Safety and Tolerability in Healthy VolunteersPhase 1NCT03010046
Single Dose Study of ANX005 in Healthy Volunteers
A Phase 1 study testing ANX005 and IVIg in people with Safety and Tolerability in Healthy Volunteers, run by Annexon, Inc. at 1 site in Australia.
Trial summary
This is a single-center, randomized, double-blind, placebo-controlled, ascending, single-infusion, sequential group study. Single, ascending doses will be administered to approximately 64 subjects, with an option for 1 additional multi-dose cohort in approximately 8 subjects. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Safety and Tolerability in Healthy Volunteers
- What is being tested?
- ANX005 and IVIg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 1 siteAustralia
- Study dates
- Dec 2016to Jun 2018
- 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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Male and females 18 years and older
- Females must be postmenopausal, surgically sterilized, or willing and able to use 2 methods of contraception throughout the study and for 1 month after the final study visit
- Willing and able to undergo vaccination if not vaccinated recently
Who cannot join
- History of any autoimmune disease, meningitis, septicemia or pneumonia
- History of hypercoagulable diseases, hyperviscosity, thrombosis, renal dysfunction or acute renal failure
- Known genetic deficiencies of the complement cascade system
- History of conditions whose symptoms and effects could alter protein catabolism or IgG utilization, e.g. protein-losing enteropathies or nephrotic syndrome
- Body weight less than 50 kg or greater than 100 kg
- Hypersensitivity or allergic reactions to any excipients in the ANX005 drug product
- (Cohorts 4b and 5b) Known selective IgA deficiency or presence of antibodies to IgA at screening
- (Cohorts 4b and 5b) Prior reaction or hypersensitivity to blood products, including IVIg or any of the excipients in IVIg.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Placebo-controlled, Double Blind, Single, Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ANX005 Monotherapy and ANX005 in Combination With IVIg in Healthy Volunteers
This is a single-center, randomized, double-blind, placebo-controlled, ascending, single-infusion, sequential group study. Single, ascending doses will be administered to approximately 64 subjects, with an option for 1 additional multi-dose cohort in approximately 8 subjects. The primary objective is to evaluate the safety of ANX005 administered as an intravenous infusion as a single agent and in combination with intravenous immunoglobulin (IVIg). The optional multi-dose cohort will evaluate either additional subjects at the maximum tolerated dose or ANX005 administered as 2 infusions.
- Being tested
- ANX005, IVIg, Placebos
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANX005Drug
Single ascending dose intravenous infusion
IVIgDrug
IVIg infusion in Cohorts 4b and 5b only. Randomized to ANX005 followed by IVIg or placebo followed by IVIg.
PlacebosDrug
0.9% saline intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| ANX005 Monotherapy | Experimental | ANX005 intravenous infusion Receives: Drug: ANX005 |
| ANX005 and IVIg Combination Therapy | Experimental | ANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg) Receives: Drug: ANX005; Drug: IVIg |
| Placebo | Placebo comparator | Placebo intravenous infusion Receives: Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with treatment-related adverse events as assessed by CTCAE v4.03
Safety is assessed throughout the study. Day 43 is the last visit.
Measured: Day 43
Secondary outcomes (4)
Peak plasma concentration
Measured: Day 43
Determine effective dose of ANX005
Percent of subjects with a biologic response, defined as a reduction of serum CH50 percent change from baseline at 21 and 28 days after the first ANX005 infusion
Measured: Day 43
Area under the plasma concentration versus time curve (AUC)
Measured: Day 43
Terminal half-life
Measured: Day 43
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jan 2017 (estimated)
- Study startDec 2016
- Primary completionJun 2018
- Study completionJun 2018
Why it stopped: ANX005 well tolerated at doses studied; study discontinued and initiated in patient population
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Annexon, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sandy Calman, MD, Study director, Annexon Medical MonitorJason Lickliter, MBBS PhD, Principal investigator, Nucleus Network Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03010046
- Sponsor's protocol ID
- ANX005-CP01
- Status last verified
- Aug 2020
- First submitted
- 21 Dec 2016
- Last updated
- 20 Aug 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Lansita JA, Mease KM, Qiu H, Yednock T, Sankaranarayanan S, Kramer S. Nonclinical Development of ANX005: A Humanized Anti-C1q Antibody for Treatment of Autoimmune and Neurodegenerative Diseases. Int J Toxicol. 2017 Nov/Dec;36(6):449-462. doi: 10.1177/1091581817740873. Epub 2017 Dec 4.
PubMed 29202623
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Placebo-controlled, Double Blind, Single, Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ANX005 Monotherapy and ANX005 in Combination With IVIg in Healthy Volunteers
- ClinicalTrials.gov ID
- NCT03010046
- Acronym / sponsor ID
- ANX005-CP01
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: ANX005 well tolerated at doses studied; study discontinued and initiated in patient population
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Annexon, Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2016
- Primary completion
- Jun 2018 (actual)
- Study completion
- Jun 2018 (actual)
- First posted
- 4 Jan 2017 (estimated)
- 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 single-center, randomized, double-blind, placebo-controlled, ascending, single-infusion, sequential group study. Single, ascending doses will be administered to approximately 64 subjects, with an option for 1 additional multi-dose cohort in approximately 8 subjects. The primary objective is to evaluate the safety of ANX005 administered as an intravenous infusion as a single agent and in combination with intravenous immunoglobulin (IVIg). The optional multi-dose cohort will evaluate either additional subjects at the maximum tolerated dose or ANX005 administered as 2 infusions.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ANX005 Monotherapy | Experimental | Drug: ANX005ANX005 intravenous infusion |
| ANX005 and IVIg Combination Therapy | Experimental | Drug: ANX005, Drug: IVIgANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg) |
| Placebo | Placebo comparator | Drug: PlacebosPlacebo intravenous infusion |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male and females 18 years and older
- Females must be postmenopausal, surgically sterilized, or willing and able to use 2 methods of contraception throughout the study and for 1 month after the final study visit
- Willing and able to undergo vaccination if not vaccinated recently
Key exclusion criteria (8 listed)
- History of any autoimmune disease, meningitis, septicemia or pneumonia
- History of hypercoagulable diseases, hyperviscosity, thrombosis, renal dysfunction or acute renal failure
- Known genetic deficiencies of the complement cascade system
- History of conditions whose symptoms and effects could alter protein catabolism or IgG utilization, e.g. protein-losing enteropathies or nephrotic syndrome
- Body weight less than 50 kg or greater than 100 kg
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 |
|---|---|---|
| ANX005Single ascending dose intravenous infusion | Drug | ANX005 Monotherapy, ANX005 and IVIg Combination Therapy |
| IVIgIVIg infusion in Cohorts 4b and 5b only. Randomized to ANX005 followed by IVIg or placebo followed by IVIg. | Drug | ANX005 and IVIg Combination Therapy |
| Placebos0.9% saline intravenous infusion | Drug | Placebo |
Primary outcome
Number of subjects with treatment-related adverse events as assessed by CTCAE v4.03
Time frame: Day 43
Safety is assessed throughout the study. Day 43 is the last visit.
4 secondary outcomes
Peak plasma concentration
Time frame: Day 43
Determine effective dose of ANX005
Time frame: Day 43
Area under the plasma concentration versus time curve (AUC)
Time frame: Day 43
Terminal half-life
Time frame: Day 43
Results and safety
The study was stopped early (reason given: ANX005 well tolerated at doses studied; study discontinued and initiated in patient population). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2020.
- Derived from this study: PubMed 29202623Lansita JA, Mease KM, Qiu H, Yednock T, Sankaranarayanan S, Kramer S. Nonclinical Development of ANX005: A Humanized Anti-C1q Antibody for Treatment of Autoimmune and Neurodegenerative Diseases. Int J Toxicol. 2017 Nov/Dec;36(6):449-462. doi: 10.1177/1091581817740873. Epub 2017 Dec 4.
- 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.
Sandy Calman, MD study director
Annexon Medical Monitor
Jason Lickliter, MBBS PhD principal investigator
Nucleus Network Ltd
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 ANX005 and IVIg 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 3 inclusion and 8 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: stopped early, as of the sponsor's last update on 20 Aug 2020. 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 was stopped early (reason given: ANX005 well tolerated at doses studied; study discontinued and initiated in patient population). Results are not available in the source record.
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?
Number of subjects with treatment-related adverse events as assessed by CTCAE v4.03 (time frame: Day 43). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03010046, which also lists 1 linked publication.
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 20 Aug 2020.