Melphalan, Thalidomide, and Dexamethasone in Treating Patients With Newly Diagnosed, Previously Untreated Primary Systemic Amyloidosis

Multiple Myeloma and Plasma Cell Neoplasm Clinical Trial

Official title:

Risk Adapted Intravenous Melphalan and Adjuvant Thalidomide and Dexamethasone for Untreated Patients With Primary Systemic Amyloidosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00089167
• Other study ID #: 02-031
• Secondary ID: MSKCC-02031
• Status: Completed
• Phase: Phase 2
• First received: August 4, 2004
• Last updated: January 15, 2013
• Start date: May 2002

Study information

• Verified date: January 2013
• Source: Memorial Sloan Kettering Cancer Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

RATIONALE: Drugs such as melphalan, thalidomide, and dexamethasone may be effective in treating patients with primary systemic amyloidosis.

PURPOSE: This phase II trial is studying how well giving melphalan together with thalidomide and dexamethasone works in treating patients with primary systemic amyloidosis.

Description:

OBJECTIVES:

Primary

• Determine the 2-year and overall progression-free survival of patients with newly diagnosed, previously untreated primary systemic (AL) amyloidosis treated with risk-adapted melphalan followed by thalidomide and dexamethasone.

Secondary

• Determine plasma cell disease response in these patients at 3, 12, and 24 months after treatment with this regimen.

• Determine amyloid-related disease response in these patients at 12 and 24 months after treatment with this regimen.

• Determine the prognostic significance of immunoglobulin light-chain variable-region germline gene expression by AL plasma cell clones in patients treated with this regimen.

• Determine whether there is molecular minimal residual disease at 12 and 24 months in patients achieving a complete hematologic response after treatment with this regimen.

OUTLINE: Patients are stratified according to the extent of amyloid-related disease (low-risk vs high-risk).

• High-risk disease: Patients receive 2 courses of low-dose melphalan IV, dexamethasone, and filgrastim (G-CSF). After 3 months, patients receive thalidomide and dexamethasone if plasma cell disease persists.

• Low-risk disease: Patients receive 1 course of high-dose melphalan IV and G-CSF. Patients then receive thalidomide and dexamethasone as in high-risk disease regimen.

Patients are followed at 3, 12, and 24 months.

PROJECTED ACCRUAL: A total of 82 patients will be accrued for this study.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. primary completion date: December 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

DISEASE CHARACTERISTICS:

• Diagnosis of primary systemic (AL) amyloidosis within the past 12 months

• High- or low-risk disease, determined by the extent of systemic organ involvement with disease and patient age

PATIENT CHARACTERISTICS:

Age

• 18 and over

Performance status

• SWOG 0-3

Life expectancy

• Not specified

Hematopoietic

• Not specified

Hepatic

• Not specified

Renal

• Not specified

Cardiovascular

• No New York Heart Association class III or IV congestive heart failure

• No restrictive cardiomyopathy requiring oxygen

• No myocardial infarction within the past 6 months

• No symptomatic cardiac arrhythmia within the past 60 days

Other

• No other active 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 cancer in complete remission

PRIOR CONCURRENT THERAPY:

Biologic therapy

• Not specified

Chemotherapy

• No prior chemotherapy for AL amyloidosis

Endocrine therapy

• Not specified

Radiotherapy

• Not specified

Surgery

• Not specified

Other

• No other prior or concurrent therapy for AL amyloidosis

Study Design

Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyloidosis
• Multiple Myeloma
• Multiple Myeloma and Plasma Cell Neoplasm
• Neoplasms, Plasma Cell
• Plasmacytoma

Intervention

Biological:
• filgrastim

Drug:
• dexamethasone

• melphalan

• thalidomide

Locations

Country	Name	City	State
United States	Memorial Sloan-Kettering Cancer Center	New York	New York

Sponsors (2)

Lead Sponsor	Collaborator
Memorial Sloan Kettering Cancer Center	National Cancer Institute (NCI)

Country where clinical trial is conducted

United States, 

References & Publications (1)

Cohen AD, Zhou P, Reich L, et al.: Risk-adapted intravenous melphalan followed by adjuvant dexamethasone (D) and thalidomide (T) for newly diagnosed patients with Systemic AL Amyloidosis (AL): interim results of a phase II study. [Abstract] Blood 104 (11)

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall progression-free survival at 2 years			No
Secondary	Plasma cell disease response at 3, 12, and 24 months after treatment			No
Secondary	Amyloid-related disease response at 12 and 24 months after treatment			No
Secondary	Prognostic significance of immunoglobulin light-chain variable-region germline gene expression by AL cell clones			No
Secondary	Molecular minimal residual disease at 12 and 24 months			No