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
Overview
- Phase
- Phase 1
- Intervention
- Placebos
- Conditions
- Safety and Tolerability in Healthy Volunteers
- Sponsor
- Annexon, Inc.
- Enrollment
- 27
- Locations
- 1
- Primary Endpoint
- Number of subjects with treatment-related adverse events as assessed by CTCAE v4.03
- Status
- Terminated
- Last Updated
- 5 years ago
Overview
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.
Investigators
Eligibility Criteria
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
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.
Arms & Interventions
Placebo
Placebo intravenous infusion
Intervention: Placebos
ANX005 Monotherapy
ANX005 intravenous infusion
Intervention: ANX005
ANX005 and IVIg Combination Therapy
ANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg)
Intervention: ANX005
ANX005 and IVIg Combination Therapy
ANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg)
Intervention: IVIg
Outcomes
Primary Outcomes
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.
Secondary Outcomes
- 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)