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

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Nanjing University School of Medicine
Sponsor:
Collaborators:
Soochow University
Nanjing Medical University
Information provided by (Responsible Party):
Zhi-Hong Liu, M.D., Nanjing University School of Medicine
ClinicalTrials.gov Identifier:
NCT01273844
First received: January 3, 2011
Last updated: August 20, 2014
Last verified: August 2014
  Purpose

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.


Condition Intervention
Amyloidosis
Drug: Bortezomib

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Study of the Combination of Bortezomib and Dexamethasone Followed by HSCT in AL

Resource links provided by NLM:


Further study details as provided by Nanjing University School of Medicine:

Primary Outcome Measures:
  • Complete response rate [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    Complete response rate at 12 months post-transplantation


Secondary Outcome Measures:
  • overall response rate [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    overall response rate(ie.,CR + PR) at 12 months post-transplantation


Other Outcome Measures:
  • progression-free survival [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
    progression-free survival (PFS) at 2 years post-transplantation


Estimated Enrollment: 30
Study Start Date: December 2010
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Bortezomib Drug: Bortezomib
Bortezomib
Other Names:
  • Bortezomib
  • autologous hematopoietic stem cell transplantation

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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01273844

Contacts
Contact: Xianghua Huang, Master 0086-025-80860017 ext 210002

Locations
China, Jiangsu
Soochow University Recruiting
Suzhou, Jiangsu, China, 215325
Contact: Xianghua Huang, Master    0086-025-80860017 ext 210002      
Principal Investigator: zhihong Liu, Master         
Sponsors and Collaborators
Zhi-Hong Liu, M.D.
Soochow University
Nanjing Medical University
Investigators
Principal Investigator: zhihong Liu, Master Research Institute of Nephrology, Jinling Hospital, Nanjing University School of Medicine, Nanjing, China
  More Information

No publications provided

Responsible Party: Zhi-Hong Liu, M.D., professor, Nanjing University School of Medicine
ClinicalTrials.gov Identifier: NCT01273844     History of Changes
Other Study ID Numbers: NJCT-1006
Study First Received: January 3, 2011
Last Updated: August 20, 2014
Health Authority: China: Food and Drug Administration

Keywords provided by Nanjing University School of Medicine:
hematopoietic stem cell transplantation
amyloidosis
Bortezomib

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Bortezomib
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 28, 2014