HD Melphalan and SCT in Patients With IGDD or LCDD

High-Dose Melphalan and Autologous Stem Cell Transplantation (HDM/SCT) in Light-Chain Deposition Disease (LCDD) and Immunoglobulin Deposition Disease (IGDD)

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00681044

Enrollment

Registered

2008-05-20

Start date

2006-10-31

Completion date

2016-01-31

Last updated

2017-04-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Multiple Myeloma

Keywords

monoclonal immunoglobulin deposition disease, light chain deposition disease

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.

Detailed description

OBJECTIVES: * 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. OUTLINE: * 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.

Interventions

BIOLOGICALfilgrastim

16 mcg/kg daily beginning 3 days prior to SCC through day before final SCC

DRUGmelphalan

70-100 mg/m2/day will be administered intravenously on Days -3 and -2

PROCEDUREStem Cell Infusion

infusion of previously collected stem cells on Day 0

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 120 Years

Healthy volunteers

Inclusion criteria

DISEASE CHARACTERISTICS: * 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 CONCURRENT THERAPY: * 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 PATIENT CHARACTERISTICS: * 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

Design outcomes

Primary

Measure	Time frame
Hematologic Response Rate	one year

Secondary

Measure	Time frame
Predictability of Early Free Light-chain Response for Heme Response	One month
Organ or Clinical Response	One year
Overall Survival	life
Tolerability	100 days

Countries

United States

Participant flow

Recruitment details

Patients were enrolled from the Boston Medical Center Stem Cell Transplant Program between 2006 and 2009.

Pre-assignment details

A patient enrolled on this study but was withdrawn due to insufficient stem cell yield. His clinical situation changed 5 months later and the investigators believed he would have a better yield so he was enrolled for a second time. Therefore, there were 5 enrollments, but 4 patients.

Participants by arm

Arm	Count
SCT With Melphalan Conditioning Mobilization with Filgrastim Stem Cell collection Melphalan Conditioning Stem Cell infusion filgrastim: 16 mcg/kg daily beginning 3 days prior to SCC through day before final SCC melphalan: 70-100 mg/m2/day will be administered intravenously on Days -3 and -2 Stem Cell Infusion: infusion of previously collected stem cells on Day 0	4
Total	4

Baseline characteristics

Characteristic	SCT With Melphalan Conditioning
Age, Categorical <=18 years	0 Participants
Age, Categorical >=65 years	0 Participants
Age, Categorical Between 18 and 65 years	4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	0 Participants
Race (NIH/OMB) Black or African American	0 Participants
Race (NIH/OMB) More than one race	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) White	4 Participants
Sex: Female, Male Female	1 Participants
Sex: Female, Male Male	3 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 4
other Total, other adverse events	4 / 4
serious Total, serious adverse events	4 / 4