Clinical trial NCT00017680: Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis
Official title: Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyloidosis
Keywords: amyloidosis
arthritis & connective tissue diseases
genetic diseases and dysmorphic syndromes
hematopoietic/lymphoid cancer
oncologic disorders
plasma cell neoplasm
primary systemic amyloidosis
rare disease
Lead sponsor: Office of Rare Diseases (ORD)
Sponsor class: NIH
First posted: 2001-06-06
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation.

II. Determine the toxicity of this regimen in these patients.

Detailed description: PROTOCOL OUTLINE: Patients may receive induction chemotherapy before study entry. Patients then receive filgrastim (G-CSF) or another growth factor for 4-6 days as peripheral blood stem cell (PBSC) mobilization. PBSC (or bone marrow) is harvested over 2-3 days.

Patients receive high-dose melphalan IV over 30 minutes twice daily on days -2 and -1. PBSC and/or bone marrow is reinfused on day 0. Patients receive G-CSF beginning on day 0 and continuing until blood counts recover. This course may be repeated 4-12 weeks later.

Patients are followed every 3 months for 1 year and then annually for 5 years.

Eligibility criteria: PROTOCOL ENTRY CRITERIA:

Disease Characteristics

* Histologically confirmed primary amyloidosis
* Ineligible for other high priority national or international study

Prior/Concurrent Therapy

* Biologic therapy: Concurrent participation in gene therapy trials allowed
* Chemotherapy: Prior chemotherapy allowed No other concurrent chemotherapy
* Endocrine therapy: No concurrent steroids unless given with amphotericin B, for adrenal failure, or for septic shock No concurrent hormones except for non-disease-related conditions (e.g., insulin for diabetes)
* Other: No concurrent barbiturates or acetaminophen Concurrent participation in supportive care trials allowed

Patient Characteristics

* Performance status: ECOG 0-3
* Hepatic: Bilirubin less than 2 times normal
* Renal: Creatinine less than 2.5 mg/dL OR On stable hemodialysis
* Pulmonary: DLCO at least 60% predicted OR Clearance by pulmonologist
* Other: HIV negative

Primary outcomes:
measure: Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction
Secondary outcomes:
measure: Toxicity of high dose chemotherapy regimen
Other outcomes:
Locations:
city: New York
country: United States
facility: Herbert Irving Comprehensive Cancer Center
state: New York
Study officials:
affiliation: Herbert Irving Comprehensive Cancer Center
name: Charles S. Hesdorffer
role: STUDY_CHAIR
References: