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A Pilot Study of Lenalidomide, Melphalan and Dexamethasone in AL Amyloidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00890552
Recruitment Status : Completed
First Posted : April 30, 2009
Results First Posted : March 22, 2017
Last Update Posted : March 22, 2017
Celgene Corporation
Information provided by (Responsible Party):
Stanley L Schrier, Stanford University

Brief Summary:
This open-label trial will evaluate the use of lenalidomide; melphalan; and dexamethasone (MDR) to treat newly diagnosed or relapsed AL amyloidosis, over the course of nine 28-day cycles.

Condition or disease Intervention/treatment Phase
Leukemia Amyloidosis Drug: Lenalidomide Drug: Melphalan Drug: Dexamethasone Not Applicable

Detailed Description:
The study will evaluate the 3-drug combination of lenalidomide; melphalan; and dexamethasone (MDR) as the absence of disease progression or toxicity, patients will complete nine 28-day cycles of MDR therapy, with the option of continuing treatment with lenalidomide as single-agent. Patients received up to nine cycles of treatment, with the option to continue on lenalidomide as a single agent if they responded to treatment.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of Lenalidomide, Melphalan and Dexamethasone in AL Amyloidosis
Study Start Date : April 2009
Actual Primary Completion Date : August 2012
Actual Study Completion Date : October 2012

Arm Intervention/treatment
Experimental: Lenalidomide+Melphalan+Dexamethasone
Patients received lenalidomide 10 mg/day orally on days 1-21, melphalan 0.18 mg/kg orally on days 1-4, and dexamethasone 40 mg orally once weekly of a 28-day cycle (MDR treatment).
Drug: Lenalidomide

Lenalidomide is a a derivative of thalidomide.

Orally-administered lenalidomide 10 mg will be taken daily on days 1 to 21 of 28-day cycle.

Other Names:
  • Revlimid
  • L04AX04

Drug: Melphalan

Melphalan is a phenylalanine derivative of mechlorethamine.

Orally-administered melphalan 0.18 mg/kg will be taken on days 1 to 4 of a 28-day cycle

Other Names:
  • Alkeran
  • Evomela
  • Sarcolysin
  • L-phenylalanine mustard (L-PAM)
  • 4-[Bis(2-chloroethyl)amino]-L-phenylalanine

Drug: Dexamethasone

Dexamethasone is an anti-inflammatory and immunosuppressant steroid medication.

Orally-administered dexamethasone 40 mg orally once weekly of a 28-day cycle

Other Names:
  • Intensol
  • Decadron
  • Baycadron
  • Dexpak® Taperpak
  • Maxidex (dexamethasone ophthalmic suspension)
  • Ozurdex (dexamethasone intravitreal implant)

Primary Outcome Measures :
  1. Hematologic Response Rate [ Time Frame: 8 weeks ]
    At the end of each treatment cycle (4 weeks), hematologic response rate as assessed. Hematologic response was considered to be amyloid complete response (normal FLC ratio and negative serum and urine immunofixation); very good partial response (difference between involved and uninvolved FLCs [dFLC] < 40 mg/L); or partial response (dFLC decrease > 50%).

Secondary Outcome Measures :
  1. Overall Survival (OS) [ Time Frame: 12 months ]
    Participants alive 12 months after starting MDR treatment.

  2. Event-free Survival (EFS) [ Time Frame: 12 months ]
    Assessed as the median value for EFS 12 months after starting MDR treatment

  3. Duration of Response [ Time Frame: 32 months ]
    Assessed as the median value for the time from first partial response until progression; death; or last follow-up.

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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


  • Newly diagnosed or relapsed AL amyloidosis
  • Biopsy-proven amyloidosis with evidence of an underlying plasma cell dyscrasia

    • abnormal clonal dominance of plasma cells in the bone marrow
    • detection of a monoclonal gammopathy by immunofixation electrophoresis of serum and/or urine
    • an abnormal serum free light chain or ratio, or AL fibrils seen on biopsy)
  • Measurable disease defined by an abnormal serum-free light chain or monoclonal protein by immunofixation

    • proteinuria ≥ 0.5 g/day, cardiac involvement with interventricular septal thickness ≥ 15 mm
    • hepatomegaly in the absence of congestive heart failure with elevated alkaline phosphatase
  • Age ≥ 18 years at the time of signing the informed consent form.
  • All previous cancer therapy must have been discontinued at least 4 weeks prior to treatment in this study
  • ECOG performance status of ≤ 3 at study entry
  • Laboratory test results:

    • Absolute neutrophil count ≥ 1.0 x 10e9 / L
    • Platelet count ≥ 75 x 10e9 / L
    • Creatinine clearance ≥ 15 mL/ minute
    • Total bilirubin ≤ 2-fold upper limits of normal
  • Disease-free of prior malignancies (excluding multiple myeloma) for ≥ 3 years with exception of:

    • currently treated basal cell
    • squamous cell carcinoma of the skin
    • carcinoma "in situ" of the cervix or breast.
  • Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test
  • Females of childbearing potential must either:

    • commit to continued abstinence from heterosexual intercourse
    • acceptable methods of birth control and agree to ongoing pregnancy testing
  • Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a successful vasectomy
  • All study participants must be registered into the mandatory RevAssist program, and able to comply with its requirements
  • Able to take aspirin (81 mg) daily • Understand and voluntarily sign an informed consent form
  • Able to adhere to the study visit schedule and other protocol requirements


  • Any serious medical condition that would prevent the subject from signing the informed consent form
  • Pregnant
  • breast-feeding females
  • Use of any other experimental drug or therapy within 28 days of baseline
  • Known hypersensitivity to thalidomide
  • Erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs
  • Any prior use of lenalidomide
  • Concurrent use of other anti-cancer agents or treatments
  • Known positivity for human immunodeficiency virus HIV)

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 identifier (NCT number): NCT00890552

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United States, California
Stanford University Cancer Institute
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Celgene Corporation
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Principal Investigator: Stanley L Schrier, MD Stanford University
Publications of Results:
Other Publications:
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Responsible Party: Stanley L Schrier, Professor of Medicine, Stanford University Identifier: NCT00890552    
Other Study ID Numbers: IRB-15213
RV-AMYL-PI-0375 ( Other Identifier: Celgene Reference number )
SU-09192008-1300 ( Other Identifier: Stanford University )
HEM0010 ( Other Identifier: OnCore )
First Posted: April 30, 2009    Key Record Dates
Results First Posted: March 22, 2017
Last Update Posted: March 22, 2017
Last Verified: February 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Immunoglobulin Light-chain Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Dexamethasone acetate
BB 1101
Anti-Inflammatory Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action