Bortezomib and Dexamethasone Followed by ASCT Compared With ASCT Alone in Treating Patients With AL Amyloidosis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01998503
• Date of registration: 15/11/2013
• Prospective Registration: No
• Primary sponsor: Nanjing University School of Medicine
• Public title: Bortezomib and Dexamethasone Followed by ASCT Compared With ASCT Alone in Treating Patients With AL Amyloidosis
• Scientific title: Induction Therapy With Bortezomib and Dexamethasone Followed by Autologous Stem Cell Transplantation Versus Autologous Stem Cell Transplantation Alone in the Treatment of AL Amyloidosis
• Date of first enrolment: December 2007
• Target sample size: 56
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01998503
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

China

Contacts

• Name: Zhihong Liu, MD
• Affiliation: Jinling Hospital, China

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with newly diagnosed AL amyloidosis

- Abnormal M protein or free light chain detected in serum and/or urine

- ECOG score 0-2 points

- No absolute neutrophil count of ANC less than or equal to 1000 within 14 days before
enrollment

- No platelet count of less than or equal to 50K within 14 days before enrollment

- Serum bilirubin must lower than 2.0 mg/dl within 14 days before enrollment

- Serum creatinine must lower than 2.0 mg/dl within 14 days before enrollment

- Must have LVEF at least 45% by ECHO within 14 days of enrollment

- Pulmonary Function Tests must show DLCO at least 50%

Exclusion Criteria:

- Subjects have received or are currently receiving systematic treatment with steroids
(not including an emergent short-term use of steroids before randomization up to 4
days, maximum dose of 40mg/d)

- Pregnant and breastfeeding women, delivery term women or unwilling to take birth
control measures during the study

- Subjects suffering from multiple myeloma

- Grade 2 or more than grade 2 peripheral neuropathy or neuropathic pain according to
National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE)
version 3

- Known or suspected hypersensitivity to dexamethasone, bortezomib, mannitol, boron, or
heparin (if use catheters)

- Subjects suffering from uncontrolled or severe cardiovascular disease, including
myocardial infarction, class III-IV heart failure defined by New York Heart
Association (NYHA), uncontrolled angina, clinical significant pericardial disease or
cardiac amyloidosis (Other contraindications are not suitable for transplant
patients) within 6 months before enrollment

- Subjects suffering from serious physical disease and mental illnesses which may
interfere the study

- Subjects receiving other pilot study or treatment within 4 weeks before enrollment



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Amyloidosis

Intervention(s)

Biological: filgrastim

Drug: Bortezomib

Drug: Melphalan

Procedure: autologous hematopoietic stem cell transplantation (ASCT)

Drug: dexamethasone

Primary Outcome(s)

Number of participants with hematologic complete response between BD+ASCT arm and ASCT alone arm in the treatment of AL amyloidosis [Time Frame: 12 months]

Secondary Outcome(s)

Progression free survival [Time Frame: 24 months]

Number of participants with organ responses between BD+ASCT arm and ASCT alone arm in the treatment of AL amyloidosis [Time Frame: 12 months]

Number of Participants with Adverse Events as a Measure of Safety and Tolerability [Time Frame: 12 months]

Overall survival [Time Frame: 24 months]

Secondary ID(s)

NJCT-0703

Source(s) of Monetary Support

Please refer to primary and secondary sponsors