Clinical Trial: Renal AL Amyloid Involvement and NEOD001

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: The RAIN Study, a Multicenter Randomized Double-blind Phase 2b Study of NEOD001 in Previously Treated Subjects With Systemic Light-chain (AL) Amyloidosis and Persistent Re

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: Tufts Medical Center

Current Primary Outcome: Confirmed renal response after treatment with NEOD001 [ Time Frame: Baseline to 13 Months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Measured GFR at study entry [ Time Frame: Baseline ]
  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 to CKD 4 or 5 [ Time Frame: Baseline to 13 Months ]
  Months to Chronic Kidney Disease level 4 or 5
• Time to eGFR ≤ 15 or dialysis [ Time Frame: Baseline to 13 Months ]
  Months to estimated Glomerular Filtration Rate ≤ 15 or dialysis
• Time to doubling of Creatinine [ Time Frame: Baseline to 13 Months ]
  Months to doubling of serum creatinine
• Time to ≥ 40% reduction in eGFR [ Time Frame: Baseline to 13 Months ]
  Months to ≥ 40% reduction in estimated glomerular filtration rate
• Renal response in patients with maintained hematologic responses at 26 months [ Time Frame: Baseline to 26 months ]
  A renal response is a ≥ 30% decrease in proteinuria or drop of proteinuria below 0.5 g/24h in the absence of renal progression.
• All cause of mortality at 26 months [ Time Frame: Baseline to 26 months ]
  Death at 26 months from Baseline due to any cause

Original Secondary Outcome: Same as current

Information By: Tufts Medical Center

Dates:
Date Received: April 26, 2017
Date Started: June 2017
Date Completion: June 2019
Last Updated: May 24, 2017
Last Verified: May 2017