Know Cancer

or
forgot password

Phase II Trial of Second Autologous Transplantation in AL Amyloidosis


Phase 2
18 Years
65 Years
Not Enrolling
Both
Multiple Myeloma and Plasma Cell Neoplasm

Thank you

Trial Information

Phase II Trial of Second Autologous Transplantation in AL Amyloidosis


OBJECTIVES:

- Determine the feasibility and tolerability of second autologous stem cell
transplantation in patients with persistent or recurrent AL amyloidosis.

- Determine the response rate and durability of response in patients treated with this
regimen.

- Determine immune reconstitution in patients treated with this regimen.

OUTLINE:

- Mobilization: Patients receive filgrastim (G-CSF) subcutaneously (SC) once daily
beginning before the initiation of stem cell collection and continuing until the day
before the completion of stem cell collection.

- Preparative regimen: Patients receive high-dose melphalan IV over 20 minutes on days -3
and -2.

- Autologous stem cell transplantation: Autologous stem cells are reinfused on day 0.

Patients are followed at 6 months, 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 19 patients will be accrued for this study within 5-6 years.

Inclusion Criteria


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

- No myelodysplastic syndromes

- No abnormal bone marrow cytogenetics

- Prior stem cell yield must have been ≥ 2 x 10^6 CD34+ cells/kg

PATIENT CHARACTERISTICS:

Age

- 18 to 65

Performance status

- SWOG 0-2

Life expectancy

- More than 6 months

Hematopoietic

- See Disease Characteristics

Hepatic

- See Disease Characteristics

Renal

- See Disease Characteristics

Cardiovascular

- See Disease Characteristics

- LVEF ≥ 45% by MUGA or echocardiogram

Pulmonary

- DLCO ≥ 50%

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

PRIOR CONCURRENT THERAPY:

Biologic therapy

- See Disease Characteristics

Chemotherapy

- See Disease Characteristics

- No chemotherapy after first transplantation

Endocrine therapy

- Not specified

Radiotherapy

- Not specified

Surgery

- Not specified

Type of Study:

Interventional

Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Feasibility and tolerability

Outcome Time Frame:

3 months after treatment and annually

Safety Issue:

Yes

Principal Investigator

Karen Quillen, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Boston Medical Center

Authority:

United States: Food and Drug Administration

Study ID:

CDR0000347379

NCT ID:

NCT00075608

Start Date:

August 2001

Completion Date:

October 2011

Related Keywords:

  • Multiple Myeloma and Plasma Cell Neoplasm
  • primary systemic amyloidosis
  • Amyloidosis
  • Neoplasms
  • Multiple Myeloma
  • Neoplasms, Plasma Cell
  • Plasmacytoma

Name

Location

Boston University Cancer Research CenterBoston, Massachusetts  02118