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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03010046




Registration number
NCT03010046
Ethics application status
Date submitted
21/12/2016
Date registered
4/01/2017
Date last updated
20/08/2020

Titles & IDs
Public title
Single Dose Study of ANX005 in Healthy Volunteers
Scientific 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
Secondary ID [1] 0 0
ANX005-CP01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Safety and Tolerability in Healthy Volunteers 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ANX005
Treatment: Drugs - IVIg
Treatment: Drugs - Placebos

Experimental: ANX005 Monotherapy - ANX005 intravenous infusion

Experimental: ANX005 and IVIg Combination Therapy - ANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg)

Placebo Comparator: Placebo - Placebo intravenous infusion


Treatment: Drugs: ANX005
Single ascending dose intravenous infusion

Treatment: Drugs: IVIg
IVIg infusion in Cohorts 4b and 5b only. Randomized to ANX005 followed by IVIg or placebo followed by IVIg.

Treatment: Drugs: Placebos
0.9% saline intravenous infusion

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
Day 43
Secondary outcome [1] 0 0
Peak plasma concentration
Timepoint [1] 0 0
Day 43
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Day 43
Secondary outcome [3] 0 0
Area under the plasma concentration versus time curve (AUC)
Timepoint [3] 0 0
Day 43
Secondary outcome [4] 0 0
Terminal half-life
Timepoint [4] 0 0
Day 43

Eligibility
Key inclusion criteria
- 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
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
- 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Nucleus Network - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Annexon, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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. 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.
Trial website
https://clinicaltrials.gov/show/NCT03010046
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Sandy Calman, MD
Address 0 0
Annexon Medical Monitor
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03010046