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

Phase 2b/3 Study to Assess the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia.

Status

Completed

Phases

Phase 2Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04031742

Enrollment

Registered

2019-07-24

Start date

2019-09-29

Completion date

2021-12-30

Last updated

2023-08-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Homozygous Familial Hypercholesterolemia

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.

Interventions

BIOLOGICALIBI306

Administered by subcutaneous injection

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Intervention model description

Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 \[ Time Frame: Baseline and Week 12 \]

Eligibility

Sex/Gender

ALL

Age

18 Years to 80 Years

Healthy volunteers

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

Design outcomes

Primary

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

Other

Measure	Time frame
Number of investigational product-related adverse events.	Baseline and Week 12
Number of ADA and Nab.	Baseline and Week 12

Countries

China

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026