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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01998503
First Posted: November 29, 2013
Last Update Posted: November 29, 2013
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Zhi-Hong Liu, M.D., Nanjing University School of Medicine
  Purpose
This randomized phase III trial is studying the side effects and how well giving induction therapy with bortezomib and dexamethasone followed by autologous stem cell transplantation (ASCT) compared with ASCT alone in treating patients with newly diagnosed renal AL amyloidosis. In this prospective, randomized control study, patients with newly diagnosed AL amyloidosis who met the criteria for ASCT were randomized to receive 2 cycles of BD as induction therapy followed by ASCT (BD+ASCT) (arm 1) or to receive ASCT alone as an initial treatment (arm 2). Hematologic and organ responses were evaluated every 3 months after ASCT. All the patients should be followed up for 12 months.

Condition Intervention Phase
Amyloidosis Drug: Bortezomib Drug: dexamethasone Biological: filgrastim Procedure: autologous hematopoietic stem cell transplantation (ASCT) Drug: Melphalan Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:


Further study details as provided by Zhi-Hong Liu, M.D., Nanjing University School of Medicine:

Primary Outcome Measures:
  • 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 Measures:
  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 12 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 ]
  • Overall survival [ Time Frame: 24 months ]
  • Progression free survival [ Time Frame: 24 months ]

Enrollment: 56
Study Start Date: December 2007
Study Completion Date: August 2013
Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: BD induction followed by ASCT
The BD regimen included bortezomib 1.3 mg/m2 i.v. and dexamethasone 40 mg p.o. on days 1, 4, 8 and 11 of the 21 day cycle. This process was repeated for 2 cycles. After two cycles of BD therapy, the collection of peripheral blood stem cells (PBSC) should be completed within 4 weeks. Patients receive filgrastim (G-CSF) on days 1 to 5 and undergo autologous hematopoietic stem cell (HSC) collection. Patients will receive ASCT therapy in 8 weeks after collection of PBSC (Recorded as day 0), while melphalan (day -2) with a dose of 140 or 200 mg/m2 (choosing a dose according to the degree of risk for patients). Melphalan will be administered by central venous catheter.
Drug: Bortezomib
Other Name: Velcade
Drug: dexamethasone
Given orally
Biological: filgrastim
Given subcutaneous
Procedure: autologous hematopoietic stem cell transplantation (ASCT)
Given on day 0
Drug: Melphalan
Experimental: ASCT alone
the patients who assigned to this arm will receive ASCT alone as an initial treatment. At first, patients receive the collection of peripheral blood stem cells (PBSC), Patients receive filgrastim (G-CSF) on days 1 to 5 and undergo autologous hematopoietic stem cell (HSC) collection. After then patients will receive ASCT therapy in 8 weeks after collection of PBSC (Recorded as day 0), while melphalan (day -2) with a dose of 140 or 200 mg/m2 (choosing a dose according to the degree of risk for patients). Melphalan will be administered by central venous catheter.
Biological: filgrastim
Given subcutaneous
Procedure: autologous hematopoietic stem cell transplantation (ASCT)
Given on day 0
Drug: Melphalan

Detailed Description:

Arm 1: The BD regimen included bortezomib 1.3 mg/m2 i.v. and dexamethasone 40 mg p.o. on days 1, 4, 8 and 11 of the 21 day cycle. This process was repeated for 2 cycles. After two cycles of BD therapy, the collection of peripheral blood stem cells (PBSC) should be completed within 4 weeks. Recommended dose of drug is as follows: granulocyte colony-stimulating factor (G-CSF) 5-10ug/kg on days 1-5 will be given, then peripheral blood stem cells will be collected on days 5-6 for 2×10^6 CD34+ cells /kg. Patients will receive ASCT therapy in 8 weeks after collection of PBSC (Recorded as day 0), while melphalan (day -2) with a dose of 140 or 200 mg/m2 (choosing a dose according to the degree of risk for patients). Melphalan will be administered by central venous catheter.

Arm 2: the patients who assigned to arm 2 will receive ASCT alone as an initial treatment. The process of ASCT is as same as arm 1.

  Eligibility

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01998503


Locations
China, Jiangsu
Research Institute of Nephrology, Jinling Hospital, Nanjing University School of Medicine
Nanjing, Jiangsu, China, 210002
Sponsors and Collaborators
Nanjing University School of Medicine
Investigators
Principal Investigator: Zhihong Liu, MD Jinling Hospital, China
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Zhi-Hong Liu, M.D., Professor, Nanjing University School of Medicine
ClinicalTrials.gov Identifier: NCT01998503     History of Changes
Other Study ID Numbers: NJCT-0703
First Submitted: November 15, 2013
First Posted: November 29, 2013
Last Update Posted: November 29, 2013
Last Verified: November 2013

Keywords provided by Zhi-Hong Liu, M.D., Nanjing University School of Medicine:
AL amyloidosis
bortezomib
autologous stem cell transplantation

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Dexamethasone acetate
Dexamethasone
Bortezomib
Melphalan
BB 1101
Lenograstim
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Adjuvants, Immunologic