HypercholesterolemiaPhase 1NCT05762276
A Study to Investigate the Safety, Tolerability, Immunogenicity, and Pharmacodynamics of VXX-401 Administered IM in Adult Participants
A Phase 1 study testing VXX-401 in people with Hypercholesterolemia, run by Axxium Life, Inc. at 5 sites in Australia.
Trial summary
This first-in-human (FIH) study of VXX-401, an anti-PCSK9 peptide-based immunotherapeutic candidate, is designed to assess the safety, tolerability, immunogenicity, and pharmacodynamics (PD) of VXX-401 and to determine an optimal dose regimen for LDL-C lowering in subsequent clinical trials. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Hypercholesterolemia
- What is being tested?
- VXX-401
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 5 sitesAustralia
- Study dates
- 7 Mar 2023to 25 Jul 2024
- Phase
- Phase 1
- 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
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Male or female participants aged 18 to 75 years old, inclusive, at time of informed consent.
- LDL-C level = 2.59 mmol/L - 4.89mmol/L
- Body mass index between 18 and 35 kg/m2, inclusive at Screening, and with a minimum weight of 50 kg.
- Male participants and their partners of childbearing potential must commit to the use of highly effective contraceptives for the study duration and for at least 12 weeks after the last dose. Men must refrain from donating sperm during this same period.
- Female participants must be of nonchildbearing potential, or, for women of childbearing potential, must be willing to practice at least one form of highly effective contraception throughout the duration of the study and for at least 24 weeks following the last dose. Female participants must refrain from donating reproductive tissue during this same period.
Who cannot join
- Subjects considered high risk or very high risk for ASCVD and requiring immediate treatment with LLT according to the clinical judgement of the investigator.
- History of confirmed anergy (i.e., not able to mount an immunological response) or history of immunization failure in the 5 years prior to the Screening Visit.
- Presence of fever >38°C or other signs or symptoms of acute disease within 1 week before the Screening and/or Visit 1; Screening and/or Visit 1 may be rescheduled at the discretion of the Investigator but must occur within the 4-week window.
- Known disturbance of coagulation or medication (see prohibited medications criterion below); bleeding disorder (e.g., factor deficiency, coagulopathy, or platelet disorder), or prior history of significant bleeding or bruising following IM injections or venipuncture.
- Triglycerides > 5.65 mmol/L
- Has a history of clinically significant medical disorder or psychiatric conditions, which in the opinion of the investigator may compromise the participant's safety and ability to comply with study procedures or abide by study restrictions.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Australia5 sites
Northern Beaches Clinical Research
Brookvale, New South Wales
Emeritus Research
Melbourne, Victoria
Sutherland Shire Clinical Research
Miranda, New South Wales
University of the Sunshine Coast (USC)
Morayfield, Queensland
Emeritus Research
Sydney, New South Wales
About this study
Official title: A Phase 1, First-in-Human, Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Pharmacodynamics of VXX-401 in Healthy Adults
This first-in-human (FIH) study of VXX-401, an anti-PCSK9 peptide-based immunotherapeutic candidate, is designed to assess the safety, tolerability, immunogenicity, and pharmacodynamics (PD) of VXX-401 and to determine an optimal dose regimen for LDL-C lowering in subsequent clinical trials.
Read the detailed description
This is multisite, multidose regimen, phase 1, first-in-human study of VXX-401, a synthetic peptide-based active immunotherapy candidate for preventing and treating hypercholesterolemia. The study will include Screening, Treatment, and Follow-up Periods. This study will enroll participants who are naïve to statin use. Each cohort from A to D is planned to randomize approximately 12 participants to receive doses of VXX-401 or placebo in a 3:1 ratio. Cohorts E and F will dose approximately 8 participants in each cohort to receive doses of VXX-401. No participants will be administered placebo in Cohorts E and F. It is planned to test up to 6 dose regimens of VXX-401, administered by IM injection into the deltoid muscle (and additionally in the thigh for the first dose in Cohorts E-F.). All eligible participants will receive a priming regimen at Week 0 (Baseline, Day 1), Week 4, and Week 12, in Cohorts A, C, E and F, and additionally at Week 8 in Cohorts B and D. The last dose administration will be at Week 12.
- Conditions
- Hypercholesterolemia
- Being tested
- VXX-401, Placebo
- Medical subject headings
- Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VXX-401Drug
A synthetic PCSK9 peptide-based immunotherapy
PlaceboBiological
Normal saline
| Study group | Role | What they receive |
|---|---|---|
| VXX-401 Cohort A | Experimental | VXX-401 100mcg administered by intramuscular (IM) injection at Week 0, Week 4, and Week 12 Receives: Drug: VXX-401 |
| VXX-401 Cohort B | Experimental | VXX-401 100mcg administered by intramuscular (IM) injection at Week 0, Week 4, Week 8 and Week 12 Receives: Drug: VXX-401 |
| VXX-401 Cohort C | Experimental | VXX-401 300mcg administered by intramuscular (IM) injection at Week 0, Week 4, and Week 12 Receives: Drug: VXX-401 |
| VXX-401 Cohort D | Experimental | VXX-401 300mcg administered by intramuscular (IM) injection at Week 0, Week 4, Week 8 and Week 12 Receives: Drug: VXX-401 |
| Placebo Cohort A and C | Placebo comparator | Placebo administered by intramuscular (IM) injection at Week 0, Week 4, and Week 12 Receives: Biological: Placebo |
| Placebo Cohort B and D | Placebo comparator | Placebo administered by intramuscular (IM) injection at Week 0, Week 4, Week 8 and Week 12 Receives: Biological: Placebo |
| VXX-401 Cohort E | Experimental | VXX-401 900mcg administered by intramuscular (IM) injection at Week 0. VXX-401 100 mcg administered by intramuscular (IM) injection at Week 4 and Week 12. Receives: Drug: VXX-401 |
| VXX-401 Cohort F | Experimental | VXX-401 900mcg administered by intramuscular (IM) injection at Week 0. VXX-401 300 mcg administered by intramuscular (IM) injection at Week 4 and Week 12. Receives: Drug: VXX-401 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of adverse events
Safety and tolerability: rates of adverse events (AEs), medically attended adverse events (MAAEs), local (injection site) and systemic (generalized) reactions (i.e., reactogenicity), clinical laboratory assessments (e.g., chemistry, hematology, urinalysis, lipid profile), serum cytokine release, vital signs, physical examinations, and electrocardiograms (ECGs) through the end of the study.
Measured: 30 weeks
Immunogenicity
Immunogenicity will be measured by serum anti-PCSK9 antibody titers
Measured: Baseline to Week 16, 20, 24, and 30
Immunogenicity
Seroconversion two-fold and four-fold from baseline
Measured: Baseline to Week 16, 20, 24, and 30
Determine optimal VXX-401 dose regimen
Measured by serum anti-PCSK9 antibody titers
Measured: Baseline to Week 16, 20, 24, and 30
Secondary outcomes (1)
Evaluation of low-density lipoprotein-cholesterol (LDL-C) reduction
Percent change from baseline in serum LDL-C concentration
Measured: Baseline to Week 16, 20, 24, and 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2023
- Study start7 Mar 2023
- Primary completion25 Jul 2024
- Study completion25 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HypercholesterolemiaStudies of the same condition, open and under way.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Axxium Life, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Responsible party
- Sponsor
- Investigators
- Sasha Rumyantsev, Study director, Vaxxinity, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05762276
- Sponsor's protocol ID
- VXX-401-101
- Status last verified
- Nov 2026
- First submitted
- 20 Feb 2023
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- 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 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, First-in-Human, Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Pharmacodynamics of VXX-401 in Healthy Adults
- ClinicalTrials.gov ID
- NCT05762276
- Acronym / sponsor ID
- VXX-401-101
- Conditions
- Hypercholesterolemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Axxium Life, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Study start
- 7 Mar 2023 (actual)
- Primary completion
- 25 Jul 2024 (actual)
- Study completion
- 25 Jul 2024 (actual)
- First posted
- 9 Mar 2023 (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 first-in-human (FIH) study of VXX-401, an anti-PCSK9 peptide-based immunotherapeutic candidate, is designed to assess the safety, tolerability, immunogenicity, and pharmacodynamics (PD) of VXX-401 and to determine an optimal dose regimen for LDL-C lowering in subsequent clinical trials.
Detailed description
This is multisite, multidose regimen, phase 1, first-in-human study of VXX-401, a synthetic peptide-based active immunotherapy candidate for preventing and treating hypercholesterolemia. The study will include Screening, Treatment, and Follow-up Periods. This study will enroll participants who are naïve to statin use. Each cohort from A to D is planned to randomize approximately 12 participants to receive doses of VXX-401 or placebo in a 3:1 ratio. Cohorts E and F will dose approximately 8 participants in each cohort to receive doses of VXX-401. No participants will be administered placebo in Cohorts E and F. It is planned to test up to 6 dose regimens of VXX-401, administered by IM injection into the deltoid muscle (and additionally in the thigh for the first dose in Cohorts E-F.). All eligible participants will receive a priming regimen at Week 0 (Baseline, Day 1), Week 4, and Week 12, in Cohorts A, C, E and F, and additionally at Week 8 in Cohorts B and D. The last dose administration will be at Week 12.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| VXX-401 Cohort A | Experimental | Drug: VXX-401VXX-401 100mcg administered by intramuscular (IM) injection at Week 0, Week 4, and Week 12 |
| VXX-401 Cohort B | Experimental | Drug: VXX-401VXX-401 100mcg administered by intramuscular (IM) injection at Week 0, Week 4, Week 8 and Week 12 |
| VXX-401 Cohort C | Experimental | Drug: VXX-401VXX-401 300mcg administered by intramuscular (IM) injection at Week 0, Week 4, and Week 12 |
| VXX-401 Cohort D | Experimental | Drug: VXX-401VXX-401 300mcg administered by intramuscular (IM) injection at Week 0, Week 4, Week 8 and Week 12 |
| Placebo Cohort A and C | Placebo comparator | Biological: PlaceboPlacebo administered by intramuscular (IM) injection at Week 0, Week 4, and Week 12 |
| Placebo Cohort B and D | Placebo comparator | Biological: PlaceboPlacebo administered by intramuscular (IM) injection at Week 0, Week 4, Week 8 and Week 12 |
| VXX-401 Cohort E | Experimental | Drug: VXX-401VXX-401 900mcg administered by intramuscular (IM) injection at Week 0. VXX-401 100 mcg administered by intramuscular (IM) injection at Week 4 and Week 12. |
| VXX-401 Cohort F | Experimental | Drug: VXX-401VXX-401 900mcg administered by intramuscular (IM) injection at Week 0. VXX-401 300 mcg administered by intramuscular (IM) injection at Week 4 and Week 12. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female participants aged 18 to 75 years old, inclusive, at time of informed consent.
- LDL-C level = 2.59 mmol/L - 4.89mmol/L
- Body mass index between 18 and 35 kg/m2, inclusive at Screening, and with a minimum weight of 50 kg.
- Male participants and their partners of childbearing potential must commit to the use of highly effective contraceptives for the study duration and for at least 12 weeks after the last dose. Men must refrain from donating sperm during this same period.
- Female participants must be of nonchildbearing potential, or, for women of childbearing potential, must be willing to practice at least one form of highly effective contraception throughout the duration of the study and for at least 24 weeks following the last dose. Female participants must refrain from donating reproductive tissue during this same period.
Key exclusion criteria (6 listed)
- Subjects considered high risk or very high risk for ASCVD and requiring immediate treatment with LLT according to the clinical judgement of the investigator.
- History of confirmed anergy (i.e., not able to mount an immunological response) or history of immunization failure in the 5 years prior to the Screening Visit.
- Presence of fever >38°C or other signs or symptoms of acute disease within 1 week before the Screening and/or Visit 1; Screening and/or Visit 1 may be rescheduled at the discretion of the Investigator but must occur within the 4-week window.
- Known disturbance of coagulation or medication (see prohibited medications criterion below); bleeding disorder (e.g., factor deficiency, coagulopathy, or platelet disorder), or prior history of significant bleeding or bruising following IM injections or venipuncture.
- Triglycerides > 5.65 mmol/L
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 |
|---|---|---|
| VXX-401A synthetic PCSK9 peptide-based immunotherapy | Drug | VXX-401 Cohort A, VXX-401 Cohort B, VXX-401 Cohort C, VXX-401 Cohort D, VXX-401 Cohort E, VXX-401 Cohort F |
| PlaceboNormal saline | Biological | Placebo Cohort A and C, Placebo Cohort B and D |
Primary outcome
Frequency of adverse events
Time frame: 30 weeks
Safety and tolerability: rates of adverse events (AEs), medically attended adverse events (MAAEs), local (injection site) and systemic (generalized) reactions (i.e., reactogenicity), clinical laboratory assessments (e.g., chemistry, hematology, urinalysis, lipid profile), serum cytokine release, vital signs, physical examinations, and electrocardiograms (ECGs) through the end of the study.
Immunogenicity
Time frame: Baseline to Week 16, 20, 24, and 30
Immunogenicity will be measured by serum anti-PCSK9 antibody titers
Immunogenicity
Time frame: Baseline to Week 16, 20, 24, and 30
Seroconversion two-fold and four-fold from baseline
Determine optimal VXX-401 dose regimen
Time frame: Baseline to Week 16, 20, 24, and 30
Measured by serum anti-PCSK9 antibody titers
1 secondary outcome
Evaluation of low-density lipoprotein-cholesterol (LDL-C) reduction
Time frame: Baseline to Week 16, 20, 24, and 30
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 4 Nov 2026.
- Individual participant dataSharing planned: No
Related trials for Hypercholesterolemia
Active, not recruitingObservational study
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A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients.
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Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
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Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
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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.
Sasha Rumyantsev study director
Vaxxinity, Inc.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VXX-401 in people with Hypercholesterolemia, run by Axxium Life, Inc. at 5 sites in Australia.
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 6 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 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Australia. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Frequency of adverse events (time frame: 30 weeks). The study lists 4 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 4 Nov 2026.