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Renal AL amyloid Involvement and NEOD001 (RAIN)
||A Multicenter Randomized Double-blind Phase 2b Study of NEOD001 in Previously Treated Subjects with Systemic Light-chain (AL) Amyloidosis and Persistent Renal Involvement
||Cindy Varga, MD
The purpose of this study is to evaluate whether NEOD001 is safe and effective specifically in subjects with light chain (AL) amyloidosis affecting the kidneys. This study will also evaluate whether NEOD001 improves the function of subjects’ organs that have been affected by amyloid deposits.
- Biopsy-proven diagnosis of AL amyloidosis by immunohistochemistry or mass spectroscopy of a tissue biopsy excluding bone marrow
- Screening renal biopsy for RAIN confirming AL amyloidosis as exclusive or dominant cause of renal damage
- Persistent renal involvement from diagnosis with proteinuria (predominantly albumin) > 500mg/day in a 24-hour urine collection
- Amyloidosis due to mutations of the transthyretin gene or presence of other non-AL amyloidosis
- Female patients who are lactating, breastfeeding, or pregnant
- Patients who have not been treated or who have received chemotherapy within 6 months, or SCT within 12 months, for the light-chain producing hematologic disease causing AL amyloidosis, at the time of the first dose of NEOD001 (month 1 day 1)
Study participation will be up to approximately 26 months long. Participants who qualify for this study will be assigned, by chance, to receive either NEOD001 or placebo. Participants will receive study drug (NEOD001 or placebo) about every 28 days for up to 12 months (12 total infusions, 1 per month). The study also involved a kidney biopsy, physical exams, ECGs, collection of blood and urine as well as other study procedures as required by protocol.