Safety and Tolerability in Healthy VolunteersPhase 1NCT04535752
A Single and Multiple Ascending Dose Study of ANX009 in Normal Healthy Volunteers (NHV)
A Phase 1 study testing ANX009 in people with Safety and Tolerability in Healthy Volunteers, run by Annexon, Inc. at 1 site in Australia.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmakokinetics and pharmacodynamics of single and repeated doses of ANX009 Read more about this study
- Who is it for?
- Ages 18 to 59 yearsHealthy volunteers can take part
- Conditions the study is for
- Safety and Tolerability in Healthy Volunteers
- What is being tested?
- ANX009
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- 1 siteAustralia
- Study dates
- 30 Oct 2020to 26 Jun 2021
- 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 59 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy male and non-pregnant, non-lactating female volunteers ≥18 to 59 years of age.
- Females must be postmenopausal, surgically sterilized or willing and able to use highly effective methods of contraception from screening through the final study visit.
- Males with a partner of childbearing potential must agree to use contraception from Screening through the final study visit.
- Documented history within 5 years of screening of previous vaccination against encapsulated bacterial pathogens (MAD cohorts only).
- Complete the full sequence of protocol-related doses, procedures and evaluations.
- No alcohol and drugs of abuse at screening and baseline or through study completion.
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- Discontinue use of nutritional supplements and prescription and over-the-counter medications (vitamins are allowed).
- No new tattoos/piercings or elective surgery from screening through the End of Study visit
- Ability to understand and provide written informed consent.
Who cannot join
- Subjects must not meet any of the following criteria:
- Clinically significant, ongoing illness or medical condition that would jeopardize the safety of the subject, limit participation, or compromise the interpretation of the safety data derived from the subject.
- Clinically significant findings on the screening or Baseline ECG or physical examination.
- Clinically significant abnormalities on screening or Baseline laboratory assessments.
- An ANA titer ≥ 1:160.
- History of any autoimmune disease.
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- History of meningitis or septicemia.
- Clinically significant infection that required medical intervention (not including antibiotic prophylaxis) within 1 month prior to study drug dosing.
- Known genetic deficiencies of the complement cascade system or immunodeficiency.
- Treatment with an investigational therapeutic agent within 30 days prior to study drug dosing.
- Use of immunosuppressants or corticosteroids within 30 days prior to study drug dosing.
- Active alcohol abuse, drug abuse or substance abuse.
- Hypersensitivity to any of the excipients in the ANX009 drug product or active substance.
- History of previous sensitivities or allergic or anaphylactic reactions to previous medication injections.
- Positive for HIV Ab, Hepatitis C Ab or Hepatitis B surface antigen (HBsAg) at screening.
- Body weight less than 50 kg or greater than 125 kg.
- BMI less than 18 or greater than 30 (Asians greater than 27).
- Current smoker defined as any occasional or daily smoking of tobacco products
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Double-blind, Placebo-controlled Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Subcutaneous ANX009 in Normal Healthy Volunteers (NHV)
The purpose of this study is to evaluate the safety, tolerability, pharmakokinetics and pharmacodynamics of single and repeated doses of ANX009
Read the detailed description
In this first in human, phase 1, randomized, double-blind, placebo-controlled study, single and multiple ascending doses of ANX009 or placebo will be administered to 48 healthy subjects.
Single Ascending Dose (SAD): Each SAD subject will participate for approximately 4 weeks (3 nights in-clinic confinement).
Multiple Ascending Dose: Each MAD subject will participate for approximately 6 weeks (17 nights in-clinic confinement).
All subjects will be contacted (in clinic visit or phone call) 6 months after study completion.
- Being tested
- ANX009, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANX009Drug
Also called 009
Single or multiple ascending dose
PlaceboDrug
Also called matching placebo
Single or multiple ascending dose
| Study group | Role | What they receive |
|---|---|---|
| ANX009, Single Ascending Doses | Experimental | Single dose of ANX009 with a 7-day follow-up before escalation to the next dose level. Receives: Drug: ANX009 |
| Placebo, Single Ascending Doses | Placebo comparator | Single doses of matching placebo Receives: Drug: Placebo |
| ANX009, Multiple Ascending Doses | Experimental | ANX009 once daily on Days 1-14 Receives: Drug: ANX009 |
| Placebo, Multiple doses | Placebo comparator | Matching placebo once daily on Days 1-14 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Number of Participants Who Experienced Treatment-Emergent Adverse Events
Incidence and severity of treatment-emergent adverse events (AEs). AEs will be coded using MedDRA and severity of AEs will be graded using National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE).
Measured: [Time Frame: Up to Day 29 for SAD; up to Day 43 for MAD]
Secondary outcomes (6)
Pharmacodynamics: Total Amount of Complement Protein in Blood (CH50)
Serum samples will be obtained to determine the amount of CH50. CH50 will be measured at a local laboratory. CH50 will be measured at a local laboratory.
Measured: Up to Week 6
Pharmacodynamics: Amount of C1 in Blood (C1q)
Serum samples will be obtained to determine the amount of C1q. C1q will be measured using a validated enzyme-linked immunosorbent assay (ELISA) method.
Measured: Up to Week 6
Pharmacokinetic: Maximum Observed Serum Concentration (Cmax) of ANX009
Single-dose Cmax (Day 1 in SAD and MAD) and multiple-dose Cmax (Day 14 in MAD) will be determined. Blood samples will be obtained, and serum concentrations determined using a validated ELISA method.
Measured: Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)
Pharmacokinetic: Time to Maximum Observed Serum Concentration (Tmax) of ANX009
Tmax will be determined on Day 1 in SAD and MAD and on Day 14 in MAD. Blood samples will be obtained, and serum concentrations determined using a validated enzyme-linked immunosorbent assay (ELISA) method.
Measured: Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)
Pharmacokinetic: Area Under the ANX009 Serum Concentration-Time Curve to Last Sample (AUC 0-t) and extrapolated through infinity (AUC 0-inf)
AUC 0-t will be determined on Day 1 in SAD and on Day 14 in MAD. Blood samples will be obtained, and serum concentrations determined using a validated enzyme-linked immunosorbent assay (ELISA) method.
Measured: Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)
Pharmacokinetic: Terminal Half-Life (t1/2) of ANX009
Half-life will be determined on Day 1 in SAD and on Day 1 and Day 14 in MAD. Blood samples will be obtained, and serum concentrations determined using a validated enzyme-linked immunosorbent assay (ELISA) method
Measured: Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2020
- Study start30 Oct 2020
- Primary completion26 Jun 2021
- Study completion26 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Safety and Tolerability in Healthy VolunteersStudies 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
- Basic science
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Annexon, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Nucleus Network Ltd
- Responsible party
- Sponsor
- Investigators
- Eric Humphriss, MBA, Study director, Annexon Director, Global Clinical Operations
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04535752
- Sponsor's protocol ID
- ANX009-NHV-01
- Status last verified
- Aug 2021
- First submitted
- 24 Aug 2020
- Last updated
- 30 Aug 2021
- 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 30 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Double-blind, Placebo-controlled Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Subcutaneous ANX009 in Normal Healthy Volunteers (NHV)
- ClinicalTrials.gov ID
- NCT04535752
- Acronym / sponsor ID
- ANX009-NHV-01
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Annexon, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Nucleus Network Ltd
- Study start
- 30 Oct 2020 (actual)
- Primary completion
- 26 Jun 2021 (actual)
- Study completion
- 26 Jun 2021 (actual)
- First posted
- 2 Sep 2020 (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.
The purpose of this study is to evaluate the safety, tolerability, pharmakokinetics and pharmacodynamics of single and repeated doses of ANX009
Detailed description
In this first in human, phase 1, randomized, double-blind, placebo-controlled study, single and multiple ascending doses of ANX009 or placebo will be administered to 48 healthy subjects.
Single Ascending Dose (SAD): Each SAD subject will participate for approximately 4 weeks (3 nights in-clinic confinement).
Multiple Ascending Dose: Each MAD subject will participate for approximately 6 weeks (17 nights in-clinic confinement).
All subjects will be contacted (in clinic visit or phone call) 6 months after study completion.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| ANX009, Single Ascending Doses | Experimental | Drug: ANX009Single dose of ANX009 with a 7-day follow-up before escalation to the next dose level. |
| Placebo, Single Ascending Doses | Placebo comparator | Drug: PlaceboSingle doses of matching placebo |
| ANX009, Multiple Ascending Doses | Experimental | Drug: ANX009ANX009 once daily on Days 1-14 |
| Placebo, Multiple doses | Placebo comparator | Drug: PlaceboMatching placebo once daily on Days 1-14 |
Who may qualify
- Ages
- 18 to 59 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Healthy male and non-pregnant, non-lactating female volunteers ≥18 to 59 years of age.
- Females must be postmenopausal, surgically sterilized or willing and able to use highly effective methods of contraception from screening through the final study visit.
- Males with a partner of childbearing potential must agree to use contraception from Screening through the final study visit.
- Documented history within 5 years of screening of previous vaccination against encapsulated bacterial pathogens (MAD cohorts only).
- Complete the full sequence of protocol-related doses, procedures and evaluations.
Key exclusion criteria (18 listed)
- Clinically significant, ongoing illness or medical condition that would jeopardize the safety of the subject, limit participation, or compromise the interpretation of the safety data derived from the subject.
- Clinically significant findings on the screening or Baseline ECG or physical examination.
- Clinically significant abnormalities on screening or Baseline laboratory assessments.
- An ANA titer ≥ 1:160.
- History of any autoimmune disease.
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 |
|---|---|---|
| ANX009Also: 009Single or multiple ascending dose | Drug | ANX009, Multiple Ascending Doses, ANX009, Single Ascending Doses |
| PlaceboAlso: matching placeboSingle or multiple ascending dose | Drug | Placebo, Multiple doses, Placebo, Single Ascending Doses |
Primary outcome
Safety: Number of Participants Who Experienced Treatment-Emergent Adverse Events
Time frame: [Time Frame: Up to Day 29 for SAD; up to Day 43 for MAD]
Incidence and severity of treatment-emergent adverse events (AEs). AEs will be coded using MedDRA and severity of AEs will be graded using National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE).
6 secondary outcomes
Pharmacodynamics: Total Amount of Complement Protein in Blood (CH50)
Time frame: Up to Week 6
Pharmacodynamics: Amount of C1 in Blood (C1q)
Time frame: Up to Week 6
Pharmacokinetic: Maximum Observed Serum Concentration (Cmax) of ANX009
Time frame: Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)
Pharmacokinetic: Time to Maximum Observed Serum Concentration (Tmax) of ANX009
Time frame: Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)
Pharmacokinetic: Area Under the ANX009 Serum Concentration-Time Curve to Last Sample (AUC 0-t) and extrapolated through infinity (AUC 0-inf)
Time frame: Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)
Pharmacokinetic: Terminal Half-Life (t1/2) of ANX009
Time frame: Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)
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 30 Aug 2021.
- Individual participant dataSharing planned: No
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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.
Eric Humphriss, MBA study director
Annexon Director, Global Clinical Operations
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ANX009 in people with Safety and Tolerability in Healthy Volunteers, run by Annexon, Inc. at 1 site 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 9 inclusion and 18 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 30 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Safety: Number of Participants Who Experienced Treatment-Emergent Adverse Events (time frame: [Time Frame: Up to Day 29 for SAD; up to Day 43 for MAD]). 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 30 Aug 2021.