HD Melphalan and SCT in Patients With IGDD or LCDD

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: NCT00681044
Recruitment Status : Terminated (Poor accrual)
First Posted : May 20, 2008
Results First Posted : March 15, 2017
Last Update Posted : April 28, 2017
Information provided by (Responsible Party):
Vaishali Sanchorawala, Boston Medical Center

Brief Summary:

RATIONALE: Giving chemotherapy before a stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as G-CSF, and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

PURPOSE: This phase II trial is studying the side effects of high-dose melphalan given together with stem cell transplant and to see how well it works in treating patients with immunoglobulin deposition disease or light-chain deposition disease.

Condition or disease Intervention/treatment Phase
Multiple Myeloma Biological: filgrastim Drug: melphalan Procedure: Stem Cell Infusion Phase 2

Detailed Description:


  • To assess the tolerability of high-dose melphalan and autologous stem cell transplantation in patients with immunoglobulin deposition disease or light-chain deposition disease.
  • To determine the hematologic response rate in patients treated with this regimen.
  • To determine the predictability of early free light-chain response for heme response in patients treated with this regimen.
  • To determine organ or clinical response in patients treated with this regimen.
  • To determine overall survival of these patients.


  • Stem cell mobilization: Patients undergo blood stem cell mobilization comprising filgrastim (G-CSF) subcutaneously once daily for 3 days (i.e., through the day before the last stem cell collection).
  • Stem cell collection: Patients undergo collection of G-CSF-mobilized blood stem cells until the target number of stem cells (at least 2 x 10^6 cluster of differentiation-34-positive cells) is reached.
  • Conditioning regimen: Patients receive high-dose melphalan IV on days -3 to -2.
  • Autologous stem cell transplantation: Patients undergo blood stem cell infusion on day 0.

After completion of study therapy, patients are followed at 3, 6, and 12 months and then annually thereafter.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: High-Dose Melphalan and Autologous Stem Cell Transplantation (HDM/SCT) in Light-Chain Deposition Disease (LCDD) and Immunoglobulin Deposition Disease (IGDD)
Study Start Date : October 2006
Actual Primary Completion Date : January 2016
Actual Study Completion Date : January 2016

Arm Intervention/treatment
Experimental: SCT with melphalan conditioning
Mobilization with Filgrastim Stem Cell Transplant Melphalan Conditioning Stem Cell infusion
Biological: filgrastim
16 mcg/kg daily beginning 3 days prior to SCC through day before final SCC
Other Name: G-CSF, neulasta

Drug: melphalan
70-100 mg/m2/day will be administered intravenously on Days -3 and -2
Other Name: alkeran

Procedure: Stem Cell Infusion
infusion of previously collected stem cells on Day 0

Primary Outcome Measures :
  1. Hematologic Response Rate [ Time Frame: one year ]

Secondary Outcome Measures :
  1. Predictability of Early Free Light-chain Response for Heme Response [ Time Frame: One month ]
  2. Organ or Clinical Response [ Time Frame: One year ]
  3. Overall Survival [ Time Frame: life ]
  4. Tolerability [ Time Frame: 100 days ]

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.

Ages Eligible for Study:   18 Years to 120 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:


  • Histologically confirmed light-chain deposition disease based on the following criteria:

    • Deposition of granular material containing free light-chain (FLC) immunoglobulins that did not bind Congo red
    • Evidence of a plasma cell dyscrasia, as defined by any of the following:

      • Monoclonal gammopathy in the serum or urine by immunofixation electrophoresis
      • Clonal plasmacytosis on bone marrow biopsy by immuno-histochemical
      • Elevated serum levels of FLC
  • Patients may enroll after stem cell collection (SCC) if all prestudy requirements are completed prior to starting SCC (i.e., ≥ 2.5 x 10^6 cells available for transplantation)


  • Prior chemotherapy with alkylating agent allowed provided there is no evidence of myelodysplastic syndromes
  • Prior total dose of melphalan < 300 mg
  • More than 4 weeks since prior cytotoxic therapy and recovered


  • Performance status 0-2
  • Left Ventricular Ejection Fraction (LVEF) ≥ 45% within the past 90 days
  • diffusing capacity of lung for carbon monoxide (DLCO) ≥ 50%

Exclusion Criteria:

  • No overt multiple myeloma, as defined by any of the following:

    • Greater than 30% bone marrow plasmacytosis
    • Extensive (i.e., > 2) lytic lesions
    • Hypercalcemia
  • No myocardial infarction, congestive heart failure, or arrhythmia refractory to therapy within the past 6 months
  • No prior malignancy except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, adequately treated stage I or II cancer from which the patient is currently in complete response, or any other cancer from which the patient has been disease-free for the past 5 years
  • No HIV positivity

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): NCT00681044

United States, Massachusetts
Boston University Cancer Research Center
Boston, Massachusetts, United States, 02118
Sponsors and Collaborators
Boston Medical Center
Principal Investigator: Vaishali Sanchorawala, MD Boston Medical Center

Responsible Party: Vaishali Sanchorawala, Principal Investigator, Boston Medical Center Identifier: NCT00681044     History of Changes
Other Study ID Numbers: CDR0000595782
BHO-H-25876 ( Other Identifier )
BUMC-H-25876 ( Other Identifier: Boston University Medical Center IRB )
First Posted: May 20, 2008    Key Record Dates
Results First Posted: March 15, 2017
Last Update Posted: April 28, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Vaishali Sanchorawala, Boston Medical Center:
monoclonal immunoglobulin deposition disease
light chain deposition disease

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Adjuvants, Immunologic