Clinical Trial: Thalidomide/Dexamethasone Treatment And PET Evaluation In Organ Involvemenet of Cardiac Amyloidosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Frontline Thalidomide for Amyloidosis Involving Myocardium: Investigation of Organ Reversing Capacity of Lenalidomide

Brief Summary: To prove the organ-reversing potential of thalidomide for amyloidosis with cardiac involvement

Detailed Summary:

Considering that dismal prognosis of amyloidosis is attributable to organ dysfunction, primary aim of amyloidosis treatment should be an organ reversal. However, due to various reasons, not much is known about organ reversal in amyloidosis. Almost all of the clinical trials evaluated hematologic response in amyloidosis. Meanwhile, besides autologous stem cell transplantation with high-dose melphalan conditioning, hematologic response rate of various agents such as bortezomib, melphalan, thalidomide and lenalidomide are similar for amyloidosis. However, organ reversing potential of these agents is not known. If there is a difference in organ reversing potential despite of similar hematologic response rate, drug with effective organ reversing potential should be a standard treatment for amyloidosis.

The investigators assume that thalidomide could make organ reversal in cardiac amyloidosis due to its specific mechanism of action. To prove this concept, the investigators propose a clinical trial that evaluates organ reversing potential of thalidomide in cardiac amyloidosis.


Sponsor: Seoul National University Hospital

Current Primary Outcome: Hematologic response [ Time Frame: through study completion, an average of 1 year ]

Complete response: Normalization of FLC levels and κ to λ ratio, with nega-tive serum and urine immunofixation Very good partial response: de-creased of dFLC to < 40mg/l Partial response: > 50% reduction of dFLC


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Cardiac response [ Time Frame: through study completion, an average of 1 year ]
    > 30% and > 300 ng/l decrease in NTproBNP levels in patients with NTproBNP levels ≥ 650 ng/l at base-line or ≥ 2-class decrease in NYHA class in patients with NYHA class 3 or 4 at baseline
  • Maximal LV myocardium-blood cavity ratio [ Time Frame: through study completion, an average of 1 year ]
    estimated by 11C-Pittsburge B PET imaging
  • Overall survival [ Time Frame: From date of enrollment until the date of death from any cause, assessed up to 60 months ]
  • Progression-free survival [ Time Frame: From date of enrollment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 60 months ]
  • Toxicity profile related to thalidomide, according to CTCAE version 4.03 [ Time Frame: through study completion, an average of 1 year ]
  • Renal response [ Time Frame: through study completion, an average of 1 year ]
    > 50% (≥ 5.0 g/d) decrease in 24h urine protein levels in patients with urine protein levels > 0.5 g/l at baseline without ≥ 25% increase in serum creatinine levels or decrease in creatinine clearance from baseline
  • Hepatic response [ Time Frame: through study completion, an average of 1 year ]
    ≥ 50% decrease in alkaline phosphatase levels and/or ≥ 2cm decrease in liver size (assessed by radiograph)
  • Mean LV myocardium-blood cavity ratio [ Time Frame: through study completion, an average of 1 year ]
    estimated by 11C-Pittsburge B PET imaging


Original Secondary Outcome: Same as current

Information By: Seoul National University Hospital

Dates:
Date Received: November 8, 2016
Date Started: October 2016
Date Completion:
Last Updated: November 16, 2016
Last Verified: November 2016