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Venetoclax is considered as a promising agent for light-chain (AL) amyloidosis due to the high percentage of t(11;14). Several retrospective studies showed venetoclax-based therapy could induce rapid and profound hematologic response in AL patients with favorable safety profile. As an oral agent with encouraging data, it is worth to prospectively evaluate the efficacy and safety of venetoclax in untreated AL amyloidosis patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ven-D | Experimental | Venetoclax combined with dexamethasone |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Venetoclax | Drug | Venetoclax 400mg po qd for 1 year |
| |
| Dexamethasone Oral |
| Measure | Description | Time Frame |
|---|---|---|
| Complete response (CR)+very good partial response (VGPR) at 3 months after treatment initiation | 3 months after treatment initiation |
| Measure | Description | Time Frame |
|---|---|---|
| Overall survival | 2 years | |
| Time to next treatment | 2 years | |
| CR+VGPR at 1 month after treatment initiation |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Beijing | China |
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| ID | Term |
|---|---|
| D000686 | Amyloidosis |
| ID | Term |
|---|---|
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C579720 | venetoclax |
| D003907 | Dexamethasone |
| ID | Term |
|---|---|
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
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| Drug |
Dexamethasone 40mg po qw for the first 6 months, then 10mg po qw for the next 6 months |
|
| 1 month after treatment initiation |
| CR+VGPR at 6 months after treatment initiation | 6 months after treatment initiation |
| CR+VGPR at 12 months after treatment initiation | 12 months after treatment initiation |
| Difference between involved and uninvolved free light chain (dFLC) < 10mg/L | at 1, 3, 6 and 12 months after treatment initiation |
| Involved free light chain (iFLC) ≤ 20mg/L | at 1, 3, 6 and 12 months after treatment initiation |
| Minimal residual disease (MRD) negativity | 12 and 24 months after treatment initiation |
| Time to hematologic response | 1 year |
| Time to hematologic CR | 1 year |
| Cardiac response | at 3, 6, 12 and 24 months after treatment initiation |
| Renal response | at 3, 6, 12 and 24 months after treatment initiation |
| Hepatic response | at 3, 6, 12 and 24 months after treatment initiation |
| Time to cardiac response | 2 years |
| Time to renal response | 2 years |
| Time to hepatic response | 2 years |
| Adverse events | treatment initiation to 30 days after last dose of treatment |
| D000072473 |
| Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |