Heterozygous Familial HypercholesterolemiaPhase 2NCT04173793
A Study of PCSK9 Inhibitor AK102 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
A Phase 2 study testing AK102 and Statins and/or Ezetimibe in people with Heterozygous Familial Hypercholesterolemia, run by Akeso at 2 sites in China.
Trial summary
This is a double-blind, randomized, placebo-controlled, multicenter study to evaluate the safety and efficacy of AK102 in patients with heterozygous familial hypercholesterolemia (HeFH).The primary objective of this study is to evaluate the efficacy of AK102 in patients with HeFH. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Heterozygous Familial Hypercholesterolemia
- What is being tested?
- AK102 and Statins and/or Ezetimibe
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 109 participants
- Where
- 2 sitesChina
- Study dates
- 18 Nov 2019to 26 Sep 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects with heterozygous familial hypercholesterolemia diagnosed by genetic confirmation or clinical diagnosis criteria.
- Stable on pre-existing, lipid-lowering therapies (statins with or without ezetimibe) for at least 4 weeks with no planned medication or dose change for the duration of study participation.
- Fasting Low-Density Lipoprotein Cholesterol (LDL-C) ≥ 70 mg/dL in patients with history of Atherosclerotic Cardiovascular Disease (ASCVD) or Fasting Low-Density Lipoprotein Cholesterol (LDL-C) ≥ 100 mg/dL in patients without history of Atherosclerotic Cardiovascular Disease (ASCVD).
- Fasting triglycerides ≤ 400 mg/dL.
- Body weight ≥ 40kg.
Who cannot join
- Subjects with homozygous FH (clinically or by genotyping).
- Receipt of LDL apheresis within 12 months prior to the first dose of Investigational product.
- Receipt of Lomitapide or Mipomersen within 5 months prior to the first dose of Investigational product.
- Prior use of PCSK9 inhibitors.
- Creatine kinase (CK) >3 times of the upper limit of normal (ULN).
- Aspartate Aminotransferase (AST) ≥ 2 x ULN.
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- Estimated Glomerular Filtration Rate (eGFR)≤ 30 mL/min/1.73m^2.
- Thyroid-Stimulating Hormone (TSH)> 1.5 x ULN or <1 x LLN.
- Type 1 diabetes, or type 2 diabetes that is or poorly controlled（HbA1c> 8.5%).
- Subjects with untreated or active chronic hepatitis B or active hepatitis C virus infections.
Trial locations and contacts
2 sites in 1 country.
China2 sites
Beijing Anzhen Hospital
Beijing, Beijing Municipality, 100029
Peking Union Medical College Hospital
Beijing, 100000
About this study
Official title: A Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Safety and Efficacy of AK102 in Patients With Heterozygous Familial Hypercholesterolemia
This is a double-blind, randomized, placebo-controlled, multicenter study to evaluate the safety and efficacy of AK102 in patients with heterozygous familial hypercholesterolemia (HeFH).The primary objective of this study is to evaluate the efficacy of AK102 in patients with HeFH.
- Being tested
- AK102, Placebo, Statins and/or Ezetimibe
- Keywords
- HeFH, LDL-C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AK102Drug
Administered by subcutaneous injection
PlaceboDrug
Administered by subcutaneous injection
Statins and/or EzetimibeDrug
Lipid-lowering therapies
| Study group | Role | What they receive |
|---|---|---|
| AK102 450 mg | Experimental | Participants received AK102 450 mg subcutaneous injection once every 4 weeks (Q4W) for 12 weeks Receives: Drug: AK102; Drug: Statins and/or Ezetimibe |
| AK102 300 mg | Experimental | Participants received AK102 300 mg subcutaneous injection once every 4 weeks (Q4W) for 12 weeks Receives: Drug: AK102; Drug: Statins and/or Ezetimibe |
| AK102 150 mg | Experimental | Participants received AK102 150 mg subcutaneous injection once every 2 weeks (Q2W) for 12 weeks Receives: Drug: AK102; Drug: Statins and/or Ezetimibe |
| Placebo Q4W | Placebo comparator | Participants received placebo subcutaneous injection once every 4 weeks (Q4W) for 12 weeks Receives: Drug: Placebo; Drug: Statins and/or Ezetimibe |
| Placebo Q2W | Placebo comparator | Participants received placebo subcutaneous injection once every 2 weeks (Q2W) for 12 weeks Receives: Drug: Placebo; Drug: Statins and/or Ezetimibe |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change from baseline in low-density lipoprotein cholesterol (LDL-C) at Week 12
Measured: At baseline and week 12
Secondary outcomes (12)
Percent change from baseline in low-density lipoprotein cholesterol (LDL-C)
Measured: From baseline through 12 weeks
Percent change from baseline in high-density lipoprotein cholesterol (HDL-C)
Measured: From baseline through 12 weeks
Percent change from baseline in non High-density lipoprotein (non-HDL) cholesterol
Measured: From baseline through 12 weeks
Percent change from baseline in serum Triglyceride (TG) cholesterol
Measured: From baseline through 12 weeks
Percent change from baseline in Apolipoprotein B (Apo B)
Measured: From baseline through 12 weeks
Percent change from baseline in Apolipoprotein A-I (ApoA-I)
Measured: From baseline through 12 weeks
Percent change from baseline in Lipoprotein(a) [Lp-(a)]
Measured: From baseline through 12 weeks
Percent change from baseline in Total Cholesterol(TC)
Measured: From baseline through 12 weeks
Incidence of treatment-emergent adverse events
Measured: From baseline through 12 weeks
Serum concentrations of AK102
Measured: From baseline through 12 weeks
Number of subjects who develop detectable anti-drug antibodies (ADAs)
The immunogenicity of AK102 will be assessed by summarizing the number of subjects who develop detectable anti-drug antibodies.
Measured: From baseline through 12 weeks
Change from baseline in proprotein convertase subtilisin/kexin type 9 (PCSK9)
Measured: From baseline through 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Nov 2019
- Study start18 Nov 2019
- Primary completion26 Sep 2022
- Study completion26 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heterozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
- More trials run by AkesoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 109 participants (actual)
Sponsor and investigators
- Lead sponsor
- Akeso
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- AD Pharmaceuticals Co., Ltd.
- Responsible party
- Sponsor
- Investigators
- Shuyang Zhang, MD, Principal investigator, Peking Union Medical College HospitalYujie Zhou, MD, Principal investigator, Beijing Anzhen Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04173793
- Sponsor's protocol ID
- AK102-201
- Status last verified
- Mar 2023
- First submitted
- 17 Nov 2019
- Last updated
- 2 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Safety and Efficacy of AK102 in Patients With Heterozygous Familial Hypercholesterolemia
- ClinicalTrials.gov ID
- NCT04173793
- Acronym / sponsor ID
- AK102-201
- Keywords
- HeFH, LDL-C
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 109 participants (actual)
- Lead sponsor
- AkesoIndustry (pharma, biotech, devices)
- Collaborators
- AD Pharmaceuticals Co., Ltd.
- Study start
- 18 Nov 2019 (actual)
- Primary completion
- 26 Sep 2022 (actual)
- Study completion
- 26 Sep 2022 (actual)
- First posted
- 22 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a double-blind, randomized, placebo-controlled, multicenter study to evaluate the safety and efficacy of AK102 in patients with heterozygous familial hypercholesterolemia (HeFH).The primary objective of this study is to evaluate the efficacy of AK102 in patients with HeFH.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| AK102 450 mg | Experimental | Drug: AK102, Drug: Statins and/or EzetimibeParticipants received AK102 450 mg subcutaneous injection once every 4 weeks (Q4W) for 12 weeks |
| AK102 300 mg | Experimental | Drug: AK102, Drug: Statins and/or EzetimibeParticipants received AK102 300 mg subcutaneous injection once every 4 weeks (Q4W) for 12 weeks |
| AK102 150 mg | Experimental | Drug: AK102, Drug: Statins and/or EzetimibeParticipants received AK102 150 mg subcutaneous injection once every 2 weeks (Q2W) for 12 weeks |
| Placebo Q4W | Placebo comparator | Drug: Placebo, Drug: Statins and/or EzetimibeParticipants received placebo subcutaneous injection once every 4 weeks (Q4W) for 12 weeks |
| Placebo Q2W | Placebo comparator | Drug: Placebo, Drug: Statins and/or EzetimibeParticipants received placebo subcutaneous injection once every 2 weeks (Q2W) for 12 weeks |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Subjects with heterozygous familial hypercholesterolemia diagnosed by genetic confirmation or clinical diagnosis criteria.
- Stable on pre-existing, lipid-lowering therapies (statins with or without ezetimibe) for at least 4 weeks with no planned medication or dose change for the duration of study participation.
- Fasting Low-Density Lipoprotein Cholesterol (LDL-C) ≥ 70 mg/dL in patients with history of Atherosclerotic Cardiovascular Disease (ASCVD) or Fasting Low-Density Lipoprotein Cholesterol (LDL-C) ≥ 100 mg/dL in patients without history of Atherosclerotic Cardiovascular Disease (ASCVD).
- Fasting triglycerides ≤ 400 mg/dL.
- Body weight ≥ 40kg.
Key exclusion criteria (10 listed)
- Subjects with homozygous FH (clinically or by genotyping).
- Receipt of LDL apheresis within 12 months prior to the first dose of Investigational product.
- Receipt of Lomitapide or Mipomersen within 5 months prior to the first dose of Investigational product.
- Prior use of PCSK9 inhibitors.
- Creatine kinase (CK) >3 times of the upper limit of normal (ULN).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AK102Administered by subcutaneous injection | Drug | AK102 150 mg, AK102 300 mg, AK102 450 mg |
| PlaceboAdministered by subcutaneous injection | Drug | Placebo Q2W, Placebo Q4W |
| Statins and/or EzetimibeLipid-lowering therapies | Drug | AK102 150 mg, AK102 300 mg, AK102 450 mg, Placebo Q2W, Placebo Q4W |
Primary outcome
Percent change from baseline in low-density lipoprotein cholesterol (LDL-C) at Week 12
Time frame: At baseline and week 12
12 secondary outcomes
Percent change from baseline in low-density lipoprotein cholesterol (LDL-C)
Time frame: From baseline through 12 weeks
Percent change from baseline in high-density lipoprotein cholesterol (HDL-C)
Time frame: From baseline through 12 weeks
Percent change from baseline in non High-density lipoprotein (non-HDL) cholesterol
Time frame: From baseline through 12 weeks
Percent change from baseline in serum Triglyceride (TG) cholesterol
Time frame: From baseline through 12 weeks
Percent change from baseline in Apolipoprotein B (Apo B)
Time frame: From baseline through 12 weeks
Percent change from baseline in Apolipoprotein A-I (ApoA-I)
Time frame: From baseline through 12 weeks
Percent change from baseline in Lipoprotein(a) [Lp-(a)]
Time frame: From baseline through 12 weeks
Percent change from baseline in Total Cholesterol(TC)
Time frame: From baseline through 12 weeks
Incidence of treatment-emergent adverse events
Time frame: From baseline through 12 weeks
Serum concentrations of AK102
Time frame: From baseline through 12 weeks
Number of subjects who develop detectable anti-drug antibodies (ADAs)
Time frame: From baseline through 12 weeks
Change from baseline in proprotein convertase subtilisin/kexin type 9 (PCSK9)
Time frame: From baseline through 12 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2023.
- Individual participant dataSharing planned: No
Related trials for Heterozygous Familial Hypercholesterolemia
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EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
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The ORIGIN-FH Study
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH)
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A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)
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Not yet recruitingPhase 2
A Study of VERVE-102 (LY4636581) in Adults With or at High Risk for Cardiovascular Disease
Atherosclerosis, Cardiovascular Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shuyang Zhang, MD principal investigator
Peking Union Medical College Hospital
Yujie Zhou, MD principal investigator
Beijing Anzhen Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AK102 and Statins and/or Ezetimibe in people with Heterozygous Familial Hypercholesterolemia, run by Akeso at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent change from baseline in low-density lipoprotein cholesterol (LDL-C) at Week 12 (time frame: At baseline and week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Mar 2023.