An observational, non-interventional study for the follow-up of patients with amyloidosis who received miridesap followed by dezamizumab in a phase 1 study

Table 1 Criteria used to guide response classification in this analysis

Criterion	Response during FIHS	Response during follow-up	Decline during follow-up
Amyloid load	Improvement* in SAP scan from baseline^† (pre-dose) at any study visit	Stable or improvement* in SAP scan compared with amyloid load from FIHS	Worsening in SAP scan compared with amyloid load from FIHS
6MWD	Not applicable	Stable or an increase ≥ 30 m [27] compared with the maximum 6MWD observed during the FIHS	Decrease ≥ 30 m compared with the maximum 6MWD observed during the FIHS
GGT (only in patients with abnormal GGT and/or hepatic amyloid during the FIHS)	Decrease (> 25%) in GGT from baseline^† (pre-dose) at any study visit	Stable or decrease (of any magnitude) compared with the final GGT level observed during the FIHS	Increase (of any magnitude) compared with the final GGT level observed during the FIHS
eGFR	No formal measure of response. Absolute values reviewed for safety and post-treatment response	Met criteria for an improvement in CTCAE grade [28] in kidney function at any point during follow-up	Met criteria for a decline in CTCAE grade in kidney function at any point during follow-up
NT-proBNP	No formal measure of response. Reviewed in respect of post-dose transient response in cardiac amyloid patients and in relation to eGFR in renal amyloid patients	Stable or decrease (> 30% reduction and > 300 ng/L [29]) compared with the last NT-proBNP level observed during the FIHS	Increase (> 30% and > 300 ng/L) compared with the last NT-proBNP level observed during the FIHS

*Improvement was based on subjective assessment of patient data
^†The baseline date was defined as the date of first pharmacologically active administration of dezamizumab (i.e. ≥ 200 mg) in the FIHS. For the one subject who received a non-pharmacologically active dose (i.e. < 200 mg), their baseline date was defined as the date of first administration of a non-pharmacologically active dose of dezamizumab
6MWD, 6-min walking distance; CTCAE, Common Terminology Criteria for Adverse Events; eGFR, estimated glomerular filtration rate; FIHS, first-in-human study; GGT, gamma-glutamyl transpeptidase; NT-proBNP, N-terminal-pro B-type natriuretic peptide; SAP, serum amyloid P

ISSN: 1750-1172