Study of Bortezomib +HSCT in Primary Systemic Amyloidosis (AL)

Study of the Combination of Bortezomib and Dexamethasone Followed by HSCT in AL

To evaluate the efficacy and safety of Vel-Dex therapy (bortezomib and dexamethasone) followed by autologous hematopoietic stem cell transplantation as an initial treatment in patients with newly diagnosed AL.

Study Overview

• Status: Completed
• Conditions: Amyloidosis
• Intervention / Treatment: Drug: Bortezomib

Detailed Description

Patients will receive two 21-day cycles of induction therapy with vel / dex regimen. Bortezomib 1.3mg/m2 on days 1, 4, 8 and 11 will be given by intravenous bolus injection while Dexamethasone 40 mg/d will be taken orally on days 1-4. After two cycles of vel / dex therapy, the collection of peripheral blood stem cells (PBSC) should be completed within 4 weeks.Patients will receive ASCT therapy in 8 weeks after collection of PBSC (Recorded as day 0), while melphalan (day -2) with a dose of 100,140 or 200 mg/m2 (choosing a dose according to the degree of risk for patients ) and Vel 1mg/m2 (days -6, -3, +1, +4) will be given. Four additional 21-day cycles of Vel treatment (with a dose of 1.6mg/m2 on day 1 and 8 of the cycle) will be conducted as consolidation therapy during the recommended 60-90 days after HSCT, or after resumption of hematopoietic function ( neutrophil count > 1.5x109/L and platelet count> 50x109/L). Later patients won't need maintenance therapy.

The efficacy criteria are the international standards set with consensus of experts in the Tenth International Symposium on Amyloid and Amyloidosis.

• Study Type: Interventional
• Enrollment (Actual): 21
• Phase: Not Applicable

Contacts and Locations

Study Locations

• China
  • Jiangsu
    • Suzhou, Jiangsu, China, 215325
      • Soochow University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Male or female;
2. aged 18-65 years;
3. Patients with newly diagnosed AL;
4. Appropriate for autologous hematopoietic stem cell transplantation;
5. Abnormal M protein or free light chain detected in serum and / or urine
6. ECOG score 0-2 points;
7. Subjects (or their legal representatives) must signed an informed consent document indicating understanding the purpose of and procedures required for the study and willingness to participate in the study.

Exclusion Criteria:

1. Subjects received systematic treatment with steroids
2. Subjects received plasmapheresis to treat clinical significant hyperviscosity syndrome within 4 weeks before enrollment.
3. Pregnant and breastfeeding women;
4. Subjects suffering from multiple myeloma.
5. hypersensitivity to dexamethasone, bortezomib, mannitol, boron, or heparin (if use catheters);
6. Subjects have severe cardiovascular disease,
7. Subjects have serious physical disease and mental illnesses which may interfere the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Bortezomib; Patients will receive two 21-day cycles of induction therapy with vel / dex regimen. Bortezomib 1.3mg/m2 on days 1, 4, 8 and 11 will be given by intravenous bolus injection while Dexamethasone 40 mg/d will be taken orally on days 1-4.	Drug: Bortezomib; Bortezomib; Other Names: autologous hematopoietic stem cell transplantation

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Complete response rate	Complete response rate at 12 months post-transplantation	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
overall response rate	overall response rate(ie.,CR + PR) at 12 months post-transplantation	12 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
progression-free survival	progression-free survival (PFS) at 2 years post-transplantation	2 years

Collaborators and Investigators

• Sponsor: Zhi-Hong Liu, M.D.
• Collaborators: Nanjing Medical University; Soochow University
• Investigators: Principal Investigator: zhihong Liu, Master, Research Institute of Nephrology, Jinling Hospital, Nanjing University School of Medicine, Nanjing, China

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2011
• Primary Completion (Actual): May 12, 2016
• Study Completion (Actual): May 12, 2016

Study Registration Dates

• First Submitted: January 3, 2011
• First Submitted That Met QC Criteria: January 10, 2011
• First Posted (Estimate): January 11, 2011

Study Record Updates

• Last Update Posted (Actual): April 7, 2017
• Last Update Submitted That Met QC Criteria: April 5, 2017
• Last Verified: April 1, 2017

More Information

Terms related to this study

• Keywords: amyloidosis; Bortezomib; hematopoietic stem cell transplantation
• Additional Relevant MeSH Terms: Metabolic Diseases; Proteostasis Deficiencies; Amyloidosis; Antineoplastic Agents; Bortezomib
• Other Study ID Numbers: NJCT-1006