Clinical trial NCT00075621: Tandem Autologous Stem Cell Transplantation in Treating Patients With Primary Systemic (AL) Amyloidosis
Official title: A Phase II Trial of Tandem Transplantation in AL Amyloidosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Multiple Myeloma
Keywords: primary systemic amyloidosis
Lead sponsor: Boston Medical Center
Sponsor class: OTHER
First posted: 2004-01-12
Enrollment: 62
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: RATIONALE: Autologous stem cell transplantation may be effective treatment for primary systemic (AL) amyloidosis.

PURPOSE: This phase II trial is studying how well tandem (two) autologous stem cell transplantation works in treating patients with primary systemic (AL) amyloidosis.

Detailed description: OBJECTIVES:

* Determine the tolerability of tandem autologous stem cell transplantation in patients with AL amyloidosis.
* Determine whether this regimen can convert a hematologic non-complete response (CR) to CR in these patients.
* Determine the overall survival of patients treated with this regimen.

OUTLINE:

* First transplantation: Patients receive filgrastim (G-CSF) subcutaneously once daily beginning 3 days before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection. Patients may undergo bone marrow harvest if an inadequate number of peripheral blood stem cells are collected.

Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2. Patients undergo autologous stem cell transplantation (ASCT) on day 0.

* Second transplantation: Within 6-12 months after the first ASCT, patients not achieving a complete response receive high-dose melphalan IV over 20 minutes on days -3 and -2 and a second ASCT on day 0.

Treatment continues in the absence of unacceptable toxicity.

Patients are followed at 3 and 6 months, 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 62 patients will be accrued for this study within 2-3 years.

Eligibility criteria: Inclusion Criteria:

DISEASE CHARACTERISTICS:

* Histologically confirmed AL amyloidosis, meeting 1 of the following criteria:

  * Plasma cell dyscrasia, evidenced by 1 of the following:

    * Monoclonal protein in the serum or urine by immunofixation electrophoresis
    * Plasmacytosis of the bone marrow with monoclonal staining for kappa or lambda light chain isotype
  * Macroglossia with at least 1 other site having biopsy proven amyloidosis and absence of a mutant transthyretin is ruled out

PATIENT CHARACTERISTICS:

Age

* 18 to 65

Performance status

* SWOG 0-2

Life expectancy

* At least 1 year

Hematopoietic

* Not specified

Hepatic

* Not specified

Renal

* Not specified

Cardiovascular

* LVEF ≥ 45% by MUGA or echocardiogram

Pulmonary

* DLCO ≥ 50%

Other

* Not pregnant or nursing
* Fertile patients must use effective contraception
* Able to tolerate 2 courses of high-dose therapy
* HIV negative

PRIOR CONCURRENT THERAPY:

Biologic therapy

* Not specified

Chemotherapy

* Prior alkylating agent chemotherapy allowed provided there is no morphologic or cytogenetic evidence of myelodysplastic syndromes
* Prior total cumulative oral melphalan dose \< 300 mg

Endocrine therapy

* Not specified

Radiotherapy

* Not specified

Surgery

* Not specified

Other

* At least 4 weeks since prior cytotoxic therapy and recovered

Exclusion Criteria:

* No senile, secondary, localized, dialysis-related, or familial amyloidosis
* No overt multiple myeloma (e.g., greater than 30% bone marrow plasmacytosis, extensive \[more than 2\] lytic lesions, hypercalcemia)

Cardiovascular

* No myocardial infarction within the past 6 months
* No congestive heart failure
* No arrhythmia refractory to therapy
* No evidence of symptomatic transient ischemic attacks or strokes
* No other malignancy within the past 5 years except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated stage I or II cancer currently in complete remission

Primary outcomes:
measure: safety
timeFrame: 100 days, 6 months, and annual
Secondary outcomes:
measure: Efficacy
timeFrame: one year
Other outcomes:
Locations:
city: Boston
country: United States
facility: Cancer Research Center at Boston Medical Center
state: Massachusetts
Study officials:
affiliation: Boston Medical Center
name: Vaishali Sanchorawala, MD
role: PRINCIPAL_INVESTIGATOR
References: