A Phase 2 interventional study of Thalidomide and Dexamethasone in Cardiac Amyloidosis, sponsored by Seoul National University Hospital. Status unknown at 1 site in Korea, Republic of. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2017-10-26.
Sponsored by Seoul National University Hospital · Phase 2, Interventional, and Treatment
To prove the organ-reversing potential of thalidomide for amyloidosis with cardiac involvement
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.
491 studies on the registry are indexed under Amyloidosis; 135 are open to participants now.
This study's planned enrollment of 30 is below the median of 40 across 304 interventional studies indexed under Amyloidosis.
Browse Amyloidosis studies →Seoul National University Hospital is the lead sponsor of 1,860 studies on the registry; 275 are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 2 (17%) have results posted.
Counted across the registry records on this site, refreshed daily.
Cardiac amyloidosis (both AL or AH type) patient who meet both (A and B) of the following criteria A. Cardiac involvement: meet one of the following criteria
Exclusion Criteria:
Patient with cardiac amyloidosis receive thalilomide with dexamethasone
Drug: Thalidomide · Drug: Dexamethasone
Hematologic response
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
Time frame: through study completion, an average of 1 year
Cardiac response
\> 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
Time frame: through study completion, an average of 1 year
Maximal LV myocardium-blood cavity ratio
estimated by 11C-Pittsburge B PET imaging
Time frame: through study completion, an average of 1 year
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
\> 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
Time frame: through study completion, an average of 1 year
Hepatic response
≥ 50% decrease in alkaline phosphatase levels and/or ≥ 2cm decrease in liver size (assessed by radiograph)
Time frame: through study completion, an average of 1 year
Mean LV myocardium-blood cavity ratio
estimated by 11C-Pittsburge B PET imaging
Time frame: through study completion, an average of 1 year
This study is status unknown, as verified in Oct 2017. You cannot join it, but the record below documents what was studied.
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Seoul National University Hospital