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Phase I/II study to evaluate the efficacy and safety of the combination treatment of melpharan, dexamethasone and bortezomib for relapsed or refractory systemic AL amyloidosis.

Phase 1
Conditions
To evaluate the efficacy and safety of the combination treatment melpharan, dexamethasone and bortezomib (BMD treatment) for relapsed or refractory systemic AL amyloidosis.
Registration Number
JPRN-UMIN000006604
Lead Sponsor
Japan Community Health care Organization Kyoto kuramaguchi Medical Center, Department of Hematology
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Complete: follow-up complete
Sex
All
Target Recruitment
21
Inclusion Criteria

Not provided

Exclusion Criteria

1)Untreated patients 2)History of botezomib exposure 3)Poor-risk patients (Skinner et al.Ann Intern Med140:85,2004) i)Decompensated heart failure (NYHA>=3) ii)Ejection fraction < 0.40 iii)Persistent pleural effusion iv)Systolic blood pressure < 90 mmHg v)Oxygen saturation < 95%,room air vi)Performance Status >= 3 4)Neurological disorders (peripheral neuropathy, orthostatic hypotension, or paralytic ileus) excluding carpal tunnel syndrome 5)Gastrointestinal symptoms 6)NT-proBNP >= 332 pg/mL (BNP >=50 pg/mL) 7)A case with pulmonary complication (interstitial pneumonia, lung fibrosis, lung amyloidosis, etc.): Check abnormalities by evaluation by CT, utilize KL-6, SP-D, and SP-A laboratory data auxiliary, and judge synthetically. 8)Subject was pregnant or potential

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Hematological response rate post 6 months treatment
Secondary Outcome Measures
NameTimeMethod
Maximum tolerant dose, Organ response rate post 6 months treatment, Plasma free light chain level change from baseline, Average progression free survival, Average overall survival, Adverse event rate
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