Summary
Systemic amyloidosis is an incurable disease, and about 20% of patients with cardiac or advanced kidney involvement experience early deaths (<1 year). Despite recent progress in proteasome inhibitors, chemotherapies, and immunotherapies that target plasma cells have greatly improved the prognosis of patients with systemic amyloidosis, median survival remains low at approximately five years.
AT-02 (INN: not yet available) is a full-length, humanized, recombinant immunoglobulin 1 (IgG1)-like glycoprotein monoclonal antibody (mAb) that is being developed to treat systemic amyloidosis.
This study has three parts.
Part 1 is a double-blind, single-center, single-ascending dose evaluation study in healthy volunteers to assess AT-02. Healthy volunteers between 18-56 years will be enrolled in the Part 1 study.
Part 2 is an open-label, single ascending dose escalation study in people with systemic amyloidosis to assess AT-02 and to identify a maximum tolerated dose (MTD). People with systemic amyloidosis over 18 years will be involved in Part 2 of the study.
Part 3 is an open-label, multiple-ascending dose, dose escalation study in people with systemic amyloidosis to assess multiple doses of AT-02. People with systemic amyloidosis aged ≥18 and ≤85 years of age will be involved in the Part 3 study.