AL amyloidosis MedDRA version: 9.1 Level: HLGT Classification code 10035227
Conditions
Interventions
Trade Name: VELCADE
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Bortezomib
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration numbe
Sponsors
E.M.N. - EUROPEAN MYELOMA NETWORK
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Histologic diagnosis of amyloidosis. 2. Genetic testing must be negative for transthyretin mutations associated with hereditary amyloidosis or immunohistochemistry of amyloid deposits must provide clear evidence of K or ? light chains in those who present with peripheral neuropathy or heart as the dominant organ involvement. 3. Not eligible for ASCT with melphalan 200 mg/m2 . Patients who are eligible for SCT with melphalan 200 mg/m2 but decline the procedure, can be enrolled in the study, but are stratified in a separate stratum before randomization. 4. Patients must be = 18 years of age. 5. ECOG performance status 0,1 or 2. 6. Measurable disease; al least one of the following criteria: • monoclonal protein >10 g/L in serum • amyloid-forming (involved) FLC >75 mg/L with an abnormal K/? ratio • difference between involved and uninvolved FLC >50 mg/L • bone marrow with a clonal predominance 7. Symptomatic organ involvement 8. Hemoglobin =11 g/dL, absolute neutrophil count =1500/mcL, platelets =140,000/mcL. 9. Total bilirubin 30 ml/min. 11. Only patients who are informed of the investigational nature of this study and sign and give written informed consent in accordance with institutional, national and European guidelines are eligible to participate. 12. Women must be either post-menopausal or surgically sterilized or willing to use an acceptable method of birth control 13. Men must agree to use an acceptable method for contraception for the duration of the study. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Amyloid-specific syndrome 2. Isolated soft tissue involvement. 3. Presence of non-AL amyloidosis. 4. Previous treatment for plasma cell disease. 5. Bone marrow plasma cells >30%. 6. Cardiac stage III disease: both cTnT > 0.035 ng/mL (or in place of cTnT the cTnI > 0.10 ng/mL) and simultaneous NT-proBNP >332 ng/L. 7. Repetitive ventricular arrhythmias on 24h Holter ECG in spite of anti-arrhythmic treatment or chronic atrial fibrillation 8. Supine systolic blood pressure 51mL/min, left ventricular ejection fraction >45%, and bilirubin <2.0 mg/dL. 11. Pregnant or nursing women. 12. Clinically overt multiple myeloma with lytic bone lesions 13. Patients with uncontrolled infection or active malignancy with the exception of adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease-free for 5 years. 14. Patients with medically documented cardiac syncope, uncompensated NYHA Class 3 or 4 congestive heart failure, or myocardial infarction within the previous 6 months are not eligible. 15. HIV positive. 16. Patients with serious medical or psychiatric illness likely to interfere with participation in this clinical study. 17. Patients with hypersensitivity to bortezomib, boron or mannitol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: 1. Complete hematologic response rate after 3 cycles and after completion of therapy; 2. Hematologic response rate at completion of therapy; 3. Organ response rates at 3, 6, 9 and 12 months; 4. Treatment-related mortality; 5. Toxicity; 6. Overall and progression-free survival; 7. Time to hematologic and organ response; 8. Quality of life.;Primary end point(s): Hematologic response after 3 cycles of therapy.;Main Objective: To compare the hematologic response after 3 cycles of therapy. | — |
Countries
Czech Republic, Denmark, Germany, Greece, Italy, Spain, Sweden, United Kingdom
Outcome results
None listed