBI306	IBI306	Participants receive open-label 450 mg Q4W subcutaneously for 12 weeks.

Primary Outcome Measures

Name	Time	Method
Part 2: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C)	Baseline and Week 12
Part 1: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C).	Baseline and Week 12

Trial Locations

Locations (1)

Beijing Anzhen Hospital, Capital Medical University; 🇨🇳 Beijing, China