Single Dose Study of ANX005 in Healthy Volunteers

NCT03010046 | Terminated Phase 1

• 1 other identifier: ANX005-CP01
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

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.

Conditions

Safety and Tolerability in Healthy Volunteers

Outcome Measures

Primary Outcomes (1)

• 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.

  Day 43

Secondary Outcomes (4)

• Peak plasma concentration

  Day 43

• Determine effective dose of ANX005

  Day 43

• Area under the plasma concentration versus time curve (AUC)

  Day 43

• Terminal half-life

  Day 43

Other Outcomes (4)

• Explore relationship of AUC with PD responses in serum

  Day 43

• Explore relationship of AUC with PD responses in CSF

  Day 43

• Explore relationship of half-life with PD responses in serum

  Day 43

Study Arms (3)

ANX005 Monotherapy

EXPERIMENTAL

ANX005 intravenous infusion

Drug: ANX005

ANX005 and IVIg Combination Therapy

EXPERIMENTAL

ANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg)

Drug: ANX005; Drug: IVIg

Placebo

PLACEBO COMPARATOR

Placebo intravenous infusion

Drug: Placebos

Interventions

ANX005 DRUG

Single ascending dose intravenous infusion

ANX005 Monotherapy; ANX005 and IVIg Combination Therapy

IVIg DRUG

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

ANX005 and IVIg Combination Therapy

Placebos DRUG

0.9% saline intravenous infusion

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Annexon, Inc.: lead

Study Sites (1)

Nucleus Network

Melbourne, Victoria, Australia

Location

Related Publications (1)

• 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.

  PMID: 29202623 DERIVED

MeSH Terms

Interventions

Immunoglobulins, Intravenous

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Sandy Calman, MD

  Annexon Medical Monitor

  STUDY DIRECTOR

• Jason Lickliter, MBBS PhD

  Nucleus Network Ltd

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2016
• First Posted: January 4, 2017
• Study Start: December 1, 2016
• Primary Completion: June 1, 2018
• Study Completion: June 1, 2018
• Last Updated: August 20, 2020

Record last verified: 2020-08

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)