Melphalan and Dexamethasone With or Without Bortezomib in Treating Patients With Previously Untreated Systemic Light-Chain Amyloidosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT01078454
First received: February 27, 2010
Last updated: November 20, 2014
Last verified: April 2014
Results First Received: November 20, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Light Chain Deposition Disease
Primary Systemic Amyloidosis
Interventions: Drug: melphalan
Drug: dexamethasone
Drug: bortezomib

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Participants were recruited from ECOG member institutions between November 1, 2010 and September 7, 2012.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Arm A (Mel-Dex) Patients receive melphalan 0.22 mg/kg orally (PO) and dexamethasone 40 mg PO on days 1-4 every 4 weeks. Treatment repeats every 4 weeks for up to 9 courses in the absence of disease progression or unacceptable toxicity.
Arm B (B-Mel-Dex) Patients receive melphalan 0.22 mg/kg PO and dexamethasone 40 mg PO on days 1-4 and bortezomib 1.3 mg/m^2 intravenously (IV) on days 1, 4, 8, and 11 every 4 weeks. Treatment repeats every 4 weeks for 2 cycles. Patients then receive melphalan PO and dexamethasone PO on days 1-4 and bortezomib IV on days 1, 8, 15, and 22 every 5 weeks. Treatment repeats every 5 weeks for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Participant Flow:   Overall Study
    Arm A (Mel-Dex)     Arm B (B-Mel-Dex)  
STARTED     6     5  
COMPLETED     5     3  
NOT COMPLETED     1     2  
Adverse Event                 1                 1  
Death                 0                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
All enrolled patients are included in this analysis.

Reporting Groups
  Description
Arm A (Mel-Dex) Patients receive melphalan 0.22 mg/kg orally (PO) and dexamethasone 40 mg PO on days 1-4 every 4 weeks. Treatment repeats every 4 weeks for up to 9 courses in the absence of disease progression or unacceptable toxicity.
Arm B (B-Mel-Dex) Patients receive melphalan 0.22 mg/kg PO and dexamethasone 40 mg PO on days 1-4 and bortezomib 1.3 mg/m^2 intravenously (IV) on days 1, 4, 8, and 11 every 4 weeks. Treatment repeats every 4 weeks for 2 cycles. Patients then receive melphalan PO and dexamethasone PO on days 1-4 and bortezomib IV on days 1, 8, 15, and 22 every 5 weeks. Treatment repeats every 5 weeks for up to 6 cycles in the absence of disease progression or unacceptable toxicity.
Total Total of all reporting groups

Baseline Measures
    Arm A (Mel-Dex)     Arm B (B-Mel-Dex)     Total  
Number of Participants  
[units: participants]
  6     5     11  
Age  
[units: years]
Median (Full Range)
  68    (61 to 78)     66    (44 to 72)     67    (44 to 78)  
Gender  
[units: participants]
     
Female     3     2     5  
Male     3     3     6  



  Outcome Measures

1.  Primary:   Proportion of Patients With Hematologic Overall Response (Partial Response [PR]+ Very Good PR [VGPR]+ Amyloid Complete Response [ACR]+ Stringent Complete Response [sCR]) After 3 Months (3 Cycles) of Therapy   [ Time Frame: Assessed at 3 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Study Statistician
Organization: ECOG Statistical Office
phone: 617-632-3012


No publications provided


Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT01078454     History of Changes
Other Study ID Numbers: NCI-2011-02010, NCI-2011-02010, E4A08, U10CA021115
Study First Received: February 27, 2010
Results First Received: November 20, 2014
Last Updated: November 20, 2014
Health Authority: United States: Food and Drug Administration