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

NCT ID: NCT02489500

Last Updated: 2018-09-10

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-06-30

Study Completion Date

2017-04-28

Brief Summary

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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.

Detailed Description

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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.

Conditions

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AL Amyloidosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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melphalan

Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion

Group Type ACTIVE_COMPARATOR

Melphalan

Intervention Type DRUG

Conditioning Regimen:

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

Neupogen

Intervention Type DRUG

granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days

Stem Cell Collection

Intervention Type PROCEDURE

collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells

Stem cell infusion

Intervention Type PROCEDURE

infusion of previously collected autologous stem cells

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

Group Type EXPERIMENTAL

Bortezomib

Intervention Type DRUG

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

Melphalan

Intervention Type DRUG

Conditioning Regimen:

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

Neupogen

Intervention Type DRUG

granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days

Stem Cell Collection

Intervention Type PROCEDURE

collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells

Stem cell infusion

Intervention Type PROCEDURE

infusion of previously collected autologous stem cells

Interventions

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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

Intervention Type DRUG

Melphalan

Conditioning Regimen:

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

Intervention Type DRUG

Neupogen

granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days

Intervention Type DRUG

Stem Cell Collection

collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells

Intervention Type PROCEDURE

Stem cell infusion

infusion of previously collected autologous stem cells

Intervention Type PROCEDURE

Other Intervention Names

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VELCADE Alkeran granulocyte colony-stimulating factor (G-CSF) apheresis infusion

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Boston Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Vaishali Sanchorawala

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Vaishali Sanchorawala, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Medical Center

Locations

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Boston Medical Center

Boston, Massachusetts, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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VelRand

Identifier Type: OTHER

Identifier Source: secondary_id

H-33808

Identifier Type: -

Identifier Source: org_study_id

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