Systemic Amyloidosis
Conditions
Brief summary
This is an open-label, multicenter, Phase 2 study in subjects with newly diagnosed or previous treated systemic light chain (AL) amyloidosis. Approximately 40 subjects will receive therapy with bortezomib, pomalidomide, and dexamethasone. The primary outcome is hematologic very good partial response and complete response rate at 6 months.
Detailed description
Proteasome inhibitor and immunmodulators have synergistic effect for plasma cell dyscrasia. Due to the fact that more than 70-80% of patients with amyloidosis have renal involvement, the application of lenalidomide is limited. Thus, the investigators designed this open-label, multicenter, phase 2 study for newly diagnosed or previously treated systemic AL amyloidosis with bortezomib, pomalidomide and dexamethasone regimen.
Interventions
DRUGBortezomib
Bortezomib 1.3mg/m2 sc weekly
DRUGPomalidomide
4mg per day taken orally on 1-21 of repeated 28-day cycles
DRUGDexamethasone
Dexamethasone 20mg-40mg weekly
Sponsors
Jin Lu, MD
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Bortezomib, pomalidomide, dexamethasone
Eligibility
Sex/Gender
ALL
Age
18 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
1. Diagnosis of AL amyloidosis, confirmed by histology and typed with immunohistochemistry, immunoelectron microscopy or mass spectrometry. 2. Newly diagnosed or previous treated AL amyloidosis 3. Patients must be ≥ 18 years of age. 4. ECOG performance status 0, 1 or 2. 5. Measurable disease defined by at least one of the following: ① serum free light chain (FLC) ≥2.0 mg/dL (20 mg/L) with an abnormal kappa:lambda ratio or the difference between involved and uninvolved free light chains (dFLC) ≥2mg/dL (20 mg/L). ②. presence of a monoclonal spike that is ≥5 g/l. 6. Symptomatic organ involvement (heart, kidney, liver/GI tract, peripheral nervous system). 7. Absolute neutrophil count (ANC) ≥1.0 X 10\^9/L, Hemoglobin ≥70 g/L, Platelets ≥50 X 10\^9/L 8. eGFR ≥20 mL/min/ 1.73 m\^2 9. Written informed consent in accordance with local and institutional guidelines. 10. Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Exclusion criteria
1. Fulfill with the criteria of active multiple myeloma or active lymphoplasmacytic lymphoma. 2. Presence of other tumors which is/are in advanced malignant stage and has/have systemic metastasis; 3. Severe or persistent infection that cannot be effectively controlled; 4. Presence of severe autoimmune diseases or immunodeficiency disease; 5. Patients with active hepatitis B or hepatitis C (\[HBVDNA+\] or \[HCVRNA+\]); 6. Patients with HIV infection or syphilis infection; 7. Any situations that the researchers believe will increase the risks for the subject or affect the results of the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hematologic VGPR + CR rate at 6 months | 6 months | Hematologic very good partial response plus complete response rate at 6 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| At least one organ response at 6 months | 6 months | At least one organ response (cardiac response, renal response, liver response) at 6 months |
| TTNT at 2 years | 2 years | Time to next treatment at 2 years |
| Overall Hematologic response rate at 6 months | 6 months | Overall Hematologic partial response, very good partial response rate and complete response at 6 months |
| Estimated OS at 2 years | 2 years | Estimated Overall Survival at 2 years |
| TRAE | 6 months | Treatment-related adverse events up to 6 months |
| Estimated PFS at 2 years | 2 years | Estimated Progression free survival at 2 years |
Countries
China
Contacts
Primary ContactYang Liu, Dr
Outcome results
None listed