Suggestions for clinical trial design in AL amyloidosis
Key considerations in trial design:
Uniform implementation of measurable disease criteria in trials of relapsed/refractory AL amyloidosis
Preference for time-limited therapy to reduce physical and financial toxicity
Response-driven de-escalation strategies
Inclusion of patients with advanced disease (eg, NT-proBNP >8500 pg/mL, ESRD, and autonomic neuropathy)
Incorporate correlative studies on novel assays for measuring tumor burden (eg, free light chain-mass spectrometry)
Incorporate patient-reported outcome assessment to measure HR-QoL
Functional assessment (6MWT) with clinically meaningful differences
Key areas of unmet need:
Standardization of supportive care regimens
Rare entities such as immunoglobulin M amyloidosis
Treatment and natural history studies of localized AL amyloidosis
Determine the most appropriate imaging modalities for assessing disease burden and response, considering factors such as sensitivity, specificity, and feasibility
Therapies targeting amyloid fibrils and misfolded light chains
Key considerations in trial design:
Uniform implementation of measurable disease criteria in trials of relapsed/refractory AL amyloidosis
Preference for time-limited therapy to reduce physical and financial toxicity
Response-driven de-escalation strategies
Inclusion of patients with advanced disease (eg, NT-proBNP >8500 pg/mL, ESRD, and autonomic neuropathy)
Incorporate correlative studies on novel assays for measuring tumor burden (eg, free light chain-mass spectrometry)
Incorporate patient-reported outcome assessment to measure HR-QoL
Functional assessment (6MWT) with clinically meaningful differences
Key areas of unmet need:
Standardization of supportive care regimens
Rare entities such as immunoglobulin M amyloidosis
Treatment and natural history studies of localized AL amyloidosis
Determine the most appropriate imaging modalities for assessing disease burden and response, considering factors such as sensitivity, specificity, and feasibility
Therapies targeting amyloid fibrils and misfolded light chains
6MWT, 6-minute walk test; ESRD, end-stage renal disease.