This is a multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who have a maintained hematologic response to their most recent treatment for AL amyloidosis (e.g., chemotherapy, autologous stem cell transplant \[ASCT\]) and have persistent renal dysfunction.
This is a multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who have a maintained hematologic response to their most recent treatment for AL amyloidosis (e.g., chemotherapy, autologous stem cell transplant \[SCT\]) and have persistent renal dysfunction. Subject screening will occur during the 28 days prior to the first administration of study drug (i.e. month 1 day 1). If screening assessments are completed and all eligibility requirements are met, the subject will be enrolled. Study visits will occur every 28 days based on scheduling from month 1 day 1. A ±5-day window is allowed for visits starting after month 1. Subjects may receive up to 12 infusions of study drug. Subjects who discontinue study drug before the initial End of Study (EOS) visit should have an Early Treatment Discontinuation (ETD) Visit 30 (±5) days after their final administration of study drug. After completing 12 months of treatment and the confirmatory EOS visit, a subject may enter an open-label extension (OLE) study, during which subjects will receive active treatment with NEOD001 for 12 months and may receive concurrent chemotherapy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
12
Mayo Clinic- Arizona
Scottsdale, Arizona, United States
University of California San Francisco
San Francisco, California, United States
Mayo Clinic- Florida
Jacksonville, Florida, United States
Confirmed Renal Response After Treatment With NEOD001
A renal response is a ≥ 30% reduction in proteinuria in the absence of a ≥ 25% decrease in eGFR. A confirmed renal response is one that has been documented as present one month after 12 monthly treatments.
Time frame: Baseline to 13 Months
Measured GFR at Study Entry
The aim of this study is to assess the performance of CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) creatinine in comparison to iothalamate clearance measured in AL amyloidosis patients. Iothalamate will be given subcutaneously. Urine and plasma samples will then be obtained. All laboratory tests for samples obtained for GFR measurement will be performed at Mayo Clinic Rochester. Quantification of iothalamate in urine and plasma will be performed using a tandem mass spectrometric method.
Time frame: Baseline
Time to CKD 4 or 5
Months to Chronic Kidney Disease level 4 or 5
Time frame: Baseline to 13 Months
Time to eGFR ≤ 15 or Dialysis
Months to estimated Glomerular Filtration Rate ≤ 15 or dialysis
Time frame: Baseline to 13 Months
Time to Doubling of Creatinine
Months to doubling of serum creatinine
Time frame: Baseline to 13 Months
Time to ≥ 40% Reduction in eGFR
Months to ≥ 40% reduction in estimated glomerular filtration rate
Time frame: Baseline to 13 Months
Renal Response in Patients With Maintained Hematologic Responses After 24 Monthly Treatments.
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Tufts Medical Center
Boston, Massachusetts, United States
Karmanos Cancer Institute
Detroit, Michigan, United States
Mayo Clinic- Minnesota
Rochester, Minnesota, United States
Memorial Sloan-Kettering Cancer Center
New York, New York, United States
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
A renal response is a ≥ 30% decrease in proteinuria or drop of proteinuria below 0.5 g/24h in the absence of renal progression.
Time frame: Baseline to 26 months
All Cause of Mortality at 26 Months
Death at 26 months from Baseline due to any cause
Time frame: Baseline to 26 months