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

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ClinicalTrials.gov Identifier: NCT02489500
Recruitment Status : Terminated (Enrollment held for toxicity evaluation; then closed due to competing trial)
First Posted : July 3, 2015
Results First Posted : August 6, 2018
Last Update Posted : September 10, 2018
Sponsor:
Information provided by (Responsible Party):
Vaishali Sanchorawala, Boston Medical Center

Brief Summary:

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.


Condition or disease Intervention/treatment Phase
AL Amyloidosis Drug: Bortezomib Drug: Melphalan Drug: Neupogen Procedure: Stem Cell Collection Procedure: Stem cell infusion Phase 3

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : June 2015
Actual Primary Completion Date : April 28, 2017
Actual Study Completion Date : April 28, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

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 Name: Alkeran

Drug: Neupogen
granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days
Other Name: granulocyte colony-stimulating factor (G-CSF)

Procedure: Stem Cell Collection
collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells
Other Name: apheresis

Procedure: Stem cell infusion
infusion of previously collected autologous stem cells
Other Name: 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: 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 Name: VELCADE

Drug: Melphalan

Conditioning Regimen:

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

Other Name: Alkeran

Drug: Neupogen
granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days
Other Name: granulocyte colony-stimulating factor (G-CSF)

Procedure: Stem Cell Collection
collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells
Other Name: apheresis

Procedure: Stem cell infusion
infusion of previously collected autologous stem cells
Other Name: infusion




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Toxicities [ Time Frame: 100 days ]
    Number of serious adverse events per participant based on Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0

  2. Overall Survival [ Time Frame: 5 years ]
    duration of overall survival measured in days

  3. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02489500


Locations
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United States, Massachusetts
Boston Medical Center
Boston, Massachusetts, United States, 02118
Sponsors and Collaborators
Boston Medical Center
Investigators
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Principal Investigator: Vaishali Sanchorawala, MD Boston Medical Center
  Study Documents (Full-Text)

Documents provided by Vaishali Sanchorawala, Boston Medical Center:

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Responsible Party: Vaishali Sanchorawala, Principal Investigator, Boston Medical Center
ClinicalTrials.gov Identifier: NCT02489500     History of Changes
Other Study ID Numbers: H-33808
VelRand ( Other Identifier: Boston Medical Center )
First Posted: July 3, 2015    Key Record Dates
Results First Posted: August 6, 2018
Last Update Posted: September 10, 2018
Last Verified: August 2018

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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
Keywords provided by Vaishali Sanchorawala, Boston Medical Center:
AL Amyloidosis
Stem Cell Transplantation
Additional relevant MeSH terms:
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Immunoglobulin Light-chain Amyloidosis
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Paraproteinemias
Bortezomib
Melphalan
Sargramostim
Lenograstim
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Adjuvants, Immunologic