NCT00075608

Brief Summary

RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly. PURPOSE: This phase II trial is studying how well autologous stem cell transplant works in treating patients with persistent or recurrent primary systemic (AL) amyloidosis.

Trial Health

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Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_2 multiple-myeloma

Timeline
Completed

Started Aug 2001

Longer than P75 for phase_2 multiple-myeloma

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2001

Completed
2.4 years until next milestone

First Submitted

Initial submission to the registry

January 9, 2004

Completed
3 days until next milestone

First Posted

Study publicly available on registry

January 12, 2004

Completed
7.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2011

Completed
5.3 years until next milestone

Results Posted

Study results publicly available

January 27, 2017

Completed
Last Updated

January 27, 2017

Status Verified

December 1, 2016

Enrollment Period

10.2 years

First QC Date

January 9, 2004

Results QC Date

August 20, 2014

Last Update Submit

December 2, 2016

Conditions

Keywords

primary systemic amyloidosis

Outcome Measures

Primary Outcomes (3)

  • Feasibility and Tolerability

    Feasibility and tolerability will be evaluated based on participants completing second transplant with tolerable adverse events

    3 months after treatment and annually

  • Response and Durability of Response

    Response and durability of response will be based on hematologic Complete Response or Partial Response and date of relapse or death

    3 months after treatment and annually

  • Evaluate Immune Reconstitution

    Evaluate immune reconstitution based on time to engraftment

    3 months after treatment and annually

Study Arms (1)

2nd Stem Cell Transplant

EXPERIMENTAL

Mobilization with filgrastim autologous stem cell transplantation with melphalan conditioning stem cell infusion

Biological: filgrastimDrug: melphalanProcedure: autologous stem cell transplantationProcedure: stem cell infusion

Interventions

filgrastimBIOLOGICAL

16mcg/kg IV daily beginning three days prior to stem cell collection through last day of stem cell collection

Also known as: G-CSF
2nd Stem Cell Transplant

140-200 mcg/kg IV over two days

Also known as: alkeran
2nd Stem Cell Transplant

infusion of previously collected stem cells on Day 0

2nd Stem Cell Transplant

infusion of previously collected stem cells on Day 0

2nd Stem Cell Transplant

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • DISEASE CHARACTERISTICS:
  • Histologically confirmed AL amyloidosis
  • Persistent or recurrent disease after 1 course of prior high-dose chemotherapy
  • Previously treated with autologous stem cell transplantation
  • Significant initial improvement in organ function after prior high-dose melphalan, defined by at least 1 of the following:
  • Complete hematologic remission (e.g., absence of monoclonal spike by immunofixation in serum and urine AND less then 5% plasma cells in bone marrow with no clonal predominance) OR partial hematologic response (e.g., any decrease in serum or urine monoclonal protein OR decrease in bone marrow plasmacytosis)
  • Greater than 50% reduction in proteinuria with preservation of creatinine clearance
  • Greater than 50% reduction in alkaline phosphatase OR at least 2 cm decrease in liver size by physical exam
  • Subjective neurologic improvement, as confirmed by neurologist
  • Cardiac stabilization of disease confirmed by echocardiography defined as less than 2 mm increase in mean wall thickness and/or less than 20 g increase in left ventricular mass
  • Improvement in performance status\* NOTE: \*This criteria alone does not constitute significant improvement in organ function
  • Prior stem cell yield must have been ≥ 2 x 10\^6 CD34+ cells/kg
  • PRIOR CONCURRENT THERAPY:
  • Biologic therapy
  • See Disease Characteristics
  • +27 more criteria

You may not qualify if:

  • No myelodysplastic syndromes
  • No abnormal bone marrow cytogenetics
  • Other
  • Not pregnant or nursing
  • Fertile patients must use effective contraception
  • Acceptable toxicity from first transplantation, confirmed by the transplant team
  • HIV negative
  • No other concurrent malignancy except treated skin cancer

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Boston University Cancer Research Center

Boston, Massachusetts, 02118, United States

Location

MeSH Terms

Conditions

Multiple MyelomaNeoplasms, Plasma CellImmunoglobulin Light-chain Amyloidosis

Interventions

FilgrastimGranulocyte Colony-Stimulating FactorMelphalan

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHematologic DiseasesHemic and Lymphatic DiseasesHemorrhagic DisordersLymphoproliferative DisordersImmunoproliferative DisordersImmune System DiseasesAmyloidosisProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological FactorsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino Acids

Results Point of Contact

Title
Principal Investigator
Organization
Boston Medical Center

Study Officials

  • Karen Quillen, MD

    Boston Medical Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Medical Director, Blood Bank

Study Record Dates

First Submitted

January 9, 2004

First Posted

January 12, 2004

Study Start

August 1, 2001

Primary Completion

October 1, 2011

Study Completion

October 1, 2011

Last Updated

January 27, 2017

Results First Posted

January 27, 2017

Record last verified: 2016-12

Data Sharing

IPD Sharing
Will not share

Locations