Homozygous Familial HypercholesterolemiaPhase 2NCT03933293
A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH
A Phase 2 study testing AK102, Statins and Ezetimibe in people with Homozygous Familial Hypercholesterolemia, run by Akeso at 2 sites in China.
Trial summary
AK102 is being developed for the treatment of HoFH. The study will be conducted in 2 parts, part 1 is open label, single arm study to evaluate the safety, tolerability and efficacy of PCSK9 inhibitor AK102, and part 2 is double blind, randomized, placebo controlled study to evaluate the efficacy and safety of PCSK9… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia
- What is being tested?
- AK102, Statins and Ezetimibe
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 2 sitesChina
- Study dates
- 13 May 2019to 15 Mar 2021
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females, ≥18 years of age with a diagnosis of homozygous familial hypercholesterolemia by genetic confirmation or a clinical diagnosis based on a history of an untreated low-density lipoprotein cholesterol (LDL-C) concentration >500 mg/deciliter (dL) [13 millimoles/liter (mmol/L)] together with either xanthoma before 10 years of age or evidence of heterozygous familial hypercholesterolemia in both parents
- Stable on pre-existing, lipid-lowering therapies (statins in combination with ezetimibe) for at least 4 weeks with no planned medication or dose change for the duration of study participation
- Fasting central lab LDL-C concentration >130 mg/dL (3.4 mmol/L) and triglyceride concentration <400 mg/dL (4.5 mmol/L).
- Body weight of 40 kilograms (kg) or greater at screening
Who cannot join
- Received LDL plasma replacement therapy within 8 weeks before Investigational product administration
- Received Lomitapide or Mipomersen within 5 months before Investigational product administration
- Received prior treatment with PCSK9 inhibitors or AK102.
- Unexplained creatine kinase (CK) ≥ 5 times the upper limit of normal (ULN)
- Subjects with untreated chronic hepatitis B or chronic hepatitis B virus (HBV) DNA exceeding 500 IU/ mL or active hepatitis C virus (HCV) should be excluded. Subjects with non-active HBsAg carriers, treated and stable hepatitis B (HBV DNA <500 IU/ mL) , and cured hepatitis C can be enrolled. Subjects with positive HCV antibodies are eligible only if the HCV RNA test results are negative.
- Known allergic reactions to any ingredients of AK102
- Any other condition(s) that would compromise the safety of the patient or compromise the quality of the clinical study as judged by the Investigator and/or Medical Monitor.
Trial locations and contacts
2 sites in 1 country.
China2 sites
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100000
Beijing Anzhen Hospital
Beijing, Beijing Municipality, 100029
About this study
Official title: A Phase 2 Study to Evaluate the Safety and Efficacy of PCSK9 Inhibitor AK102 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
AK102 is being developed for the treatment of HoFH. The study will be conducted in 2 parts, part 1 is open label, single arm study to evaluate the safety, tolerability and efficacy of PCSK9 inhibitor AK102, and part 2 is double blind, randomized, placebo controlled study to evaluate the efficacy and safety of PCSK9 inhibitor AK102. The treatment period will last 12 week.
- Conditions
- Homozygous Familial Hypercholesterolemia
- Being tested
- AK102, Statins, Ezetimibe
- Keywords
- HoFH, LDL-C
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AK102Drug
450mg, Q4W, subcutaneous injection
StatinsDrug
Lipid-lowering therapies
EzetimibeDrug
Lipid-lowering therapies
| Study group | Role | What they receive |
|---|---|---|
| AK102 | Experimental | 450mg AK102, Q4W, subcutaneous injection Receives: Drug: AK102; Drug: Statins; Drug: Ezetimibe |
| placebo | Placebo comparator | Placebo, Q4W, subcutaneous injection Receives: Drug: Statins; Drug: 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: Week 12
Incidence of treatment-emergent adverse events as assessed by CTCAE V5.0（only for part 1）
Measured: From baseline through 12 weeks
Secondary outcomes (9)
Percent Change From Baseline in High-density lipoprotein (HDL) cholesterol
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)
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 (Apo A-I)
Measured: From baseline through 12 weeks
Percent Change From Baseline in Total Cholesterol(TC)
Measured: From baseline through 12 weeks
Change From Baseline in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9)
Measured: From baseline through 12 weeks
Concentrations of AK102 in Serum
Measured: Part 1: Day 1,Day 2, Day 4, Day 8, Day 15, Day 22, D29, D57. Part 2: Day 1, Day 29, Day 57
Number of subjects who develop detectable anti-drug antibodies (ADAs)
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 posted1 May 2019
- Study start13 May 2019
- Primary completion15 Mar 2021
- Study completion15 Mar 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Homozygous 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 10 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 HospitalLvya Wang, Principal investigator, Beijing Anzhen Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03933293
- Sponsor's protocol ID
- AK102-202
- Status last verified
- Mar 2023
- First submitted
- 28 Apr 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 Phase 2 Study to Evaluate the Safety and Efficacy of PCSK9 Inhibitor AK102 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
- ClinicalTrials.gov ID
- NCT03933293
- Acronym / sponsor ID
- AK102-202
- Conditions
- Homozygous Familial Hypercholesterolemia
- Keywords
- HoFH, LDL-C
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- AkesoIndustry (pharma, biotech, devices)
- Collaborators
- AD Pharmaceuticals Co., Ltd.
- Study start
- 13 May 2019 (actual)
- Primary completion
- 15 Mar 2021 (actual)
- Study completion
- 15 Mar 2021 (actual)
- First posted
- 1 May 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.
AK102 is being developed for the treatment of HoFH. The study will be conducted in 2 parts, part 1 is open label, single arm study to evaluate the safety, tolerability and efficacy of PCSK9 inhibitor AK102, and part 2 is double blind, randomized, placebo controlled study to evaluate the efficacy and safety of PCSK9 inhibitor AK102. The treatment period will last 12 week.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AK102 | Experimental | Drug: AK102, Drug: Statins, Drug: Ezetimibe450mg AK102, Q4W, subcutaneous injection |
| placebo | Placebo comparator | Drug: Statins, Drug: EzetimibePlacebo, Q4W, subcutaneous injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Males and females, ≥18 years of age with a diagnosis of homozygous familial hypercholesterolemia by genetic confirmation or a clinical diagnosis based on a history of an untreated low-density lipoprotein cholesterol (LDL-C) concentration >500 mg/deciliter (dL) [13 millimoles/liter (mmol/L)] together with either xanthoma before 10 years of age or evidence of heterozygous familial hypercholesterolemia in both parents
- Stable on pre-existing, lipid-lowering therapies (statins in combination with ezetimibe) for at least 4 weeks with no planned medication or dose change for the duration of study participation
- Fasting central lab LDL-C concentration >130 mg/dL (3.4 mmol/L) and triglyceride concentration <400 mg/dL (4.5 mmol/L).
- Body weight of 40 kilograms (kg) or greater at screening
Key exclusion criteria (7 listed)
- Received LDL plasma replacement therapy within 8 weeks before Investigational product administration
- Received Lomitapide or Mipomersen within 5 months before Investigational product administration
- Received prior treatment with PCSK9 inhibitors or AK102.
- Unexplained creatine kinase (CK) ≥ 5 times the upper limit of normal (ULN)
- Subjects with untreated chronic hepatitis B or chronic hepatitis B virus (HBV) DNA exceeding 500 IU/ mL or active hepatitis C virus (HCV) should be excluded. Subjects with non-active HBsAg carriers, treated and stable hepatitis B (HBV DNA <500 IU/ mL) , and cured hepatitis C can be enrolled. Subjects with positive HCV antibodies are eligible only if the HCV RNA test results are negative.
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 |
|---|---|---|
| AK102450mg, Q4W, subcutaneous injection | Drug | AK102 |
| StatinsLipid-lowering therapies | Drug | AK102, placebo |
| EzetimibeLipid-lowering therapies | Drug | AK102, placebo |
Primary outcome
Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12
Time frame: Week 12
Incidence of treatment-emergent adverse events as assessed by CTCAE V5.0（only for part 1）
Time frame: From baseline through 12 weeks
9 secondary outcomes
Percent Change From Baseline in High-density lipoprotein (HDL) cholesterol
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)
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 (Apo A-I)
Time frame: From baseline through 12 weeks
Percent Change From Baseline in Total Cholesterol(TC)
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
Concentrations of AK102 in Serum
Time frame: Part 1: Day 1,Day 2, Day 4, Day 8, Day 15, Day 22, D29, D57. Part 2: Day 1, Day 29, Day 57
Number of subjects who develop detectable anti-drug antibodies (ADAs)
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 Homozygous Familial Hypercholesterolemia
Active, not recruitingObservational study
Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE)
RecruitingPhase 3
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Recruiting
The ORIGIN-FH Study
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH)
RecruitingPhase 3
Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies
Heterozygous or Homozygous Familial Hypercholesterolemia
RecruitingPhase 1
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
Cardiovascular, Metabolic 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
Lvya Wang 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, Statins and Ezetimibe in people with Homozygous 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 4 inclusion and 7 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: Week 12). The study lists 2 primary outcomes in all. 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.