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.

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-UMIN000006604
• Date of registration: 24/10/2011
• Prospective Registration: Yes
• Primary sponsor: Japan Community Health care Organization Kyoto kuramaguchi Medical Center, Department of Hematology
• Public title: 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.
• Scientific title: 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. - BMD treatment for relapsed or refractory systemic AL amyloidosis.
• Date of first enrolment: 2011/11/01
• Target sample size: 21
• Recruitment status: Complete: follow-up complete
• URL: https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000007812
• Study type: Interventional
• Study design: Single arm Non-randomized
• Phase: Phase I,II

Countries of recruitment

Japan

Contacts

• Name: Chihiro Shimazaki
• Affiliation: Japan Community Health care Organization Kyoyo kuramaguchi Medical Center Department of Hematology

• Name: Yukio Ando
• Affiliation: Kumamoto University Faculty of life science, Department of Diagnostic Medicine

Key inclusion & exclusion criteria

Inclusion criteria:
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

Age minimum: 20years-old
Age maximum: 65years-old
Gender: Male and Female

Health Condition(s) or Problem(s) studied

To evaluate the efficacy and safety of the combination treatment melpharan, dexamethasone and bortezomib (BMD treatment) for relapsed or refractory systemic AL amyloidosis.

Intervention(s)

Patients receive oral melphalan 8 mg/m2 on days 1-4, bortezomib SC (IV) on days 1, 4, 8 and 11, and dexamethasone orally on days 1-2, 4-5 8-9 11 and 12. Treatment repeats every 4 weeks (28 days) for up to 4 cycles in the absence of disease progression or unacceptable toxicity. *The preventive medication on last medication day after the 28th is recommended in principle from acyclovir 200 or 400 mg/the bortezomib medication opening day of a day. Moreover, when the symptoms of herpes are shown, the acyclovir or the rose cyclo building of a therapeutic amount is promptly prescribed for the patient.

Primary Outcome(s)

Hematological response rate post 6 months treatment

Secondary Outcome(s)

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

Source(s) of Monetary Support

Janssen Pharmaceutical K.K (Investigational drug provider)

Japanese Ministry of Health, Labour and Welfare

Ethics review

Status: YES
Approval date:
Contact: