Single Dose Study of ANX005 in Healthy Volunteers

Phase 1

Terminated

• Conditions: Safety and Tolerability in Healthy Volunteers
• Interventions: Drug: ANX005; Drug: Placebos; Drug: IVIg

• Registration Number: NCT03010046
• Lead Sponsor: Annexon, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-12
• Study First Submitted: 2016-12-21
• Study First Submitted QC: 2017-01-03
• Study First Posted: 2017-01-04
• Primary Completion: 2018-06
• Study Completion: 2018-06
• Status Verified: 2020-08
• Last Update Submitted: 2020-08-19
• Last Update Posted: 2020-08-20

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 27

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

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ANX005 Monotherapy	ANX005	ANX005 intravenous infusion
ANX005 and IVIg Combination Therapy	ANX005	ANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg)
ANX005 and IVIg Combination Therapy	IVIg	ANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg)
Placebo	Placebos	Placebo intravenous infusion

Primary Outcome Measures

Name	Time	Method
Number of subjects with treatment-related adverse events as assessed by CTCAE v4.03	Day 43	Safety is assessed throughout the study. Day 43 is the last visit.

Secondary Outcome Measures

Name	Time	Method
Peak plasma concentration	Day 43
Determine effective dose of ANX005	Day 43	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
Area under the plasma concentration versus time curve (AUC)	Day 43
Terminal half-life	Day 43

Trial Locations

Locations (1)

Nucleus Network; 🇦🇺 Melbourne, Victoria, Australia