Trial of High Dose Melphalan/Stem Cell Transplant With or Without Bortezomib (VelRand)

August 10, 2018 updated by: Vaishali Sanchorawala, Boston Medical Center

Phase III Trial of High-dose Melphalan and Stem Cell Transplantation Versus High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis

Standard treatment for AL Amyloidosis is high-dose melphalan and stem cell transplant.

This study will compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

This study seeks to enroll patients with AL amyloidosis who have been recommended for standard treatment with high-dose melphalan and stem cell transplant.

Standard treatment for this disease is high-dose melphalan and stem cell transplant.

The purpose of this study is to compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis.

Patients enrolled in this study will receive either standard treatment with high-dose melphalan and stem cell transplant, or investigational bortezomib used in combination with standard treatment with high-dose melphalan and stem cell transplant.

Study Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02118
        • Boston Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Histological diagnosis of primary systemic (AL) amyloidosis based on:

    • Deposition of amyloid material by Congo red stain showing characteristic apple green birefringence,AND…
    • evidence of a clonal plasma cell dyscrasia with monoclonal protein in the serum or urine by immunofixation electrophoresis studies AND/OR abnormal serum free light chain assay AND/OR clonal plasma cells in the bone marrow exam demonstrated by immunohistochemistry, flow cytometry or in situ hybridization AND…
    • evidence of organ involvement other than carpal tunnel syndrome. Patients with senile, secondary, localized, dialysis-related or familial amyloidosis are not eligible. Confirmation of tissue diagnosis at all sites of organ dysfunction is encouraged, but not required.
  2. Patients must be > 18 years of age.
  3. Patients must have a performance status of 0-2 by Eastern Cooperative Oncology Group (ECOG) criteria
  4. Patients must have left ventricular ejection fraction (LVEF) > 45% by echocardiogram within 60 days of enrollment
  5. Pulmonary Function Tests must show diffusing capacity of lung for carbon monoxide (DLCO) > 50%.
  6. All patients must be informed of the investigational nature of this study and must sign and give written informed consent in accordance with institutional and federal guidelines.

Exclusion Criteria:

  1. Patients with recent (< 6 months) myocardial infarction, congestive heart failure, New York Heart Association (NYHA) class III/IV or arrhythmia which are refractory to medical therapy are ineligible.
  2. Prior chemotherapy with alkylating agent allowed only if no evidence of Myelodysplastic Dysplastic Syndrome (MDS) morphologically or cytogenetically. Total cumulative dose of oral melphalan must be < 300 mg. Patients should not have received any cytotoxic therapy < 4 weeks prior to registration and should have fully recovered from the effects of such therapy.
  3. Patients must not have overt multiple myeloma (>30% bone marrow plasmacytosis and, extensive (>2) lytic lesions and hypercalcemia).
  4. No other prior malignancy is allowed except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease-free for 5 years.
  5. Patients must not be HIV positive.
  6. Pregnant or nursing women may not participate. Women and men of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: melphalan
Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion
Drug: Melphalan

Conditioning Regimen:

Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0

Other Names:
  • Alkeran
Drug: Neupogen
granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days
Other Names:
  • granulocyte colony-stimulating factor (G-CSF)
Procedure: Stem Cell Collection
collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells
Other Names:
  • apheresis
Procedure: Stem cell infusion
infusion of previously collected autologous stem cells
Other Names:
  • infusion
Experimental: melphalan + Bortezomib
Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion
Drug: Melphalan

Conditioning Regimen:

Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0

Other Names:
  • Alkeran
Drug: Neupogen
granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days
Other Names:
  • granulocyte colony-stimulating factor (G-CSF)
Procedure: Stem Cell Collection
collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells
Other Names:
  • apheresis
Procedure: Stem cell infusion
infusion of previously collected autologous stem cells
Other Names:
  • infusion
Drug: Bortezomib

Conditioning Regimen:

Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1

Other Names:
  • VELCADE

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Hematologic Response
Time Frame: 6 months
Hematologic response defined as: at least 50% improvement in the difference between involved and uninvolved free light chains
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Toxicities
Time Frame: 100 days
Number of serious adverse events per participant based on Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
100 days
Overall Survival
Time Frame: 5 years
duration of overall survival measured in days
5 years
Number of Participants With Organ Response
Time Frame: 5 years
analysis of number of patients with organ response, as defined on page 13 of the detailed protocol for kidney, heart and liver.
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Boston Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2015

Primary Completion (Actual)

April 28, 2017

Study Completion (Actual)

April 28, 2017

Study Registration Dates

First Submitted

March 9, 2015

First Submitted That Met QC Criteria

July 1, 2015

First Posted (Estimate)

July 3, 2015

Study Record Updates

Last Update Posted (Actual)

September 10, 2018

Last Update Submitted That Met QC Criteria

August 10, 2018

Last Verified

August 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • H-33808
  • VelRand (Other Identifier: Boston Medical Center)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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