Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Registry Information

Alternate Title

Second Autologous Stem Cell Transplant in Treating Patients With Persistent or Recurrent Primary Systemic (AL) Amyloidosis

Basic Trial Information

Phase	Type	Status	Age	Sponsor	Protocol IDs
Phase II	Treatment	Active	18 to 65	Other	BUMC-2001-0156 H-22603, NCT00075608

Objectives

1. Determine the feasibility and tolerability of second autologous stem cell transplantation in patients with persistent or recurrent AL amyloidosis.
2. Determine the response rate and durability of response in patients treated with this regimen.
3. Determine immune reconstitution in patients treated with this regimen.

Entry 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 10⁶ CD34+ cells/kg

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

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

Expected Enrollment

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

Outcomes

Feasibility and tolerability 3 months after treatment and annually
Response and durability of response 3 months after treatment and annually
Evaluate immune reconstitution 3 months after treatment and annually

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.

Trial Contact Information

Trial Lead Organizations

Boston University Cancer Research Center

Karen Quillen, MD, Principal investigator		Ph: 617-638-7828

U.S.A.
Massachusetts
	Boston
	Boston University Cancer Research Center
		Clinical Trials Office - Boston University Cancer Research Center	Ph:  617-353-7571

Registry Information
Official Title		Phase II Trial of Second Autologous Transplantation in AL Amyloidosis
Trial Start Date		2001-08-14
Trial Completion Date		2009-12-01 (estimated)
Registered in ClinicalTrials.gov		NCT00075608
Date Submitted to PDQ		2003-11-24
Information Last Verified		2009-06-07

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