Clinical trial NCT00679367: Melphalan, Lenalidomide, and Dexamethasone in Treating Patients With Primary Systemic Amyloidosis
Official title: A Phase II Trial of MRD (Melphalan, Lenalidomide and Dexamethasone) for Patients With 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: 2008-05-16
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: RATIONALE: Drugs used in chemotherapy, such as melphalan and dexamethasone, work in different ways to stop the growth of abnormal plasma cells, either by killing the cells or by stopping them from dividing. Biological therapies, such as lenalidomide, may stimulate the immune system in different ways and stop the abnormal plasma cells from growing. Giving melphalan together with lenalidomide and dexamethasone may be an effective treatment for primary systemic amyloidosis.

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

Detailed description: OBJECTIVES:

Primary

* To determine the tolerability and safety of melphalan, lenalidomide, and dexamethasone, in terms of toxicity, in patients with primary systemic amyloidosis.
* To determine the hematologic response rate in patients treated with this regimen.

Secondary

* To assess organ response in patients treated with this regimen.

OUTLINE: Patients receive oral lenalidomide once daily on days 1-21, oral melphalan once daily on days 1-4, and oral dexamethasone once on days 1, 8, 15, and 22. Treatment repeats every 28 days for up to 12 months in the absence of disease progression or unacceptable toxicity.

After completion of study therapy, patients are followed every 3 months until disease progression and then annually thereafter.

Eligibility criteria: Inclusion Criteria:

DISEASE CHARACTERISTICS:

* Diagnosis of primary systemic amyloidosis

PATIENT CHARACTERISTICS:

* Not pregnant
* Negative pregnancy test
* Able to tolerate an anticoagulation regimen (e.g., 325 mg of aspirin per day, therapeutic warfarin, or low molecular weight heparin)

PRIOR CONCURRENT THERAPY:

* Recovered from prior therapy

  * Permanent or stable side effects/changes allowed
* Prior chemotherapy, thalidomide, lenalidomide, or steroids for amyloidosis allowed
* More than 4 weeks since prior and no other concurrent cytotoxic chemotherapy or radiotherapy

Exclusion Criteria:

* No secondary or familial amyloidosis
* No multiple myeloma (≥ 30% plasma cells in bone marrow biopsy or lytic bone lesions)
* No prior cumulative doses of oral melphalan \> 200 mg
* No more than one prior course of high-dose melphalan with stem cell transplant

Primary outcomes:
measure: Number of Participants With Hematologic Response
timeFrame: one year
Secondary outcomes:
measure: Number of Organs Improved or Stable Based on Description Below:
timeFrame: one year
measure: Number of Participants Removed From Study Due to Toxicities
timeFrame: One year
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston University Cancer Research Center
state: Massachusetts
Study officials:
affiliation: Boston Medical Center
name: Vaishali Sanchorawala, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Sanchorawala V, Patel JM, Sloan JM, Shelton AC, Zeldis JB, Seldin DC. Melphalan, lenalidomide and dexamethasone for the treatment of immunoglobulin light chain amyloidosis: results of a phase II trial. Haematologica. 2013 May;98(5):789-92. doi: 10.3324/haematol.2012.075192. Epub 2012 Nov 9.